Archive for October, 2020

India just conducted its first successful experiment with savior sibling therapy, in which a baby was conceived through in-vitro fertilization for the purposes of donating bone marrow to an older ailing brother struggling with thalassemia, a condition characterized by low levels of hemoglobin in the blood that requires frequent blood transfusions. While the doctors involved in the therapy celebrated their success this week, some on social media challenged the ethics of such a therapy, in which a baby was essentially birthed to save her sibling.

In this case, the child with the genetic disorder needed a bone marrow transplant to cure his disease, and the chances of a successful cure are higher if coming from a person whose proteins (human leukocyte antigens, or HLA) exactly match those of the child. None of the childs existing family members was a match, further complicating the process of getting a bone marrow transplant an already difficult process to execute. The parents, in an effort to create a perfect bone marrow match for their child, underwent three cycles of in-vitro fertilization, out of which 18 embryos were created, and one perfectly matched that of the child, and was disease-free, using a technique called pre-implantation genetic diagnosis (PGD). The embryo was then implanted in the mothers uterus, carried to term, and a baby girl was born.

We had to wait for the baby to grow. She had to weigh 10 kg before we could draw bone marrow, Deepa Trivedi, program director of Sankalp Bone Marrow Unit in Ahmedabad, told The Hindu. Its been approximately seven months since the transplant, and the older sibling has not needed any more blood transfusions, indicating he has been cured of his thalassemia, his doctors announced.

Savior sibling therapy has already been used in countries such as the United Kingdom, the U.S.A., New Zealand, and France. Its mainly used to cure genetic blood disorders in children, such as sickle cell anemia or as seen in the Indian case, thalassemia major. The main way this is done, which is a departure from the Indian case, is by harvesting stem cells from a newborns umbilical cord, which are then injected into the bone marrow of the sibling with the disease, a practice that works 90% of the time. In case it doesnt, doctors can take bone marrow from the savior sibling as they grow, in a process that is painful but not known to be dangerous.

Related on The Swaddle:

Designer Babies Are Far From Reality, Even After the Gene-Edited Babies in China

The first ethical concern with this practice is treating a baby as a source for spare parts, as a means to an end, as a commodity. A study of the bioethics of savior sibling therapy, published in the Journal of Medical Ethics, surmised that treating a baby as a means to an end was not by itself a concern that devalued the utility of savior sibling therapy, as long as theyre also treated as human beings. Bioethicists surmise that using cord blood, something that is frequently discarded after birth, cannot endanger a newborn, or prove to be an ethical quandary used against the therapy.

But what has happened in the most recent case in India actually complicates the issue, because its not the umbilical cord blood that was harvested from the savior sibling at birth, but bone marrow 10 months into her life, which makes her an organ donor. This traverses thorny territory, as governments strictly regulate organ donation by minors due to issues related to consent. Can a baby consent to donating bone marrow to their sibling, or a 10-year-old consent to donating a kidney to their parent? It depends on where the individual resides, and how old the person being asked to donate is. In India, for example, it was only recently that the Delhi High Court ruled that minors could donate organs or tissues, as long as the procedure didnt pose a danger to their lives, and only in exceptional circumstances. However, where minors are mostly dependent upon their families, an element of coercion can also manifest. Also, determining whether a child rationally consented to donate an organ to their parent, for example, becomes difficult when we factor in the emotional element of their relationship that can perhaps override their judgments about their own safety.

Another concern is the well-being of the savior sibling throughout their life, both physical and psychological. Whats to stop a parent from asking the savior sibling to be on standby for their entire lives for their siblings health, available to be tapped for tissues and organs at any point in their lives? This is the plot of Jodi Picoults My Sisters Keeper (also turned into a film of the same name starring Cameron Diaz), but it is an unlikely scenario in real life, ethics experts have said. The aforementioned organ donation rules can prevent such an exploitative situation from arising, they say, with governments around the world tasked with ensuring the consent of the donor remains at the forefront of organ donation.

The third issue with savior sibling therapy arises out of the process itself if a parent can select an embryo that perfectly matches their child, whats to stop them from selecting an embryo for intelligence, or athleticism? This wades into the territory of the production of designer babies, which is an ethical slippery slope that critics have said goes against the natural reproductive order. However, the bioethics study asserts that the connection between savior sibling therapy and the production of designer babies is less of a slippery slope and more of a reach, as the technology might be similar, but the utility of both poles apart the former is used to save childrens lives, while the latter is a superficial, hypothetical fantasy.

For now, the world of savior sibling therapy, and its perception, remains similar to when parents first selected an embryo to create a savior sibling in the U.S. in 2000. As appeared in a New York Times article at the time, It is the kind of talk heard with every scientific breakthrough, from the first heart transplant to the first cloned sheep. We talk like this because we are both exhilarated and terrified by what we can do, and we wonder, with each step, whether we have gone too far. But though society may ask, How could you? the only question patients and families ask is, How could we not? 20 years later, savior sibling therapy still centers the children that can be saved, while government stipulations around the world try to ensure the savior siblings are protected, cared for, and treated as human beings, like any other child.

While a few critics argue for a ban, the bioethics study sums up the dilemma, and perhaps a solution to this ethical debate given that a ban will be fatal for a section of the population, the onus of proof rests clearly with the prohibitionists who must demonstrate that these childrens deaths are less terrible than the consequences of allowing this particular use of PGD.

You have got to have a very powerful reason to resist the means by which a childs life can be saved.

Read more from the original source:
An Indian Baby 'Savior Sibling' Just Gave Her Brother Bone Marrow. But Is It Ethical? - The Swaddle

Cecelia Ahern, the author of P.S. I love you, once beautifully said: Moments are precious; sometimes they linger and other times theyre fleeting, and yet so much could be done in them; you could change a mind, you could save a life and you could even fall in love.

Helping save lives is what we decided to dedicate some of our lives to at Manchester Marrow.More specifically, we are the student-ran arm of the charity Anthony Nolan, which signs up students/young people (aged 16-30 years) to the stem cell register. This is required in finding matches for patients suffering from blood cancers and blood disorders who desperately need transplants. The more people we sign up for this register, the higher the chance of finding a blood stem cell or bone marrow match.

Anthony Nolan was initially founded by Shirley Nolan in 1974, realising the hardships associated with requiring an urgent bone marrow transplant. This was due to her three-year-old son suffering from a rare blood disorder known as Wiskott-Aldrich Syndrome. This inspired her to set up the worlds first register to match donors with people in desperate need. Today, there are over 800,000 people on Anthony Nolans UK register list, and each of these people could be a potential donor and save a life.

Although there are many resources at hand, without you, theres no cure! In Marrow, we have three important missions: raise awareness of Anthony Nolan and blood cancer within UK universities through our events, encourage every student to join stem cell register through our donor recruitment opportunities, and lastly, raise funds to help support this vital work.

As a student, in addition to signing up to the register, you have the amazing opportunity of volunteering for us and to save a life! One of our most outstanding achievements is signing up over 100,000 people to the Anthony Nolan register and raising over 92,000 in a year. Additionally, 1 in 4 people who go on to donate stem cells is recruited via Marrow!

Being a volunteer for us is no hard work. You could do many things, including spreading the word and talking to people about why they should sign up to the register. Furthermore, you need to inform them what the donation involves if they ever found a match, checking medical backgrounds for donor eligibility, assisting them with cheek swabs, and filling out an application form.

If youre interested in this opportunity, there will be several volunteer training sessions held throughout the year. Unfortunately, due to the current situation, all these events will be held online. We can assure you, however, that were doing our best to make the most of it.

To sign up to the register visit the Anthony Nolan website!

Make sure to follow Manchester Marrows social media accounts to keep updated with all the news and events:

Facebook: @ManchesterMarrow

Facebook: Manchester Marrow Volunteers Group

Instagram: @manchestermarrow

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Looking to save lives? Here's how - The Mancunion

DetailsCategory: DNA RNA and CellsPublished on Friday, 16 October 2020 14:20Hits: 378

First therapy recommended for full marketing authorization in the EU for eligible patients with confirmed diagnosis of late infantile or early juvenile MLD variants

One-time treatment with Libmeldy has been shown to preserve cognitive and motor function in most patients

Libmeldy is backed by data across 35 patients with follow-up of up to 8 years post-treatment, demonstrating the potential durability of HSC gene therapy

BOSTON, MA, USA and LONDON, UK I October 16, 2020 I Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene), an investigational gene therapy for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

The CHMPs positive opinion will now be reviewed by theEuropean Commission(EC), which has the authority to grant marketing authorization for Libmeldy in theEuropean Union(EU). A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a very rare, severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk and interact with the world around them. A majority of these patients pass away in childhood, with palliative care often as their only option.

Todays positive CHMP opinion for marketing authorization of Libmeldy is a remarkable achievement that we share with the MLD community, as it brings us closer to delivering a one-time, potentially transformative therapy for eligible children suffering from this devastating disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. Data from the Libmeldy clinical program have demonstrated the potential for long-term positive effects on cognitive development and maintenance of motor function, translating to individual preservation of motor milestones such as the ability to sit, stand and/or walk without support, as well as attainment of cognitive skills like social interactions and school attendance, at ages at which untreated patients show severe motor and cognitive impairments.

Libmeldy is designed as a one-time gene therapy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, in which the patients own hematopoietic stem cells (HSCs) are selected, and functional copies of the ARSA gene are inserted into the genome of the HSCs using a lentiviral vector before these genetically modified cells are infused back into the patient. The ability of the gene-corrected HSCs to migrate across the blood-brain barrier into the brain, engraft, and express the functional enzyme has the potential to persistently correct the underlying genetic condition with a single treatment.

This is an important milestone toward making the availability of HSC gene therapy a reality for more patients, and it also is extremely rewarding for our multi-disciplinary team at SR-Tiget who has worked relentlessly along this 15-year journey to move the seminal proof of principle studies to the first in-human testing of this therapy, said SR-Tiget director Luigi Naldini, M.D, Ph.D. The robust and durable clinical benefits observed in early-onset MLD patients who received HSC gene therapy are compelling, especially when compared to the natural history of the disease. These results also further illustrate our view that the HSC gene therapy approach has the potential to deliver transformative effects in other storage diseases as well, especially when the cells are designed to overexpress the functional enzyme and provide an enhanced supply of it to the affected tissues.

As a parent, watching your child start down a seemingly normal developmental path only to suddenly and rapidly lose some or all of his or her abilities is heart-wrenching, and the agony is even more acute knowing no approved therapies currently exist for MLD, said Georgina Morton, Chair of ArchAngel MLD Trust. Todays decision to advance Libmeldy to the final EC approval stage represents a huge step forward for the parents of these young children and for all of us in the MLD community.

We are extremely appreciative of the EMAs expedited and thorough review of Libmeldys marketing authorization application, considering the severity of MLD coupled with the limited treatment options available today for young patients, said Anne Dupraz, chief regulatory officer, Orchard Therapeutics. The Agencys collaboration on this assessment is a testament to their broader public health commitment to ensure timely evaluation of new medicines for diseases where a pressing unmet need exists.

Data Supporting the Clinical Profile of Libmeldy

The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Early-onset MLD encompasses the disease variants traditionally referred to as late infantile (LI) and early juvenile (EJ).

Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh (non-cryopreserved) formulation:

Clinical safety was evaluated in 35 patients with early-onset MLD:

Co-primary endpointsThe co-primary endpoints of the integrated efficacy analysis were Gross Motor Function Measure (GMFM) total score and ARSA activity, both evaluated at 2 years post-treatment. Results of this analysis indicate that a single-dose intravenous administration of Libmeldy is effective in modifying the disease course of early-onset MLD in most patients.

Pre-symptomatic LI and EJ patients treated with Libmeldy experienced significantly less deterioration in motor function at 2 years and 3 years post-treatment, as measured by GMFM total score, compared to age and disease subtype-matched untreated patients (p0.008). The mean difference between treated pre-symptomatic LI patients and age-matched untreated LI patients was 71.0% at year 2 and 79.8% at year 3. Similarly, the mean difference between treated pre-symptomatic EJ patients and age-matched untreated EJ patients was 52.4% at year 2 and 74.9% at year 3. Although not statistically significant, a clear difference in GMFM total score was also noted between treated early-symptomatic EJ patients and age-matched untreated EJ patients (28.7% at year 2; p=0.350 and 43.9% at year 3; p=0.054).

A statistically significant increase in ARSA activity in peripheral blood mononuclear cells was observed at 2 years post-treatment compared to pre-treatment in both pre-symptomatic patients (20.0-fold increase; p<0.001) and early-symptomatic patients (4.2-fold increase; p=0.004).

At the time of the integrated data analysis, all treated LI patients were alive with a follow-up post-treatment up to 7.5 years and 10 out of 13 treated EJ patients were alive with a follow-up post-treatment of up to 6.5 years. No treatment-related mortality has been reported in patients treated with Libmeldy.

Key secondary endpointsFor EJ patients who were early-symptomatic when treated with Libmeldy, meaningful effects on motor development were demonstrated when these patients were treated before entering the rapidly progressive phase of the disease (IQ85 and Gross Motor Function Classification (GMFC)1). By 4 years post-disease onset, an estimated 62.5% of treated, early-symptomatic EJ MLD patients survived and maintained locomotion and ability to sit without support compared with 26.3% of untreated early-symptomatic EJ MLD patients, representing a delay in disease progression following treatment with Libmeldy.

A secondary efficacy endpoint that measured cognitive and language abilities as quantified by Intelligence Quotient/Development Quotient (IQ/DQ) found:

Clinical safetySafety data indicate that Libmeldy was generally well-tolerated. The most common adverse reaction attributed to treatment with Libmeldy was the occurrence of anti-ARSA antibodies (AAA) reported in 5 out of 35 patients. Antibody titers in all 5 patients were generally low and no negative effects were observed in post-treatment ARSA activity in the peripheral blood or bone marrow cellular subpopulations, nor in the ARSA activity within the cerebrospinal fluid. Treatment with Libmeldy is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies, the safety profiles of these interventions were consistent with their known safety and tolerability.

About MLD and Investigational Libmeldy

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in thearylsulfatase-A(ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. Currently, there are no approved treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1Libmeldy (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase-A (ARSA) gene), formerly OTL-200, is being studied for the treatment of MLD in certain patients. Libmeldy was acquired from GSK inApril 2018and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their jointSan Raffaele-Telethon Institute for Gene TherapyinMilan, initiated in 2010.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature.Journal of Child Neurology2010, DOI:http://doi.org/10.1177/0883073809341669

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Receives Positive CHMP Opinion for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD) | DNA RNA and...

INTRODUCTION

Over the past decade, there have been great successes in assembling high-performance biological and electronic materials with thin and sophisticated architecture. For example, monolayered cell sheets have shown to reproduce physiological activities of original tissue and exhibit enhanced therapeutic efficacy than individual cells because of increased cell-cell interactions and the presence of an extracellular matrix (14). These cell sheets are being studied extensively to assemble in vitro disease models and treat wounded or defective tissues and organs. Separately, minimizing the thickness of wearable electronic devices enables conformal adhesion without an interfacial gap and, in turn, improves performance for sensing, diagnosis, and therapies (58). However, handling such delicate and thin materials for transport and assembly remains a grand challenge. External forces used for gripping, holding, and discharging such materials often deform, wrinkle, or damage materials (9). Such damage can be avoided by attaching thin materials to sacrificial polymeric supports including water-soluble or thermal release tapes (1012). However, these supports should be removed with chemical or long-lasting heat treatment following the placement of thin materials onto a target site, thus making them not reusable.

Recently, efforts have emerged to transport thin electronic materials by simulating the ability of cephalopods (e.g., octopus and squid) to capture and release their preys (1315). Cephalopods use many muscle-based suction cups, called suckers, on their arms to attain conformal adhesion to target preys in both wet and dried environments (16, 17). Bioelectrical signals control the rapid contraction and relaxation of the soft muscle and, in turn, change the internal pressure of the suckers. However, most material-handling systems that were devised to mimic the suction cups focus on recapitulating the anatomical structure but overlook the roles of the bioelectrical signal for control. Therefore, these strategies require mechanical force to be applied externally to attach and detach materials of interests. In addition, synthetic suction cups made with polydimethylsiloxane (PDMS) or polyurethane acrylates are more rigid than biological suction cups by two or three orders of magnitude (13, 15). Such rigid suction cups require higher external pressure for gripping than biological ones, thus increasing the possibility to damage thin and soft materials. Certain efforts were made to assemble a device that can hold and detach materials with heat by coating porous PDMS with thermally responsive poly(N-isopropyl acrylamide) (PNIPAAm) (14). However, the manipulation process was only possible while submerged in a water bath. In addition, it takes 30 min to hours for the device to move one material from one place to another.

To this end, we demonstrate a soft manipulator that can repeat the holding and unloading of thin and fragile materials within 10 s in response to an electrical signal. We hypothesized that a rapid thermo-responsive, microchanneled hydrogel layered with a microelectric heater would lift and release materials of interests without applying an external force due to temperature-induced internal pressure change in microchannels of the gel (Fig. 1). In addition, gels tailored to be as soft as biological suction cups would allow fast and notable changes in internal pressure in response to small temperature changes while minimizing the amount of force imparted onto the thin material to be transported. We examined this hypothesis by attaching a flexible electric heater, which converts electrical signals into heat, to a microchanneled PNIPAAm hydrogel. We examined the extent that the electrothermal signal controls the shrinkage and expansion of microchannels of the gel along with subsequent pressure change inside microchannels. The resulting soft manipulator was assessed for its ability to lift up and release thin materials onto target tissues promptly in response to the electrothermal signal. These thin materials include therapeutic stem cell sheets and ultrathin, wearable bioelectronic devices.

Schematic illustration of (A) the soft, electrothermally controlled manipulator and (B) the process to transport a thin material using the soft manipulator. (A) The soft manipulator consists of a supporter, flexible heater that can convert electrical current to heat, cyanoacrylate-based wet adhesive, and a thermo-responsive PNIPAAm hydrogel with aligned microchannels. (B) Process to transport materials of interests using the soft manipulator. First, the soft manipulator is lowered to let the gel contact a thin material such as a therapeutic cell sheet or an ultrathin film device. During this step, the heater is turned on to contract microchannels of the gel. Second, the heater is turned off to open microchannels of the gel and generate negative pressure in microchannels. As a consequence, the gel serves to hold, lift up, and transport the thin material. Third, the heater is turned on to close microchannels of the gel and, in turn, generate positive pressure in the microchannels. The positive pressure serves to release the thin material onto the target surface.

We prepared a hydrogel that undergoes a rapid volumetric change in response to a temperature change by introducing anisotropically aligned microchannels into the PNIPAAm gel. The microchanneled gel was assembled by placing the pregelled NIPAAm solution on top of a liquid nitrogen reservoir. Then, ice crystals nucleated from the bottom and grew to the top surface due to the temperature gradient (step 1 in Fig. 2A). Simultaneously, solutes, including NIPAAm monomer, cross-linker, and photo-initiator in the solution, were separated from the growing ice crystals because of the decreased solubility in ice crystals (step 2 in Fig. 2A). This continuous and directional segregation of the solutes formed a cryo-concentrated phase between growing ice crystals. Subsequent exposure of the frozen sample to ultraviolet (UV) lightactivated polymerization and cross-linking reaction fixed the anisotropically aligned PNIPAAm network (step 3 in Fig. 2A) (18, 19). The final washing process with the water removed ice crystals and created a PNIPAAm gel with continuously aligned microchannels (Fig. 2B). The resulting gel exhibited an average microchannel diameter of ~20 4 m and an average wall thickness of 0.2 m in the gel at room temperature (Fig. 2C). The porosity reached 95 1%.

(A) Schematic illustrating the fabrication process of the gel with anisotropically aligned microchannels. The gel is prepared by directional crystallization and subsequent polymerization. (B) Photograph of the resulting microchanneled hydrogel after swelling in water. (C) Microstructure of the gel: (C-1) scanning electron microscopy (SEM) micrograph of the top surface, (C-2) 3D imaging of the microchanneled hydrogel via microcomputed tomography (micro-CT), and (C-3) SEM micrograph of microchannels that connect the top and bottom of the gel. (D) ESR of gels at different temperatures. (E) Compressive elastic moduli of gels. Samples were compressed in parallel with microchannel direction (axial compression) and perpendicular to microchannel direction (radial compression). (F) Time-dependent volumetric changes of microchanneled gel on heating (F-1) and cooling (F-2). The samples were placed on 40 or 25C plate. The resulting volumetric change was recorded. (G) Effective diffusion coefficient of water in gels quantified by the reswelling plot (F-2). * represents the statistical significance of the difference of values between conditions indicated with line (*P < 0.01). Photo credit: Byoungsoo Kim, University of Illinois at Urbana-Champaign.

For comparison, randomly oriented water crystals were created in the PNIPAAm gel by placing the pregelled NIPAAm solution in a freezer at 25C and curing it under UV light. The resulting hydrogel showed a similar porosity to the PNIPAAm gel prepared by directional crystallization. However, the microchannels of varying diameters were oriented randomly (fig. S1). In addition, PNIPAAm gel free of microchannels was prepared by skipping the crystallization step.

We examined the equilibrium swelling ratios (ESRs) of the resulting gels. All samples showed the volumetric swelling change at around 32C, which corresponds to the lower critical solution temperature (LCST) of PNIPAAm (Fig. 2D). The difference in the ESR between 25 and 35C was dependent on the microchannel architecture of the gel. In particular, gels with anisotropically aligned microchannels showed a 2.7-fold higher swelling ratio than those with randomly oriented microchannels and a 1.4-fold higher swelling ratio than those free of microchannels. The elastic modulus of the gel with anisotropically aligned microchannels was dependent on the direction of microchannels (Fig. 2E). The elastic modulus measured by compressing the gel perpendicular to the microchannel was 2.4 kPa, which was twofold lower than that measured by compressing the gel in parallel with the microchannels. In contrast, the gel with randomly oriented microchannels and the gel free of microchannels showed the minimal dependency of the elastic modulus on the direction of compression.

Next, we examined the extent that the microchannel architecture of the gel modulates the volumetric swelling rate in response to temperature change. The gel without microchannels exhibited minimal volumetric change over 10 s when the temperature was increased from 25 to 40C. In contrast, the gel with anisotropically aligned microchannels reduced its volume by 60% within 10 s when temperature increased to 40C (Fig. 2F-1 and movie S1). This heat-triggered shrinkage is attributed to the decrease of the average cross-sectional diameter of microchannels from 20 to 9 m as examined with scanning electron microscope images (fig. S2). The microchannel alignment was maintained during the shrinkage. The gel with randomly oriented microchannels also shrank within 10 s when temperature increased to 40C (Fig. 2F-1). However, the degree of shrinkage was approximately 0.4, which was 20% lower than the gel with anisotropically aligned microchannels. The electron microscopic images showed lots of open voids as well as micropores collapsed incompletely (fig. S3). In contrast, the gel with aligned microchannels exhibited a more uniform decrease in the microchannel diameter and minimal macro-sized voids after heating (fig. S2). This result indicates that micropores of varying diameters and orientation limit heat-induced collapse, thus leading to the decreased volume shrinkage.

Cooling the gel from 40 to 25C resulted in gel expansion. The speed and degree of volumetric expansion were dependent on the microchannel architecture. The gel without microchannels did not recover its original volume even after 1 hour (Fig. 2F-2 and fig. S4). In contrast, both of the gels with microchannels restored their original volume within 10 s due to reswelling. The reswelling plot displayed in Fig. 2F-2 was used to quantify the effective water diffusion coefficient (Fig. 2G). We used the Higuchi equation derived under the steady-state approximation of Ficks law of diffusion as follows (20)Vt=V25(S/V40)(Dt/)1/2(1)where Vt is the volume of a gel at time t, D is an effective diffusion coefficient, and S is an effective surface area. V40 and V25 are the volume of a gel at 40C and 25C, respectively. We assumed that water diffusion occurred exclusively on the gel surface. Anisotropically microchanneled gels had a 75-fold higher water diffusion rate than the gel free of microchannels (Fig. 2G). In addition, the gel with anisotropically aligned microchannels showed a 10% higher water diffusion rate than that of the gel with randomly oriented microchannels.

Separately, a flexible electric (joule) heater was fabricated to be attached to the gel by photolithographic patterning of a copper/polyimide film (thickness, 9-m copper/12-m polyimide). The linewidth and spacing of the copper pattern was kept at 300 m to provide uniform heat across the gel disk (Fig. 3A). The heater was additionally coated with a layer of tin (thickness, ~1 m) to prevent oxidation of the copper at an increased temperature within a humid environment. The heater was then connected to an external power supply with a voltage range of 2 to 5 V (Fig. 3B). The activated temperature was examined using an infrared camera, showing that the heater reached the target temperature at 37C within 5 s after applying a voltage of 2 V (Fig. 3, A and B). After the power was turned off, the temperature was dropped immediately back to 25C. Such electrothermal heater was attached to the gel disk using a cyanoacrylate-based adhesive (21). The bilayered hydrogel-heater construct was finally attached to a three-dimensional (3D) printed supporter (Fig. 3C).

(A) Photograph (top) and a thermal image of the flexible heater captured using an infrared camera (bottom). (B) Temperature change over time at differently applied voltages. The temperature profiles of the heater were obtained using an infrared camera. (C) Structural configuration of the soft manipulator (left) and a photograph of the soft manipulator (right). (D and E) Top: Snapshots of the microchanneled gel in the soft manipulator when the heater was turned on (D) and off (E). Images on the second row represent optical microscopic images of the gel surface when the heater was turned on and off. When the heater was turned on, aligned microchannels of the gel pushed water out while being closed for 20 s (D). When the heater was switched off, the gel in the soft manipulator opened microchannels and pulled water back into microchannels within 20 s (E). Scale bar, 100 m. Photo credit: Byoungsoo Kim, University of Illinois at Urbana-Champaign.

With the resulting electrothermal soft manipulator, we examined the response of the gel disk to the electrical signal. The test was conducted outside water. Figure 3 (D and E) shows the side view of the gel disk and the microstructural changes of the gel surface during the electrically controlled heating and cooling cycle. Switching on the heater triggered shrinkage of microchannels within 10 to 20 s and simultaneously released a fraction of water from the gel (Fig. 3D and movie S2). With the power off, the gel expanded the microchannels and reabsorbed the water within a few seconds (Fig. 3E and movie S2). The shrinkage and expansion of microchannels could be repeated hundreds of times by turning the power on and off. No structural failure of the gel was observed during repeated operation. We further examined the heat transfer through the gel layer placed on the heater at a temperature of 40C (fig. S5A and movie S3). With the heater on, the temperature of the gel increased rapidly from the bottom (point 1 in fig. S5B) to a top (point 4 in fig. S5B) within 20 s. This result confirms heat propagation along the gel thickness direction. The gel temperature increased at a rate of 0.3C/mms, independent of the region of observation. Last, the temperature of the entire gel became equal to that of the heater within 30 s (fig. S5C).

The gel with randomly oriented microchannels also underwent shrinkage and expansion in response to the electrothermal signal. However, the area undergoing microchannel shrinkage and expansion was not as uniform as in the gel with anisotropically aligned microchannels (fig. S6). Therefore, the gel released water locally. The gel without microchannels showed a very slow release and limited absorption of water when the electric heater was on and off, respectively (fig. S7).

The shrinkage and expansion of anisotropically aligned microchannels allowed the gel to grip, lift, and release materials of interest (Fig. 4A and movies S4 and S5). The manipulator with a diameter of 25 mm was used in this study. The manipulation process was conducted as follows. First, we shrank the upper part of the microchannels of the hydrogel by activating the heater (stage 1 in Fig. 4A). During this process, the gel released a fraction of water, thus creating an empty pocket between the heater and residual water in the microchannels. The gel was then placed on a 4-inch-diameter silicon wafer, a model material that should be transported (stage 2 in Fig. 4A). Next, the heater was deactivated to expand the shrunken microchannels and move residual water upward [stage 3 (i) in Fig. 4A]. The subsequently formed vacant space between water within the microchannels and the silicon wafer decreased the pressure inside microchannels, thus making the gel adhere to the silicon substrate. Thus, the soft manipulator could lift the substrate [stage 3 (ii) in Fig. 4A]. Last, with the power on, microchannels adjacent to the heater shrank and pushed water out of the microchannel (stage 4 of Fig. 4A). The subsequent pressure increases inside the microchannels served to dislodge the silicon wafer quickly. This mechanism is distinct from artificial handling systems assembled with an inspiration from anatomy of the cephalopods suction cup. These handling systems, however, require external force to hold and release materials of interest. In contrast, the manipulation process performed by our soft manipulator resembles the neuromuscular actuation in which cephalopods grip and release materials of interests. Through control of electricity, the rapid electrothermal actuation of the gel enabled the manipulator to systematically lift up and release target materials without external forces.

(A) Snapshots showing the transport of a 4-inch-diameter silicon wafer using a soft manipulator (upper images). Schematic illustrating the shrinkage and expansion of microchannels and subsequent water movement in microchannels controlled by the electrothermal signal (bottom images). The operating power of the soft manipulator was 5 W. (B) The time-dependent variation of normal adhesion strength measured by the dynamic mechanical analyzer (DMA) during stages 2 and 3 in (A). An initial contact strength of 0.05 kPa was applied to the soft manipulator for this measurement. (C) Fluorescence images of water in microchannels of the gel. The image was obtained from a 3D z-stack confocal microscope before (top) and after adhesion (bottom) of the soft manipulator to a target surface. The heater was attached to the upper part of the gel. (D) Dependency of the adhesion strengths on the initial load. (E) Variation in the adhesion strength as a function of cycle number. (F) Adhesion strength of the soft manipulator measured with the various target substrates in water and air. An initial contact strength of 0.5 kPa was applied to the soft manipulator using DMA for this measurement. Photo credit: Byoungsoo Kim, University of Illinois at Urbana-Champaign.

The normal pressure development of the gel to the silicon surface was further measured, particularly during stages 2 and 3. This measurement was conducted by attaching the bilayered gel-heater construct to a dynamic mechanical analyzer (DMA) (Fig. 4B). First, the gel was preheated by the heater and brought into contact with a 4-inch silicon wafer (Approaching stage in Fig. 4B). Next, when the power was turned off to expand microchannels, the load was increased in the negative direction for 25 s (Gel expansion stage in Fig. 4B). This bilayered gel-heater construct was then slowly pulled upward at 0.1 mm/s by DMA to monitor the increase of the adhesion strength (Adhesion stage in Fig. 4B). The maximum adhesion strength reached 1.5 kPa. Once the power was turned on before the stress reached 1.5 kPa, the normal adhesion strength decreased quickly to 0 kPa within 5 s (Unloading stage in Fig. 4B).

Without temperature control, the manipulator does not exhibit adhesion. We further examined whether temperature-induced contraction and expansion of microchannels are essential to create adhesion. The soft manipulator preheated to 37C was placed on the silicon wafer immersed in water with controlled temperatures. Then, the heater of the soft manipulator was turned off. At temperatures below LCST of the gel layer (i.e., ~32C), the adhesion strength increased rapidly with decreasing temperatures (fig. S8). This result confirms that temperature of the heating layer in the manipulator controls the degree of expansion of the microchanneled gel layer and, in turn, regulates adhesion strength.

We propose that the electrothermally controlled adhesion of the gel to the silicon wafer results from the pressure difference (P) between two ends of microchannels. We introduced the mixture of rhodamine B and water into microchannels of the gel and monitored the vertical movement of water through the individual microchannel during stage 3 (i) in Fig. 4A. According to the side view of the gel captured with confocal microscopy, the microchanneled gel disk was fully filled with water (Fig. 4C, top). Heating and the subsequent cooling process resulted in the space in the lower part of the microchannel adjacent to the silicon substrate by moving residual water upward in the microchannels (Fig. 4C, bottom). This image is similar to the scheme that represents stage 3 in Fig. 4A. The average height of space in the microchannel was approximately 50 m. The pressure difference of a single microchannel in the gel was quantified with a height of the empty part in the microchannel as followsP=wg(hihf)(2)where w is the density of water, g is the gravitational acceleration, and hi and hf are the height of the space in microchannels when the power was turned on and off, respectively. According to the calculation, each microchannel in the gel produced 0.5 Pa of negative pressure after the cooling process.

The adhesion strength of the gel to the silicon wafer was dependent on the initial load applied to the soft manipulator (Fig. 4D). The maximum adhesion strength reached 65 kPa with the initial pressure of 5.0 kPa. The maximum adhesion strength reached 65 kPa with the initial pressure of 5.0 kPa. To underlie the mechanism, we examined the normal pressure development that varies with the initial contact pressure using a DMA. As shown in fig. S9A, the heated soft manipulator was placed on the target silicon wafer. As soon as the heater was turned off, the gel layer expanded and pushed the silicon wafer more strongly. As a consequence, the normal pressure developed in the opposite direction. The normal pressure increased with the initial contact pressure (fig. S9B). Increasing the initial contact pressure enlarged the effective suction area of the soft manipulator and also augmented the normal pressure.

We also examined the effect of elastic modulus of target materials on the adhesion strength. We prepared alginate hydrogels with elastic moduli of 22.5 and 69.8 kPa as target materials for transport (fig. S10A). As confirmed with the pressure development profiles, with a given initial contact pressure of 0.25 kPa, the soft manipulator exhibited a similar magnitude of the adhesion strength to the alginate gels as well as the silicon wafer with a much higher elastic modulus of 140 to 180 GPa. This result suggests that it is not necessary to vary the initial contact pressure with the target material stiffness (fig. S10B). The adhesion strength was not reduced during the repeated cycles of closure and opening of microchannels (Fig. 4E). No chemical contamination or residue was observed on the silicon wafer after the process (fig. S11). The soft manipulator could transport plastic and glass materials by exerting a similar magnitude of the adhesion strength regardless of material hydrophobicity (Fig. 4F). The soft manipulator functioned to transport materials immersed in aqueous media and those in the air.

Last, we examined the capability of the soft manipulator to lift up, transport, and release ultrathin and delicate materials, such as living cell sheets and ultrathin thin film devices. We prepared a single-layered mouse skeletal myoblast cell sheet on a culture dish. In general, monolayered cell sheets were easily damaged or crumpled when picking up the sheet from the cell culture dish with forceps (Fig. 5A and movie S6). By switching the heater of the soft manipulator on and off, it was possible to lift the myoblast cell sheet and transport them to the new target sites. First, we transferred the cell sheet to a glass dish using the soft manipulator (Fig. 5B). Then, we examined whether the soft manipulator damages the sheet during transplantation. Off-axis deformation and viability of the cell sheet before and after delivering process were measured using the spatial light interference microscopy (SLIM) and the live-dead assay kit, respectively. According to SLIM observation and live-dead assay results, there was no substantial wrinkling nor loss of viability of cells that formed the cell sheet during this transport process (Fig. 5C and fig. S12). This simple transportation process allowed us to fabricate a 3D tissue by stacking multiple myoblast sheets using the soft manipulator (Fig. 5D). The resulting three-layered myoblast tissue showed a dense construct with three different layers.

(A) Snapshots of a process to pick up a skeletal myoblast sheet with forceps. The cell sheet was deformed when picking up the sheet using forceps (right). The cell sheet was stained with methylene blue for visualization. (B) Snapshot of a process to transport the skeletal myoblast sheet onto a glass surface using the soft manipulator. (C) Spatial light interference microscopy (SLIM) images of the cell sheet before (left) and after (right) the transfer, showing off-axis diffraction of the cell sheet. (D) Fluorescence image of a multilayered cell sheet consisting of three different myoblast sheets. The multilayered sheet was prepared by stacking cell sheets using the soft manipulator. (E) Snapshots of a process to transport a skeletal myoblast sheet onto a muscle tissue. It took 30 s for the entire transfer process. (F) Photographs of a rat eye before and after transplantation of a stem cell sheet. The cell sheet transplanted to the corneal epithelium of a rat eye using the soft manipulator. It took 30 s for the entire transfer process. (G) Histological examination of the rat eye before (left) and after (right) a stem cell sheet transfer. Hematoxylin and eosin staining revealed that the stem cell sheet was able to be successfully transplanted onto the anterior corneal surface without substantial interface space generation. Photo credit: Byoungsoo Kim, University of Illinois at Urbana-Champaign.

The soft manipulator allowed us to pick up various types of cell sheets and deliver them rapidly to any target surfaces. As a demonstration, we delivered the myoblast cell sheet to an ex vivo muscle tissue without any structural breakages (Fig. 5E and movie S7). The entire transport process could be completed within 30 s. In contrast, the soft manipulator assembled using a gel with randomly oriented micropores could not uniformly deliver the cell sheet due to the nonuniform micropore shrinkage (fig. S13). We also used the soft manipulator as a device to support atraumatic transplantation of a stem cell sheet to the anterior surface of the cornea. Similar to the myoblast cell sheet, mesenchymal stem cell sheets on a donor substrate could be easily transferred to the corneal epithelium of a rat eye (Fig. 5F). We confirmed the stable attachment of the stem cell sheet to the anterior surface of the cornea, in the position of the corneal epithelium of the rat eye by histological observation (Fig. 5G). A method to atraumatically transplant ex vivo generated stem cell sheets could simplify surgical technique and expand access to corneal epithelial stem cell transplants and it could have useful application in the treatment of corneal epithelial injuries, persistent epithelial defects, limbal stem cell deficiencies, nonhealing corneal ulcers, and blast injuries (22, 23).

In addition, the soft manipulator was used to transport an ultrathin electrophysiological (EP) sensor (thickness, ~1 m) without causing wrinkling. We fabricated the EP sensor that consists of reference, ground, and measurement electrodes allowing high-quality recording of electrocardiogram (ECG) signals (Fig. 6A) (24, 25). Generally, such ultrathin film devices were easily crumpled when picking up from a donor substrate, which typically requires the use of a temporary handling support (fig. S14 and movie S8). By using the soft manipulator, it was possible to controllably transfer the EP sensor from the donor substrate to the surface of the pig heart within a minute (Fig. 6B and movie S9). No substantial wrinkles were observed after completing the transport (Fig. 6C). A waveform generator was used to apply a preprogrammed ECG signals across the pig heart using an Ag/AgCl electrode. The resulting ECG signals captured from the EP sensor were nearly identical to those generated from the waveform generator (Fig. 6D and fig. S15). The Pearsons correlation coefficient of the signals was 0.98.

(A) Device configuration of the ultrathin EP sensor (t = 1 m) tailored for the measurement of ECG signals. (B) Snapshot of a process to transport the device to the surface of the pig heart. It took 30 s to capture and deliver the device onto the pig heart. (C) Photograph of the device transplanted to the pig heart using the soft manipulator. (D) Representative ECG signals measured using the transplanted device. Photo credit: Byoungsoo Kim, University of Illinois at Urbana-Champaign.

Together, this study demonstrates that the soft manipulator assembled by integrating a rapid thermal-responsive microchanneled gel and an electrothermal heater can transport ultrathin biological and electronic materials quickly and safely. The resulting soft manipulator could be switched on and off with electricity to lift and release thin and delicate materials within tens of seconds. This rapid handling could be attained with the electrothermally controlled change in the adhesion force between the soft manipulator and target materials. Such an actuation mechanism is very similar to the muscular action of cephalopod suction cups. Therefore, this soft manipulator is distinct from previous suction cupmimicking platforms that need external force for detachment of materials. In addition, the soft manipulator could move thin materials of interest in both wet and dry conditions. Using this unique functionality, we could assemble multilayered cell sheets and place an ultrathin biosensor to the target tissue without impairing its function.

We envisage that further modification of this soft manipulator with an electronic sensor would allow robots to transport ultrathin materials autonomously. For instance, the resulting smart soft manipulator would be able to monitor the degree of deformation of transporting materials during contact and, in turn, adjust the suction force to a level at which materials retain their structural integrity and functionality. By doing so, the soft manipulator would improve its performance from the standpoint of safety and accuracy of material handling and assembly. We believe that the present design concept may be widely used as a new soft handling tool for the fabrication of ultrathin film devices, tissue engineering, and transplant surgery.

This study demonstrated an electrically controllable soft machinery useful to transport ultrathin, delicate objects, including therapeutic cell sheets and thin, wearable biosensing devices. This system, named as the electrothermal soft manipulator, consisted of a flexible heater attached with a rapid thermo-responsive PNIPAAm hydrogel disk with controlled microchannel architecture and tissue-like softness. Compared with hydrogels free of microchannels or those with randomly oriented microchannels, the anisotropically aligned PNIPAAm hydrogel could shrink and expand in response to the electrically induced heat much faster, on the order of seconds. Such a fast-volumetric change of the microchannels on the surface of an object could produce and remove pressure-induced adhesion repeatedly. This controlled actuation mechanism is similar to the activity of cephalopod suction cups that hold and release objects of interest using bioelectric signals. As a consequence, the soft manipulator could move thin biological and bioelectronic devices quickly in both wet and dry conditions without causing wrinkling or damage of the thin materials. Such an electrothermally controlled soft manipulator would be useful to various applications that require the sophisticated manipulation of fragile and delicate biological tissues and bioelectronic devices.

NIPAAm (1.25 g) and N,N-methylenebisacrylamide {12.5 mg [0.01 weight % (wt %) of NIPAAm]} were dissolved in distilled water (8.75 ml) for 1 day at 25C to ensure the complete dissolution. Then, 25 mg (0.5 wt % of NIPAAm) of radical photo-initiator (Irgacure 2959) was added into the obtained solution and stirred until all the solids completely dissolved. The resulting pregelled NIPAAm solution was poured onto a Si-wafer substrate (4 inches, 550 m thick) with silicone mold (50 mm by 50 mm by 1 mm or 20 mm by 20 mm by 10 mm). Then, the Si-wafer substrate was put on a liquid nitrogen reservoir for the directional crystallization of the pregelled NIPAAm solution. The distance between the bottom surface of the Si-wafer and the top surface of liquid nitrogen was 1 cm. After complete crystallization of the pregelled NIPAAm solution, the samples were irradiated with a UV lamp ( = 365 nm) for 6 hours at a 25C freezer for the radical cryo-polymerization. The as-prepared poly-NIPAAm gel (PNIPAAm) was then washed with fresh water three times to remove the ice crystals.

For comparison, PNIPAAm gel with randomly oriented microchannels was prepared by placing the pregelled NIPAAm solution in a freezer at 25C for random crystallization. Then, the resultant samples were cryo-polymerized and washed at the same condition described above. PNIPAAm gel free of microchannels was prepared by skipping the crystallization and subsequently irradiated with a UV lamp for 1 hour at 4C. All hydrogel samples were soaked in 250-ml distilled water at 25C, which was repeatedly replaced for 1 day to remove unreacted impurities before using them.

The morphology of microchanneled PNIPAAm gels was examined using an environmental scanning electron microscope (ESEM; Quanta FEG 450, FEI) and microcomputed tomography (micro-CT, MicroXCT-200, Xradia Inc.). For cross-sectional analysis, the samples were immersed in liquid nitrogen for 30 min and immediately cryo-fractured. One hundred points from 10 different ESEM images were taken to determine the average pore size. The porosity of gels was determined by the gravimetric method. The pore volume of gels was divided by the total volume of gels as followsPorosity(%)={(WswollenWdry)/w}/{(WswollenWdry)/w+(Wdry/PNIPAAm)}(3)where Wswollen and Wdry are the weights of swollen and dry gels, respectively; w is the water density; and PNIPAAm is the NIPAAm density (1.1 g/cm3).

For ESR measurement, we measured the weight of PNIPAAm gels at different temperatures (4 to 40C) with 4C increments. The ESR was defined using the following equationESR(%)={(WsWd)/Wd}100(4)

The hydrogel samples were equilibrated at each temperature for 12 hours and weighted (Ws) after removing excess water. The dry weight of the samples (Wd) was measured after lyophilization. Five samples of each PNIPAAm gel were averaged.

For dynamic deformation analysis of hydrogels in response to temperature change, hydrogel samples immersed in 25C were trimmed into a cylinder shape (d = 25 mm, t = 1 mm) and placed on a copper plate (t = 1 mm). Then, the plate was put onto a heated Peltier stage (40C) to investigate the deswelling kinetics of samples. For reswelling kinetics, deswelled samples were transferred to a cooled Peltier stage (25C). We monitored the volume change in response to temperature using an optical microscope that connected with the Peltier device (TP104SC-mK2000A, Instec). All optical images were analyzed using ImageJ software.

The compressive modulus of hydrogels was measured on an electronic universal testing machine (Instron 5943, Instron) equipped with a water bath. Samples were cut into a square shape (10 mm by 10 mm by 10 mm). All mechanical tests were conducted in a water bath (25C). There were five replicates for all mechanical tests.

The heater was fabricated on a copper/polyimide film (t = 9 m/12 m, Pyralux AC091200EV, Dupont). A standard photolithographic patterning with a dry film photoresist (Riston MM540, Dupont) followed by the wet etching method (CE-100, Transene Inc.) defined the copper layer into a joule heating element. The copper traces were coated with 1-m layer of tin (Sn) (421 Liquid Tin, MG Chemicals) to protect the copper from oxidation in elevated temperatures within a humid environment. The resulting heater was then connected to an external power supply, where a voltage range of 2 to 5 V and its thermal characterizations over time were recorded using an infrared camera (E40, FLIR Systems).

The cyanoacrylate-based adhesive was spread on top of the flexible heating array (21). Immediately after, the hydrogel was trimmed into a cylinder shape (d = 25 mm, t = 1 mm) and pressed onto the substrate. The bonding occurs within 30 s. The resulting gel/heater was attached to a 3D printed supporter using double-sided tape (VHB, 3M). Then, the soft manipulator was connected to an electrical power supply.

For dynamic deformation analysis of the soft manipulator in response to activation of a heater, a monochrome camera (DS-Qi2, Nikon) was attached to an optical microscope (Eclipse LV100, Nikon) for top-view analysis of the gel in the soft manipulator. A digital camera with an optical zoom macro lens (Canon, MP-E 65 mm) was used for the side view analysis of the soft manipulator. Gels in the soft manipulator were incubated with colored water (Green, McCormick) for visualization of water.

Adhesion tests were performed with a DMA (ESM303, Mark-10). The soft manipulator was mounted on a load cell of the DMA (M5-5 or M5-200, Mark-10), and the vertical approach and retraction speeds of the soft manipulator were 0.1 mm/s. Force-displacement profiles with time were measured at room temperature.

To examine the capability of the soft manipulator to handling materials with different elastic moduli, alginate hydrogels with elastic moduli of 23 and 70 kPa were used in this study. Pregelled alginate solution was prepared by mixing 2 wt % alginate solution in MES buffer (pH 6.5) with sulfonated N-hydroxysuccinimide and 1-ethyl-3-(3-dimethylaminopropyl) carbodiimide. Then, the pregelled alginate solution was cross-linked by adding adipic acid dihydrazide (AAD). The elastic modulus of the alginate gels was controlled by varying the molar ratio between AAD and uronic acids of alginate (MAAD).

Cross-sectional fluorescence images were obtained from 3D z-stack confocal images (LSM 880, Carl Zeiss). We used Rhodamine B mixed with water for tracking of water inside the soft manipulator before and after the attachment process.

To investigate surface contamination of the soft manipulator, we performed adhesion tests to silicon wafers using either the soft manipulator or a commercial medical grade tape (Transpore, 3M). After detachment of the samples, the resulting wafer was incubated with a dye (Rhodamine B) for 30 min. All samples were washed with distilled water three times in total. Then, we dried the wafer surface using N2 gas and subsequently observed the wafer surfaces using fluorescent optical microscopy.

C2C12 cells (mouse skeletal myoblast cell line, CRL1772) and D1 cells (bone marrowderived mesenchymal stem cell line, CRL12424) were obtained from the American Type Culture Collection (ATCC). C2C12 or D1 cells were plated on temperature-responsive PNIPAAm-grafted culture dishes (d = 35 mm, UpCell, Thermo Fisher Scientific) with seeding density of 5 105 cells. The cells were then cultivated for 3 days according to the guidelines of ATCC. To harvest sheets, confluent cells were rinsed twice with warmed Dulbeccos phosphate-buffered saline (DPBS). Then, the monolayers were detached from the culture dish by lowering the incubation temperature from 37 to 20C.

The viability of cell sheets was examined using the LIVE/DEAD Viability/Cytotoxicity Assay Kit for mammalian cells (Invitrogen) according to the manufacturers instructions. The cultured cells or transferred cells were gently washed three times with DPBS. Calcein acetoxymethyl (AM) and ethidium homodimer-1 (EthD-1) were diluted together in DPBS. Diluted calcein AM and EthD-1 solution (1 ml) was added to cultured cells and kept for 45 min at room temperature. The live cells were stained with calcein AM, and dead cells were stained with EthD-1. After staining, cells were gently washed with 1 DPBS for three times and imaged with a fluorescence microscope (LSM-880, Carl Zeiss). Off-axis deformation of the cell sheets before and after the delivery process was quantified using SLIM. The optical system was assembled by attaching a SLIM module (CellVista SLIM Pro, Phi Optics) to the output port of an existing inverted phase-contrast microscope (26).

C2C12 cells were cultured onto a temperature-responsive culture dish to produce cell sheets as described above. After incubation, confluent cells were stained with Cell Tracker Orange CMRA (Invitrogen) or calcein AM (Invitrogen). Then, cell sheets were detached from the culture dish by lowering the incubation temperature from 37 to 20C. The detached cell sheets were captured and transferred using the soft manipulator with electrical heater control. A multilayered cell sheet was fabricated by repeating the transfer procedure. The resulting multilayered tissue structure was imaged using a fluorescence microscope (LSM-880, Carl Zeiss).

Long-Evans/BluGill rats were used in this study. All experimental protocols were in compliance with the National Institutes of Health Public Health Service Policy on Humane Care and Use of Laboratory Animals and were approved by the University of Illinois at Urbana-Champaign (UIUC) Institutional Animal Care and Use Committee. For fixation of the cornea, the perfusion needle was inserted into the left ventricle of the heart. A cut was made within the right atrium to allow blood evacuation. Saline was injected at a rate of 300 ml/min to clear the blood from the rat, followed by injection of paraformaldehyde (PFA) at 300 ml/min. The perfusion was confirmed by checking PFA dripping from the nose of the rate, stiffening of the extremities and the liver, and contractures of the musculature. After completing the perfusion, the stem cell sheet was placed on the rats cornea using the soft manipulator. The other rat eye was used as a control. Enucleation was then performed using microscissors.

Enucleation was followed by placement of the eyeball on dry ice then into a mold. The mold was subsequently filled with an optimal cutting temperature (OCT) compoundembedding medium to ensure OCT. Cryosectioning at 40-m slices was performed using a cryostat. Slices were then fixed using 4% PFA because the eyeball was fixed but not the stem cell sheet. The sample was washed three times in tris-buffered saline (TBS) for 5 min. The section was stained with hematoxylin and eosin staining, followed by dehydration in citrasol for 5 min. The stained tissue section was imaged using Axio Zoom.V16.

The fabrication of the EP sensor began by spin-coating a layer of poly(methyl methacrylate) (PMMA; ~1 m thick) on a glass substrate, followed by thermal annealing at 180C for 1 min. A subsequent layer of polyimide (~1 m thick) was coated and cured in a vacuum oven at 250C for 1 hour. Thin films of Cr and Au (t = 5 nm/150 nm thick) were deposited by using an electron beam evaporation. Photolithographic patterning using a negative-type photoresist (Riston MM540, DuPont) followed by wet etching with Au and Cr etchants (Transene) defined the joule-heating element. The resulting structure was submerged in acetone to dissolve the bottom PMMA layer. An anisotropic conductive film (ACF; HST-9805-210, Elform) was bonded to the terminals and was connected to an external data acquisition system. The measurement of ECG signals began by attaching two commercial conducting electrodes (30 mm by 24 mm, H124SG, Kendall) diagonally across the pig heart. The electrodes were then connected to an arbitrary waveform generator (3390, Keithley) to apply a preprogrammed cardiac waveform (1-Hz frequency, 50-mV amplitude). The EP sensor was transferred onto the surface of the pig heart with the soft manipulator. The sensor was connected to an external preamplifier (Octal Bio Amp, ADInstruments) and data acquisition unit (PowerLab 16/35, ADInstruments), where the captured ECG signal was digitally filtered with a band-pass filter at the bandwidth of 0.5 to 100 Hz.

Acknowledgments: Funding: This work was supported by the National Science Foundation (STC-EBICS grant nos. CBET-0939511 and CBET-1932192), the National Institutes of Health (1R21 HL109192), the Department of Defense Vision Research Program under Award (W81XWH-17-1-022), and the Jump ARCHES endowment through the Health Care Engineering Systems Center at University of Illinois at Urbana-Champaign and partly by Korea Institute of Science and Technology-Europe. C.H.L. is funded by the NIH National Institute of Biomedical Imaging and Bioengineering (NIBIB: 1R21EB026099-01A1). E.E.H. acknowledges the financial support from the University of Illinois Beckman Institute Graduate Fellowship. H.C. and N.M. gratefully acknowledge funding support from the National Science Foundation under award no. 1554249 and the International Institute for Carbon Neutral Energy Research (WPI-I2CNER), sponsored by the Japanese Ministry of Education, Culture, Sports, Science and Technology. V.K.A. acknowledges financial support from National Institutes of Health, National Eye Institute K08 EY024339 and R01EY029409; Department of Defense, Congressionally Directed Medical Research Program, Vision Research Program W81XWH-17-1-0122 (V.K.A. and H.K.); Veterans Affairs Office of Research and Development I01BX004080; Unrestricted Grant from Research to Prevent Blindness, New York; and National Institutes of Health, National Eye Institute P30 EY001792. Author contributions: B.S.K. and H.K. designed this project and wrote the manuscript. M.K.K., Y.P., and C.H.L. developed the flexible heater and performed the electrophysiology recordings. Y.C. and S.G.I. supported the cell sheet preparation. E.E.H. and M.U.G. performed and analyzed animal experiments. H.C. and N.M. helped to investigate electrothermal actuation of the gel. K.M.S. and L.B.S. supported and advised on animal experiments. V.K.A., K.K., and K.-N.S. advised on the tissue transplantation. C.H. and G.P. performed SLIM observation of the cell sheet, W.C.B. and S.Y. performed adhesion tests, B.S.K. performed all other experiments. J.L., C.H.L., and H.K. supervised the project. All authors discussed the results and contributed to the final version of the manuscript. Competing interests: H.K., B.S.K., C.H.L., J.L., and M.K.K. are inventors on a provisional patent application related to this work filed by the University of Illinois at Urbana-Champaign, Purdue University, and Chung Ang University (filed on 31 August 2020; no. 63/072,634). The authors declare no other competing interests. Data and materials availability: All data needed to evaluate the conclusions in the paper are present in the paper and/or the Supplementary Materials. Additional data related to this paper may be requested from the authors.

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Electrothermal soft manipulator enabling safe transport and handling of thin cell/tissue sheets and bioelectronic devices - Science Advances

by Manasi Vaidya in New York.

Fate TherapeuticsandCelyadsnatural killer (NK) cell biology-focused cell therapies could overcome cell persistence challenges and consequent efficacy concerns with redosing strategies, experts said.

One of Fate Therapeutics lead products, FT596, is an allogeneic, multitargeted, chimeric antigen receptor (CAR) NK cell product. Celyads autologous CYAD-01 and CYAD-02 and allogeneic CYAD-101 are CAR T cell products using NK cell specificity to target T-cells. One analyst considered the potential to redose allogeneic products as a key item to consider while assessing clinical potential. While clinical data establishing the additive efficacy advantages of giving multiple doses is still preliminary, redosing allogeneic products could increase their expansion and persistence, experts said. Autologous therapies carry source constraints, so the ability to manufacture and administer allogeneic therapies is an advantage, they said.

While past NK cell therapy data has been mixed, experts saw potential in CAR NKs like FT596 or CAR T-cell products engineered to express NKG2D like CYAD-101, given the advancements in cell production.

Phase I FT596 results in B-cell lymphomas/ CLL are expected at either the American Society of Hematology (ASH) meeting in December or an investor meeting in early 2021, as per a second analyst report. Phase I data for CYAD-01 and CYAD-02 in relapsed/refractory (r/r) acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) are expected by YE20, as per the companys August corporate presentation. Celyads allogeneic CYAD-101 is being tested in a Phase I alloSHRINK trial (NCT03692429) in metastatic colorectal cancer (CRC), which has a primary completion date of November 2020.

FT596s sales are expected to reach $136m in 2026, according to a GlobalData Consensus forecast. Celyad did not respond to a request for comment.

Increasing the persistence of cell therapies once they are infused into a patient has been a challenge, especially with NK cell-based therapies, experts said. The issue of persistence and consequent efficacy is significant because the potential efficacy with Celyad and Fate Therapeutics platforms remains largely unknown, they added.

Because the immune system can recognise foreign cells, cell products would not last for more than a few weeks, said Dr Marco Davila, medical oncologist, in the Department of Blood and Marrow Transplantation, Moffitt Cancer Center, Tampa, Florida. With CAR T-cell therapies, the expansion and persistence of CAR cells are said to correlate with the durability of response, said Dr David Sallman, assistant member, Department of Malignant Hematology, Moffitt Cancer Center.

Strategies involving multiple doses of cell therapies could maximise the total dose, improve duration, and increase efficacy magnitude with both autologous and allogeneic cell therapies, said Dr Tara Lin, associate professor of medicine, University of Kansas Medical Center, Kansas City. Multiple infusions of therapy could also potentially lead to complete remission, said Sallman. In Fate Therapeutics Phase I FT500 (NCT03841110) study, patients had been given up to six doses of the therapy, which was not found to be toxic, according to Fate Therapeutics CEO Scott Wolchko. Redosing has the potential to offer multiple infusions as maintenance therapy, said Dr Jeffrey Miller, professor of Medicine, Division of Hematology, Oncology and Transplantation, University of Minnesota, Minneapolis.

The persistence of allogeneic therapies is not well understood, and it is unknown how long cells need to persist to be effective or whether persisting cells confer durability of response, said Wolchko. Giving multiple doses is one way to overcome the lack of persistence if it is an important factor for efficacy, he said. In a 4Q19 call, the FDA said it was allowing the dose to be repeated on a patient-by-patient basis, Wolchko said. In the alloSHRINK study, CYAD-101 is administered three times with a two-week interval between each administration in metastatic CRC, as per ClinicalTrials.gov.

However, even if the engineered cells do not persist in the body, the response rate and ability to eradicate the disease should not be limited, said Davila. With a limited lifespan, allogeneic cell therapies would dissipate as the patients immune system recovers, said Dan Kaufman. With the incorporation of interleukin (IL)-12 or IL-15 into the cell product, the cell therapy could persist without exogenous cytokines, said Kaufman. The FT596 construct contains an IL-15 fusion protein.

Experts cited the data from a Phase I / II (NCT03056339) investigator-led effort at MD Anderson Cancer Center using cord blood-derived anti-CD19 CAR NK cells as an example of an effective CAR NK therapy. The study by Rezvani and colleagues showed a persistence challenge did not seem to hamper the response, because once a critical threshold for cell expansion is crossed, the activity can be mediated, Davila said. Eleven r/r patients with CD19-positive cancers, such as non-Hodgkins lymphoma or CLL, were treated with a single infusion; eight had a response, including seven with a complete remission (Rezvani et al. [2020] N Engl J Med, 382, pp. 545553). Even if the cells do not persist, they expand to sufficient levels to eradicate the disease before they are lost, Davila added.

In the Phase I THINK(NCT03018405) CYAD-01 data, decreased bone marrow blasts were observed in eight patients, including five objective responses and one stable disease for three or more months, as per the company presentation. Responding patients did have blast clearances, but some of the remissions were short-lived and the cells did not persist in the system, said Sallman. However, the short hairpin (sh) ribonucleic acid (RNA) technology employed CYAD-02, which could increase persistence and expansion, said Sallman (Fontaine et al., [2019]Blood, 134[Suppl 1], p. 3931). ShRNA technology allows T cell engineering without the need for gene editing to inhibit alloreactivity and increase persistence, according to Celyad.

Ongoing research on improving preconditioning regimens by combining additional drugs could also help with the persistence of allogeneic products, said Davila. It is not known whether every dose needs a conditioning regimen, but since conditioning regimens can suppress a patients immune system for several months, it may not be necessary before every therapy infusion, he added.

Patients will not have to receive a preconditioning regimen before every cell infusion, said Wolchko, adding redosing FT500 was found to be safe. Celyads protocol does not specify the preconditioning strategy for redosing. No predictive biomarkers are available to explain why some patients respond well and others do not, said Sallman, adding it is critical to identify potential responders. Nonetheless, there is no way to predict clinical efficacy based only on preclinical data, so data is still needed, said Miller.

The economic advantage to developing off-the-shelf therapies has driven interest in NK-cell based platforms, said Miller and Davila. If quick treatment is needed, then an allogeneic NK cell therapy would be better than an autologous therapy, which may take up to six weeks to manufacture, said Sallman. While the results with autologous CAR T-cell therapies have been significant, their scale-up and costs are challenging, said Kaufman. Related Report

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The ability to use induced pluripotent stem cell (iPSCs) or cord blood cells as a source would help scale up the cell manufacture and allow effective results, said Kaufman. iPSCs provide advancement in expansion protocols, which can provide multiple doses, Miller added. Fate Therapeutics has an iPSC-derived NK cell franchise. Also, since T cell therapies require donor apheresis to collect cells in a process lasting four to five hours, it is not feasible to keep going back to the same donor, said Miller.

Moreover, newer platforms are expected to improve on past NK cell therapy trials, specifically those showing mixed efficacy. Past studies had feasibility limitations in getting the required number of cells, said Miller. Those small studies were conducted at a time when cell isolation and production systems were not as advanced as they are now, said Davila.

Manasi Vaidya is a Senior Reporter for Clinical Trials Arena parent company GlobalDatas investigative journalism team. A version of this article originally appeared on the Insights module of GlobalDatas Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.

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Fate Therapeutics' and Celyad's CAR therapies in oncology offer potential - pharmaceutical-technology.com

LOS ANGELES--(BUSINESS WIRE)--Glancy Prongay & Murray LLP (GPM) reminds investors of the upcoming December 7, 2020 deadline to file a lead plaintiff motion in the class action filed on behalf of investors who purchased or otherwise acquired Mesoblast Limited (Mesoblast or the Company) (NASDAQ: MESO) securities between April 16, 2019 and October 1, 2020, inclusive (the Class Period).

If you suffered a loss on your Mesoblast investments or would like to inquire about potentially pursuing claims to recover your loss under the federal securities laws, you can submit your contact information at https://www.glancylaw.com/cases/mesoblast-limited/. You can also contact Charles H. Linehan, of GPM at 310-201-9150, Toll-Free at 888-773-9224, or via email at shareholders@glancylaw.com to learn more about your rights.

Mesoblast develops allogeneic cellular medicines using its proprietary mesenchymal lineage cell therapy platform. Its lead product candidate, RYONCIL (remestemcel-L), is an investigational therapy comprising mesenchymal stem cells derived from bone marrow. In February 2018, the Company announced that remestemcel-L met its primary endpoint in a Phase 3 trial to treat children with steroid refractory acute graft versus host disease (aGVHD).

In early 2020, Mesoblast completed its rolling submission of its Biologics License Application (BLA) with the FDA to secure marketing authorization to commercialize remestemcel-L for children with steroid refractory aGVHD.

On August 11, 2020, the FDA released briefing materials for its Oncologic Drugs Advisory Committee (ODAC) meeting to be held on August 13, 2020. Therein, the FDA stated that Mesoblast provided post hoc analyses of other studies to further establish the appropriateness of 45% as the null Day-28 ORR for its primary endpoint. The briefing materials stated that, due to design differences between these historical studies and Mesoblasts submitted study, it is unclear that these study results are relevant to the proposed indication.

On this news, the Companys share price fell $6.09, or approximately 35%, to close at $11.33 per share on August 11, 2020, on unusually heavy trading volume.

On October 1, 2020, Mesoblast disclosed that it had received a Complete Response Letter (CRL) from the FDA regarding its marketing application for remestemcel-L for treatment of SR-aGVHD in pediatric patients. According to the CRL, the FDA recommended that the Company conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. The CRL also identified a need for further scientific rationale to demonstrate the relationship of potency measurements to the products biologic activity.

On this news, the Companys stock fell $6.56, or 35%, to close at $12.03 per share on October 2, 2020, on unusually heavy trading volume.

The complaint filed in this class action alleges that throughout the Class Period, Defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts about the Companys business, operations, and prospects. Specifically, Defendants failed to disclose to investors: (1) that comparative analyses between Mesoblasts Phase 3 trial and three historical studies did not support the effectiveness of remestemcel-L for steroid refractory aGVHD due to design differences between the four studies; (2) that, as a result, the FDA was reasonably likely to require further clinical studies; (3) that, as a result, the commercialization of remestemcel-L in the U.S. was likely to be delayed; and (4) that, as a result of the foregoing, Defendants positive statements about the Companys business, operations, and prospects were materially misleading and/or lacked a reasonable basis.

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If you purchasedor otherwise acquired Mesoblast securities during the Class Period,you may move the Court no later thanDecember 7, 2020 to ask the Court to appoint you as lead plaintiff. To be a member of the Class you need not take any action at this time; you may retain counsel of your choice or take noaction and remain an absent member of the Class. If you wish tolearn moreabout this action, or if you have any questions concerning this announcement or your rights or interests with respect to these matters, please contactCharlesLinehan, Esquire, of GPM, 1925 Century Park East, Suite 2100, Los Angeles California 90067 at 310-201-9150, Toll-Free at 888-773-9224, by email toshareholders@glancylaw.com, or visit our website atwww.glancylaw.com. If you inquire by email please include your mailing address, telephone number and number of shares purchased.

This press release may be considered Attorney Advertising in some jurisdictions under the applicable law and ethical rules.

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Glancy Prongay & Murray LLP Reminds Investors of Looming Deadline in the Class Action Lawsuit Against Mesoblast Limited (MESO) - Business Wire

Mercks New VirusExpress Platform Speeds Development of Cell and Gene Therapies

Darmstadt, Germany, October 13, 2020 Merck, a leading science and technology company, has bolstered its viral vector manufacturing capabilities with the launch of its VirusExpress Lentiviral Production Platform. This new platform helps to overcome lentiviral production challenges and can reduce process development time by approximately 40 percent, based on Mercks experience as a contract development and manufacturing organization.

Cell and gene therapies offer the potential for curative treatments and are being developed and commercialized in half the time it has taken traditional therapies, said Angela Myers, head of Gene Editing & Novel Modalities, Life Science, at Merck. We are committed to accelerating manufacturing of cell and gene therapies with the ultimate goal of getting these lifesaving treatments to patients faster. By increasing dose yields and dramatically reducing process development time, this new platform will help us reach this goal.

Using a suspension cell line rather than an adherent-based production, coupled with a chemically defined cell culture media and process with built-in scalability, Mercks VirusExpress Platform meets multiple market needs. In addition to accelerating process development, the suspension culture format allows each batch of virus to be larger yielding more patient doses. Additionally, suspension culture is amenable to true scale-up, while being less labor-intensive. The chemically defined medium eliminates the safety, regulatory and supply chain concerns related to animal- and human-derived materials.

Mercks VirusExpress Platform offers a simplified upstream workflow, making processes easier to manage, adjust and scale. Flexible licensing allows companies to manufacture vectors by using either Mercks contract manufacturing capabilities, a third-party contract development and manufacturing organization, or in-house development.

The Life Science business of Merck is a leading contract development and manufacturing organization combining an integrated portfolio of manufacturing solutions with proven commercialization experience. This new offering underscores Mercks continued investment in cell and gene therapies. In April 2020, the company announced a new 100 million, 140,000-square-foot manufacturing center at its Carlsbad, California, USA, location that will double the existing production capacity and support large-scale commercial manufacturing. Today, the Life Science business of Merck manufactures vectors for two of the first five FDA-approved cell and gene therapies.

The cell and gene therapy market is growing rapidly and continues to show great promise. According to market research leader Arizton, the cell and gene therapy market is expected to reach more than $6.6 billion by 2024[1]. Merck has been involved in this space since clinical trials for gene therapy began in the 1990s.

Operator manufacturing viral vector in a cGMP environment. Mercksnew VirusExpressPlatformincreases dose yields and reduces process development time for cell and gene therapies.

All Merck news releases are distributed by email at the same time they become available on the Merck Website. Please go to http://www.merckgroup.com/subscribe to register online, change your selection or discontinue this service.

About Merck

Merck, a leading science and technology company, operates across healthcare, life science and performance materials. Around 57,000 employees work to make a positive difference to millions of peoples lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2019, Merck generated sales of 16.2 billion in 66 countries.

Scientific exploration and responsible entrepreneurship have been key to Mercks technological and scientific advances. This is how Merck has thrived since its founding in 1668. The founding family remains the majority owner of the publicly listed company. Merck holds the global rights to the Merck name and brand. The only exceptions are the United States and Canada, where the business sectors of Merck operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials.

[1] http://www.prnewswire.com/news-releases/the-cell-and-gene-therapy-market-to-reach-revenues-of-over-6-6-billion-by-2024---market-research-by-arizton-300957463.html

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PHILADELPHIA, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (NASDAQ: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today announced publication of data in a murine model of GM1 gangliosidosis (GM1) demonstrating that a single intracerebroventricular injection of an optimized adeno-associated virus (AAV) into the cerebral spinal fluid (CSF) resulted in significant expression of Beta-galactosidase (-gal) in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, neurological impairment and improvement in survival. These data were published online ahead of print in the November issue of the peer-reviewed scientific journal Human Gene Therapy (HGT).

This study suggests that delivery of an AAV vector optimized to express b-gal directly into the CSF restored b-gal activity in the brain and, if further developed and tested in human clinical trials, may be effective in modifying and preventing the devastating effects of the genetic disease GM1, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio. The AAV vector used in the study is the same as Passage Bios PBGM01 gene therapy, which is designed to deliver a functional human GLB1 gene into the brain and optimized to express -gal. These preclinical study data support the further development of PBGM01 as a potential therapy for patients suffering from GM1.

GM1 is a rare and often life-threatening monogenic lysosomal storage disease caused by mutations in the GLB1 gene, which encodes lysosomal acid -gal. Reduced -gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressing neurodegeneration. GM1 manifests as a continuum of disease and is most severe in the infantile form, which is characterized by onset in the first six months of life with hypotonia (reduced muscle tone), progressive CNS dysfunction, and rapid developmental regression. Life expectancy for infants with GM1 is two to four years, and infantile GM1 represents approximately 60 percent of the incidence of 0.5 to 1 in 100,000 live births. Currently, there are no approved disease-modifying therapies available.

Results of the PBGM01 preclinical study were reported in the paper titled, A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis, by Christian Hinderer, M.D., Ph.D., and colleagues, including gene transfer pioneer Dr. Wilson, from the Gene therapy Program, Department of Medicine, University of Pennsylvania Perlman School of Medicine. The study in part was previously presented at the 22nd annual Meeting of the American Society for Cell and Gene Therapy (ASCGT) in 2019.

This research evaluated the impact of single intracerebroventricular administration of the human -gal containing AAV vector on -galactosidase enzyme activity in the murine brain and peripheral tissues, lysosomal storage lesions, neurological function (including neurological exams and gait analysis) and survival in mice lacking the -galactosidase gene. The mice received the single administration at age one month and were evaluated over 300 days. -gal activity was increased significantly in the cerebral spinal fluid and serum of the vector-treated mice compared to vehicle control-treated mice. Significant improvements in gait assessments as measured by stride length and hind paw print length and significant preservation of neurological function as measured by neurological exam scores were observed throughout the study period in the human -gal vector-treated mice. There were significant decreases in lysosomal storage lesions of vector-treated animals and by day 300 all animals that received the two highest doses were still alive, whereas none of the vehicle control-treated animals had survived.

Were excited about being able to soon advance PBGM01 into the clinic, and the potential promise it holds for patients with GM1, the majority of whom are infants and for whom there are no approved disease modifying treatments, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. Our plan is to administer PBGM01 through intra-cisterna magna delivery into the brain, which we believe may offer several benefits in terms of safety, efficiency and distribution compared to other approaches.

Passage Bio expects to initiate dosing of PBGM01 in a Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

This research was supported by a research, collaboration and license agreement with Passage Bio. HGT is the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies. Click here to read the full-text article on the HGT website.

About PBGM01PBGM01 is an AAV-delivery gene therapy currently being developed for the treatment of infantile GM1, in which patients have mutations in the GLB1 gene causing little or no residual -gal enzyme activity and subsequent neurodegeneration. PBGM01 utilizes a next-generation AAVhu68 capsid administered through intra-cisterna magna (ICM) to deliver a functional GLB1 gene encoding -gal to the brain and peripheral tissues. By reducing the accumulation of GM1 gangliosides, PBGM01 has the potential to halt or prevent neuronal toxicity, thereby restoring developmental potential. In preclinical models, PBGM01 has demonstrated broad brain distribution and wide uptake of the -gal enzyme in both the central nervous system (CNS) and critical peripheral organs, suggesting potential treatment for both the CNS and peripheral manifestations of GM1. The Company has received Orphan Drug and Rare Pediatric Disease designation for PBGM01 for patients with GM1 and expects to initiate dosing of its Phase 1/2 trial late in the fourth quarter of 2020 or early in the first quarter of 2021 and remains on track to report initial 30-day safety and biomarker data late in the first half of 2021.

About Passage BioPassage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia and Krabbe disease.

University of Pennsylvania (Penn)Financial DisclosureDr. Wilson is a Penn faculty member and also a scientific collaborator, consultant and co-founder of Passage Bio. As such, he holds an equity stake in the company, receives sponsored research funding from Passage Bio, and as an inventor of certain Penn intellectual property that is licensed to Passage Bio, he may receive additional financial benefits under the license in the future. He is an inventor of intellectual property covering the technology described in paper published in HGT that is licensed from Penn to Passage Bio, and he may receive financial benefits under this license in the future. Penn also holds equity and licensing interests in Passage Bio.

Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including our planned IND submissions, initiation of clinical trials and the availability of clinical data from such trials; our expectations about our collaborators and partners ability to execute key initiatives; our expectations about manufacturing plans and strategies; our expectations about cash runway; and the ability of our lead product candidates to treat the underlying causes of their respective target monogenic CNS disorders. These forward-looking statements may be accompanied by such words as aim, anticipate, believe, could, estimate, expect, forecast, goal, intend, may, might, plan, potential, possible, will, would, and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials;; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions caused by the coronavirus pandemic; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with theSecurities and Exchange Commission(SEC), and other reports as filed with theSEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:Sarah McCabe and Zofia MitaStern Investor Relations, Inc.212-362-1200sarah.mccabe@sternir.comzofia.mita@sternir.com

Media:Gwen FisherPassage Bio215.407.1548gfisher@passagebio.com

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Passage Bio Announces Publication of Preclinical Data That Show Single Injection of Optimized AAV Vector into Cerebral Spinal Fluid - BioSpace

The success of the approved gene therapies has led to an upward surge in the interest of biopharmaceutical developers in this field, resulting in a significant boost in clinical research initiatives and several high value acquisitions

Roots Analysis has announced the addition of Gene Therapy Market (3rd Edition), 2019-2030 report to its list of offerings.

Encouraging clinical results across various metabolic, hematological and ophthalmic disorders have inspired research groups across the world to focus their efforts on the development of novel gene editing therapies. In fact, the gene therapy pipeline has evolved significantly over the past few years, with three products being approved in 2019 alone; namely Beperminogene perplasmid (AnGes), ZOLGENSMA (AveXis) and ZYNTEGLO (bluebird bio). Further, there are multiple pipeline candidates in mid to late-stage (phase II and above) trials that are anticipated to enter the market over the next 5-10 years.

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Key Market Insights

Around 470 gene therapies are currently under developmentNearly 45% of pipeline drugs are in the clinical phase, while rest are in the preclinical / discovery stage. Gene augmented therapies presently represent 66% of the total number of such interventions that are in the pipeline. It is worth mentioning that majority of such product candidates are being developed as in vivo gene therapies.

More than 30% of clinical stage pipeline therapies are being designed for treating oncological disordersConsidering the overall pipeline, over 20% of product candidates are being developed to treat various types of cancers, followed by those intended for the treatment of metabolic (15%) and ophthalmic disorders (12%). It is also worth highlighting that adenovirus vectors are presently the preferred vehicles used for the delivery of anticancer gene therapies.

Over 60% of gene therapy developers are based in North AmericaOf the 110 companies developing gene therapies in the abovementioned region, 64 are start-ups, 26 are mid-sized players, while 18 are large and very large companies. Further, within this region, most of the developers are based in the US, which has emerged as a key R&D hub for advanced therapeutic products.

More than 31,000 patents have been filed / published related to gene therapies, since 2016Of these, 17% of patent applications / patents were related to gene editing therapies, while the remaining were associated with gene therapies. Leading assignees, in terms of the size of intellectual property portfolio, include (industry players) Genentech, GSK, Sangamo Therapeutics, Bayer and Novartis, (non-industry players) University of California, Massachusetts Institute of Technology, Harvard College, Stanford University and University of Pennsylvania.

USD 16.5 billion has been invested by both private and public investors, since 2014Around USD 3.3 billion was raised through venture capital financing, representing 20% of the total capital raised by industry players till June 2019. Further, there have been 28 IPOs, accounting for more than USD 2.2 billion in financing of gene therapy related initiatives. These companies have also raised significant capital in secondary offerings.

30+ mergers / acquisitions have been established between 2014 and 2019Examples of high value acquisitions reported in recent past include the acquisition of AveXis by Novartis (2018, USD 8,700 million) and Bioverativ by Sanofi (2018, USD 11,600 million).

North America and Europe are anticipated to capture over 85% of market share by 2030With a promising development pipeline and encouraging clinical results, the market is anticipated to witness an annualized growth rate of over 40% during the next decade. In addition to North America and Europe, the market in China / broader Asia Pacific region is also anticipated to grow at a relatively faster rate.

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Key Questions Answered

The USD 10 billion (by 2030) financial opportunity within the gene therapy market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom gene therapies are likely to be the most promising treatment options for genetic disorders. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles, featuring an overview of the therapy, current development status and clinical results. Each profile includes information on therapeutic indication, targeted gene, route of administration, special designations, mechanism of action, dosage, patent portfolio, technology portfolio, clinical trials and recent developments (if available).

For additional details, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapy-market-3rd-edition-2019-2030/268.html

or email [emailprotected]

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Tag: Gene Therapy - The Think Curiouser

Prophecy Market Insights presented the Personalized Gene Therapy Treatment market research report which severs comprehensive and iterative research methodology. The company focuses on minimizing deviance in order to offer the most accurate estimations and forecast possible. The company utilizes a combination of bottom-up and top-down approaches for calculation and authenticate of the market size and for estimating quantitative aspects of the market.

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Parameters involved in the Personalized Gene Therapy Treatment market includes:

Segmentation Overview:

Personalized Gene Therapy Treatment Market Key Players:

Amgen, Inc., Chengdu Shi Endor Biological Engineering Technology Co., Ltd., SynerGene Therapeutics, Inc., Cold Genesys, Inc., Bellicum Pharmaceuticals, Inc., Takara Bio, Inc.,Ziopharm Oncology, Inc., , Sevion Therapeutics, Inc., OncoSec Medical, Inc., and Burzynski Clinic.

The report provides an in-depth geographical analysis of the Personalized Gene Therapy Treatment market, covering important regions, viz, North America, Europe, Asia Pacific, Middle East & Africa, and Latin America. It also covers key countries (regions), viz, U.S., Canada, France, Germany, U.K., Italy, Russia, India, China, Japan, South Korea, Australia, Taiwan, Thailand, Indonesia, Malaysia, Vietnam, Philippines, Mexico, Brazil, GCC, Israel, South Africa, etc.

The competitive analysis section of the report includes prominent players of the Personalized Gene Therapy Treatment market that are broadly studied on the basis of several key factors.

Highlights of the Report

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The report on the Personalized Gene Therapy Treatment market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have been included in the report.

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Personalized Gene Therapy Treatment Market: Dynamics, Segment, Application and Supply Demand Analysis 2020-2030 - The Think Curiouser

For an outstanding business growth, companies must take up market research report service which is vital in todays market place. An influential Gene Therapy Market report also offers top to bottom examination of the market for estimating income, return on investment (ROI) and developing business strategies. This market research report helps out the business in every sphere of trade to take the unmatched decisions, to tackle the toughest business questions and diminish the risk of failure. The industry report highlights general market conditions, estimates market share and possible sales volume of industry. The facts and figures described in this Gene Therapy Market document aids industry in taking sound decisions and planning advertising and sales strategy more successfully.

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Market Analysis: Global Gene Therapy Market

Global gene therapy market is rising gradually with a healthy CAGR of 36.1% in the forecast period of 2019-2026. Increasing incidence of cancer and rare life threatening diseases and strong clinical pipeline drugs for gene therapy are major drivers for market growth.

Key Market Players:

Few of the major competitors currently working in the globalgene therapy marketarePfizer Inc., Thermo Fisher Scientific Inc., F. Hoffmann-La Roche Ltd, Spark Therapeutics, Inc., bluebird bio, Inc., ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc., Sarepta Therapeutics, Novartis AG, MeiraGTx Limited, Rocket Pharmaceuticals, Lonza, Biogen, Gilead Sciences, Inc., REGENXBIO Inc., uniQure N.V., Solid Biosciences Inc., Audentes Therapeutics among others.

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Market Definition: Global Gene Therapy Market

Gene therapy is a technique of insertion of genes into cells and tissues for treatment of any disease. In this technique the defective gene is replaced with a functional gene. It is the strategy of manipulation of expression of specific genes responsible for the disease. This therapy is a promising treatment option for a number of diseases. The application of gene therapy is wide and it is mostly used for treatment of cancer, cystic fibrosis, heart disease, diabetes, AIDS among others.

Gene Therapy Market Drivers

Gene Therapy Market Restraints

Segmentation:Global Gene Therapy Market

Gene Therapy Market : By Type

Gene Therapy Market : By Gene Type

Gene Therapy Market : By Viral Vector

Gene Therapy Market : By Non-Viral Vector

Gene Therapy Market : By Application

Gene Therapy Market : By End Users

Gene Therapy Market : By Distribution Channels

Gene Therapy Market : ByGeography

Key Developments in the Market:

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Gene Therapy Market : Share, Size, Regional Trend, Future Growth, Forecast || Leading Players ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics,...

Pune, Maharashtra, India, October 14 2020 (Wiredrelease) MarketResearch.Biz :The Global Gene Therapy Market Outlook to 2029, Capacity, Generation, Investment Trends, laws, and Company Profiles. The business analysis specialists that provide comprehensive data and understanding of the Gene Therapy market within the globe.

The report provides an in-depth analysis of the world Gene Therapy market with forecasts up to 2029. The report analyzes the market state of affairs and provides future outlook with forecasts up to 2029. The report highlights capability and generation trends from 2017 to 2029 in Gene Therapy market. in-depth coverage of the market with specific policies regarding Gene Therapy is provided within the report. The analysis in addition provides company snapshots of a variety of the most market participants.

For Better Understanding, Request A Free Pdf Sample Copy Of Gene Therapy Market Here@ https://marketresearch.biz/report/gene-therapy-market/request-sample

(***Our FREE SAMPLE COPY of the report offers a quick advent to the studies report outlook, TOC, a listing of tables and figures, an outlook to key players of the market, and comprising key regions.)

Further, the report acknowledges that in these growing and promptly enhancing the market surroundings, the foremost recent advertising and promoting details are vital to conclude the performance within the forecast amount and create the essential selections for profitableness and growth of the Gene Therapy market. additionally, the report contains An array of things that impact the expansion of the world Gene Therapy market within the forecast amount. Further, this specific analysis additionally concludes the impact on the individual segments of the market.

Prominent players of Gene Therapy including:

Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

Download Now And Browse Complete Information On The COVID 19 Impact Analysis On Gene Therapy Market:https://marketresearch.biz/report/gene-therapy-market/covid-19-impact

Global Gene Therapy Market Segmentation:

By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

The Gene Therapy market report consists of associate analysis of the market size for price in Million USD and volume in elements. The analysis report estimate and validate the market size of Gene Therapy market, completely different all different dependent sub-markets inside the general Gene Therapy trade by using top-down and bottom-up approaches. The Secondary analysis has been accustomed to decide the key players in Gene Therapy market and market shares, rate, and Gene Therapy market future trends are discovered through primary and secondary analysis. The target of this Gene Therapy report is to produce a full study of Gene Therapy market by analyzing all completely different regions.

Any Query? Feel Free To Enquire Here: https://marketresearch.biz/report/gene-therapy-market/#inquiry

The market research report precisely provides to the client:

To achieve a penetrating study of the Gene Therapy Market associate degreed have an exhaustive perception of the market and its economic condition analysis.

Appraise the manufacturing procedure, tidy affairs, and solutions.

Market policies that area unit being acquired by top-most specific organizations

Get associate degree exhaustive delineation of the Gene Therapy Market business.

Comprehend the combative circumstances, important competitors, and Gene Therapy Market leading brands

The main objectives of the market research report are:

To appear at international Gene Therapy Market position, succeeding predict, growth scope, prime market, and prime players.

To gift the Gene Therapy Market advancement among the u. s., Europe, and China.

To strategically profile the Gene Therapy Market key players and fully analyze their growth policies and techniques.

to stipulate, justify, and forecast the Gene Therapy Market by product sort, application, and key regions.

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The report is readily available and can be dispatched immediately after payment confirmation.

Global Gene Therapy Market Report mainly covers ten significant points:

1. An outlook of the Gene Therapy industry.

2. Business Competitive Landscape.

3. Global Gene Therapy Market share from 2020 to 2029.

4. Supply Chain Analysis.

5. Top Players Company Profiles.

6. Analysis of the product types of Gene Therapy.

7. Analysis of the Applications/End-Users of Gene Therapy.

8. Consumption and Export, Import Value by Major Countries.

9. Global Gene Therapy Market Forecast to 2029.

10. Critical success factors and Conclusions.

Table Of Content:-

Chapter 1:Gene Therapy Market Overview.

Chapter 2:Gene Therapy Market Segment Upstream and Downstream and Cost Analysis

Chapter 3:Gene Therapy industry by Type( Market Size & Forecast, Major Company of Product Type)

Chapter 4:Gene Therapy industry by Top Key Players(Sales Revenue, Gross Margin, Price, Main Products, etc)

Chapter 5 and 6:Gene Therapy Industry Competition and Market Demand(Demand Situation, Demand Forecast, Regional Demand Comparison)

Chapter 7:Global Gene Therapy Market report additionally depicts Region Operation (Demand & Forecast by Countries, Regional Output etc).

Chapter 8:Global Gene Therapy Market Price Trends, Manufacturers Gross Margin Analysis, Factors of Price Change.

Chapter 9:This report additionally depicts deals channel, merchants, brokers, wholesalers and market Research Findings and Conclusion, addendum and information source.

Click to View Figures, TOC Mentioned in the Gene Therapy Market Report at : https://marketresearch.biz/report/gene-therapy-market/#toc

At the ending, the Gene Therapy Market report decisions investment embody investigation and development tendency investigation. the first opportunities of this quickest growing international Gene Therapy Market business sections that area unit coated throughout this report. The Gene Therapy Market product specification and services and product value structure with production divided into the most effective regions, technology, and applications.

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Global Gene Therapy Market Investment Feasibility, Evolutionary Production and Regional Analysis | N - PharmiWeb.com

The research report onGlobal Hemophilia Gene Therapy Market2020 deeply studied remarkable features of the industry. The study provides market size, Hemophilia Gene Therapy ongoing trends, drivers, risks, opportunities, as well as major Hemophilia Gene Therapy market segments. It is based on historical information and presents Hemophilia Gene Therapy market requirements. Also, includes different Hemophilia Gene Therapy business approaches preferred by the decision-makers. That enhanced Hemophilia Gene Therapy growth and makes a phenomenal stand in the industry. The Hemophilia Gene Therapy market will raise with a prominent CAGR between 2020 to 2026.

The Hemophilia Gene Therapy Market has witnessed continuous growth in the past few years and is predicted to rise even further during the estimated period. In adding to the inclusive assessment of the market, the report presents upcoming trends, up-to-date Growth Factors, attentive opinions, facts, historical data, and statistically supported and industry validates market data.

Get Sample [emailprotected]:

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Hemophilia Gene Therapy Market Major Industry Players 2020:

Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline Therapeutics

Firstly, it figures out the main Hemophilia Gene Therapy industry structure, guidelines, deals, agreements, Hemophilia Gene Therapy regulations, and policies. Then covers the prediction of Hemophilia Gene Therapy market share, dynamics, and dominant players. Next, it lineup new Hemophilia Gene Therapy assumption to updates business values. in addition, it examines the Hemophilia Gene Therapy market position, current, and future projects, growth rate, and exploitation. It also scrutinizes world Hemophilia Gene Therapy market chain analysis, cost of raw material. Further, it reveals Hemophilia Gene Therapy downstream/upstream analysis and import-export landscape.

Hemophilia Gene Therapy Market Type Analysis:

Hemophilia AHemophilia B

Hemophilia Gene Therapy Market Applications Analysis:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

Get Huge [emailprotected]:

https://www.globalmarketers.biz/discount_inquiry/discount/130750

The analysis covers basic information about the Hemophilia Gene Therapy product like industry scope, segmentation, an overview of the market. Likewise, it provides supply-demand data, Hemophilia Gene Therapy investment feasibleness, and elements that limiting the growth of a Hemophilia Gene Therapy industry. Particularly, it serves Hemophilia Gene Therapy product demand, annual revenue, and growth prospects of the industry. The foreseen Hemophilia Gene Therapy market regions along with the present ones assist leading vendors, decision-makers, and viewers/readers to plan effectively Hemophilia Gene Therapy business strategies respectively.

Inquire Before [emailprotected]:

https://www.globalmarketers.biz/report/medicine/global-hemophilia-gene-therapy-market-2019-by-company,-regions,-type-and-application,-forecast-to-2024/130750#inquiry_before_buying

Global Hemophilia Gene Therapy Industry Research Report Benefits:

* Product executives, industry administrator, Hemophilia Gene Therapy chief regulative officers of the industries.

* Researchers, Hemophilia Gene Therapy examiners, research executives, and laboratory expertise.

* Universities, professors, students, interns, and distinct other academic organizations involved in the Hemophilia Gene Therapy market.

* Writer, reporters, journalists, editors, and webmasters want to know regarding Contract Lifecycle Management System.

* Private/governmental organizations, project managers involved in the Hemophilia Gene Therapy industry.

* Present or future Hemophilia Gene Therapy market players.

TABLE OF CONTENT:

Chapter 1: Hemophilia Gene Therapy Market Overview

Chapter 2: Global Economic Impact on Industry

Chapter 3: Hemophilia Gene Therapy Market Competition by Manufacturers

Chapter 4: Global Production, Revenue (Value) by Hemophilia Gene Therapy Market Region

Chapter 5: Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6: Global Production, Revenue (Value), Price Trend by Type

Chapter 7: Global Hemophilia Gene Therapy Market Analysis by Application

Chapter 8: Manufacturing Cost Analysis

Chapter 9: Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10: Hemophilia Gene Therapy Market Analysis, Distributors/Traders

Chapter 11: Hemophilia Gene Therapy Market Effect Factors Analysis

Chapter 12: Global Hemophilia Gene Therapy Market Forecast to 2027

Click here to see full [emailprotected]:

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Global Hemophilia Gene Therapy Market New Trends, Future Growth, Outlook, Industry Overview, Application and Forecast 2020-2027 - PRnews Leader

Hemophilia Gene Therapy Market Forecast 2020-2026

The Global Hemophilia Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Hemophilia Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

Get a PDF Copy of the Sample Report for free @ https://industrygrowthinsights.com/request-sample/?reportId=168211

The Report Covers the Following Companies:Spark TherapeuticsUltragenyxShire PLCSangamo TherapeuticsBioverativBioMarinuniQureFreeline TherapeuticsHemophilia Gene Therap

By Types:Hemophilia AHemophilia BHemophilia Gene Therap

By Applications:Hemophilia A Gene TherapyHemophilia B Gene Therapy

Furthermore, the report includes growth rate of the global market, consumption tables, facts, figures, and statistics of key segments.

By Regions:

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

Important Facts about Hemophilia Gene Therapy Market Report:

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The rest is here:
Global Hemophilia Gene Therapy Market 2026 Size, Key Companies, Trends, Growth And Regional Forecasts Research - PRnews Leader

Oct. 14, 2020 11:00 UTC

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has received rare pediatric disease designation and orphan drug designation from the U.S. Food and Drug Administration (FDA) for TSHA-102, an AAV9-based gene therapy in development for the treatment of Rett syndrome. Taysha anticipates that it will submit an Investigational New Drug (IND) application for TSHA-102 to the FDA in 2021.

Rett syndrome is one of the most common genetic causes of severe intellectual disability worldwide, with a prevalence of over 25,000 cases in the U.S. and European Union (EU). It is an X-linked disease that primarily occurs in females, but it can be seen very rarely in males. It is usually recognized in children between six to 18 months of age as they begin to miss developmental milestones or lose abilities they had developed. Individuals with Rett syndrome also show symptoms that include loss of speech, loss of purposeful use of hands, loss of mobility, seizures, cardiac impairments, breathing issues and sleep disturbances.

Patients with Rett syndrome are currently managed with symptomatic treatments as there are no therapies approved to treat the underlying cause of disease, said Berge Minassian, M.D., Chief Medical Advisor of Taysha and Chief of Pediatric Neurology at the University of Texas Southwestern Medical Center (UT Southwestern). Dr. Minassian is credited with describing the CNS isoform of the MECP2 gene which is responsible for neuronal and synaptic function throughout the brain. Gene therapy offers a potentially curative option for patients suffering with Rett syndrome.

Rett syndrome is caused by mutations in the MECP2 gene. TSHA-102 is designed to deliver a healthy version of the MECP2 gene as well as the miRNA-Responsive Auto-Regulatory Element, miRARE, platform technology to control the level of MECP2 expression. TSHA-102 represents an important step forward in the field of gene therapy, where we are leveraging a novel regulatory platform called miRARE to prevent the overexpression of MECP2, said Steven Gray, Ph.D., Chief Scientific Advisor of Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. In collaboration with Sarah Sinnett, Ph.D. to develop miRARE, our goal was to design a regulated construct that allowed us to control MECP2 expression to potentially avoid adverse events that are typically seen with unregulated gene therapies.

The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as "rare" affect under 200,000 people in the U.S. The Rare Pediatric Disease Priority Review Voucher Program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, companies are eligible to receive a priority review voucher following approval of a product with rare pediatric disease designation if the marketing application submitted for the product satisfies certain conditions. If issued, a sponsor may redeem a priority review voucher for priority review of a subsequent marketing application for a different product candidate, or the priority review voucher could be sold or transferred to another sponsor.

Orphan drug designation is granted by the FDA Office of Orphan Products Development to investigational treatments that are intended for the treatment of rare diseases affecting fewer than 200,000 people in the U.S.

Obtaining these designations is a validation of decades-long work to identify and optimize a potential gene therapy treatment for this devastating disease, said RA Session II, President, CEO and Founder of Taysha. We are also excited to advance our miRARE platform whereby regulated expression of a transgene is possible on a cellular basis. The miRARE platform has broad applicability across a wide range of monogenic CNS disorders where there is a need to control transgene expression.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

About miRARE

For disorders that require replacement of dose-sensitive genes, we have combined high-throughput microRNA, or miRNA, profiling and genome mining to create miRNA-Responsive Auto-Regulatory Element, or miRARE, our novel miRNA target panel. This approach is designed to enable our product candidates to maintain safe transgene expression levels in the brain. This built-in regulation system is fully endogenous, and does not require any additional exogenous drug application. Instead, the miRARE system utilizes endogenous transgene-responsive miRNA to downregulate transgene expression in the event that overexpression occurs. miRARE may be applicable to a range of diseases where overexpression of a therapeutic transgene is a concern.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning or implying the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, the benefits of, and our ability to develop product candidates using, miRARE, our research, development and regulatory plans for our product candidates, the potential benefits of rare pediatric disease designation and orphan drug designation to our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed. Forward-looking statements are based on management's current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission filings, including in our prospectus dated September 23, 2020, as filed with the Securities and Exchange Commission (SEC) on September 24, 2020, pursuant to Rule 424(b) under the Securities Act of 1933, as amended, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201014005319/en/

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Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome - BioSpace

The Cell and Gene Therapy market research report added by Market Study Report, LLC, is an in-depth analysis of the latest trends persuading the business outlook. The report also offers a concise summary of statistics, market valuation, and profit forecast, along with elucidating paradigms of the evolving competitive environment and business strategies enforced by the behemoths of this industry.

Executive Summary:

The recent report on Cell and Gene Therapy market offers an in-depth analysis of this industry vertical and talks about the various growth drivers, opportunities, challenges, and other prospects influencing the remuneration. According to the report, the Cell and Gene Therapy market is predicted to expand with a CAGR of XX% during the study duration.

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Crucial insights pertaining to geographical landscape and competitive scenario as well as factors impacting the several market segmentations are encompassed in the document. The report also analyzes the impact of COVID-19 pandemic on the market outlook in the approaching years.

Market Rundown:

Regional outlook:

Product landscape summary:

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Application scope overview:

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Competitive landscape Review:

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Cell and Gene Therapy Market Overview with Detailed Analysis, Competitive landscape, Forecast to 2025 - Eurowire

LEXINGTON, Mass. and AMSTERDAM, Oct. 13, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that two additional patient procedures have been completed in the Phase I/II clinical trial of AMT-130 for the treatment of Huntingtons disease. The ongoing patient enrollment follows a meeting last month of the trials independent Data Safety Monitoring Board (DSMB) to review 90-day follow-up data from the first two patients. The DSMB observed no significant safety concerns to prevent further dosing. The Phase I/II study is a double-blinded, randomized and controlled clinical trial being conducted in the United States. A total of four patients have been enrolled in the study thus far, including two patients treated with AMT-130 and two patients who received imitation surgery.

We are very pleased with the progress being made to advance this first-in-human AAV gene therapy for Huntingtons disease, said Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. This is an important achievement that puts us on our original clinical development timeline, making up for the modest delay in the study earlier this year due to COVID-19. In accordance with the study protocol, patient enrollment is expected to continue after a DSMB meeting to review 90-day follow-up data on these two new patients and 6-month data on the first two patients. We expect that this DSMB review will take place early next year and that patient enrollment in the 10-patient first dose cohort will be completed by mid-2021.

The Phase I/II clinical trial of AMT-130 for the treatment of Huntingtons disease will explore the safety, tolerability, and efficacy signals in 26 patients with early manifest Huntingtons disease randomized to treatment with AMT-130 or an imitation (sham) surgery across two dose cohorts. The multi-center trial consists of a blinded 12-month core study period followed by unblinded long-term follow-up for 5 years after administration of AMT-130. Patients will receive a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen). Additional details are available on http://www.clinicaltrials.gov (NCT04120493).

AMT-130 is uniQures first clinical program focusing on the central nervous system (CNS) incorporating its proprietary miQURE platform.

About Huntingtons Disease

Huntingtons disease is a rare, inherited neurodegenerative disorder that leads to motor symptoms including chorea, and behavioral abnormalities and cognitive decline resulting in progressive physical and mental deterioration. The disease is an autosomal dominant condition with a disease-causing CAG repeat expansion in the first exon of the huntingtin gene that leads to the production and aggregation of abnormal protein in the brain. Despite the clear etiology of Huntingtons disease, there are no currently approved therapies to delay the onset or to slow the diseases progression.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

uniQure Forward-Looking Statements

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, whether patient enrollment will continue after a DSMB meeting to review follow-up data, whether the DSMB review will take place early next year, and whether patient enrollment in the first dose cohort will be completed by mid-2021.Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQures periodic securities filings, including its Annual Report on Form 10-K filed March 2, 2020 and Quarterly Report on Form 10-Q filed on July 30, 2020. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:

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uniQure Announces Enrollment of Next Two Patients in Phase I/II Clinical Trial of AMT-130 for the Treatment of Huntington's Disease - GlobeNewswire

DUBLIN, Oct. 13, 2020 /PRNewswire/ -- The "Viral Vector Purification / Virus Purification Products Market (kit, prepacked column, resin, cassette, filter plate, capsule, reagent and others), 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The Viral Vector Purification / Virus Purification Products Market 2020-2030' report features an extensive study of various products available for viral vector purification, in addition to the current market landscape and future potential of product developers.

Overview

Since the approval and launch of cell and gene therapy products, such as Zolgensma (2019), ZYNTEGLO (2019), Luxturna (2017), KYMRIAH (2017) and YESCARTA (2017), there has been a significant increase in demand for viral vectors. Experts believe that the number of such therapies is likely to double over the next couple of years. It is also worth mentioning that this particular field of medical research received close to USD 10 billion in funding in 2019.

Currently, over 1,000 cell and gene therapy-related clinical trials are being conducted, worldwide. Genetic manipulation is a basic requirement of cell and gene therapy development, and, as a result, gene delivery vectors are considered to be of utmost importance in this domain. So far, viral vectors (such as those based on adeno-associated viruses (AAV), adenoviruses, lentivirus, retroviruses and other viruses) have been shown to be the most versatile gene delivery tools available, having demonstrated high transfection efficiencies in both preclinical and clinical settings.

Further, taking into account the therapeutic efficacy and low side effects profiles of cell and gene therapies, the demand for such interventions is anticipated to grow at a rapid pace, resulting in a proportional increase in need for appropriate vector systems, as well. However, viral vector development and manufacturing is a complex and cost intensive process. One of the primary concerns associated with viral vector production is related to yield; in fact, a singular batch run is estimated to incur losses of up to 70% during the purification step alone.

A number of techniques are presently used for viral vector purification. Over the years, size-based viral purification strategies, such as density-gradient ultracentrifugation, ultrafiltration, precipitation and size-exclusion chromatography (SEC), have become part of the accepted industry standard. However, recently, stakeholders have begun relying more on affinity chromatography-based purification regimens, given its robustness and high selectivity. Presently, several companies claim to offer a diverse range of virus purification solutions, including, filter plates, prepacked chromatography columns and resins, and consolidated kits, for viral vector (virus) purification. As indicated earlier, downstream processing of viral vector products is challenging.

Scope of the Report

An overview of the current market landscape of companies providing products for purification of viruses / viral vectors, using different techniques, such as chromatography, centrifugation and filtration. It features information on the type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others) and details on other physical and operational parameters of the product (such as matrix, pore size, volume of bed, flow rate, operating pressure, working temperature, pH, filtration area and process time).

In addition, the chapter includes information on the purification product developers, including details on the year of establishment, company size and location of headquarters. Elaborate profiles of key players, including an overview of the company, product portfolio (viral vector purification products), recent developments and an informed future outlook.

An analysis evaluating the potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on several parameters, such as type of viral vector, developer strength, operational strength, therapeutic area, strength of clinical pipeline and strength of preclinical pipeline.

A clinical trial analysis of completed, ongoing and planned studies of various viral vector-based cell therapies, gene therapies and vaccines (approved / under development). It features detailed analyses of clinical studies of different viral-vector based therapies on the basis of their registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor/collaborator, geographical location, number of patients enrolled and key players.

An informed estimate of the annual clinical and commercial demand (in terms of number of patients) for viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. Further, the demand has been segregated on the basis of type of viral vector, type of therapy, therapeutic are and geographical location. A case study on tangential flow filtration (TFF), highlighting the role, advantages and disadvantages of the technique for purification of viral vectors; the chapter features details of products used for TFF, including product type, scale of operation, membrane material, flow rate and filtration area.

A case study featuring the viral vector manufacturers providing commercial scale production, highlighting details on their year of establishment, company size, type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), and location of headquarters and manufacturing facilities.

One of the key objectives of the report was to estimate the existing market size and identify potential growth opportunities for viral vector purification product developers, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies related to viral vector-based therapies, anticipated growth in target patient population, existing price variations across different purification techniques, and the success of cell and gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2020-2030.

Companies Mentioned

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Viral Vector Purification / Virus Purification Products Markets, 2030: Focus on Kit, Prepacked Column, Resin, Cassette, Filter Plate, Capsule, Reagent...

Trusted Business Insights answers what are the scenarios for growth and recovery and whether there will be any lasting structural impact from the unfolding crisis for the Cell Therapy market.

Trusted Business Insights presents an updated and Latest Study on Cell Therapy Market. The report contains market predictions related to market size, revenue, production, CAGR, Consumption, gross margin, price, and other substantial factors. While emphasizing the key driving and restraining forces for this market, the report also offers a complete study of the future trends and developments of the market.The report further elaborates on the micro and macroeconomic aspects including the socio-political landscape that is anticipated to shape the demand of the Cell Therapy market during the forecast period.It also examines the role of the leading market players involved in the industry including their corporate overview, financial summary, and SWOT analysis.

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Industry Insights, Market Size, CAGR, High-Level Analysis: Cell Therapy Market

The global cell therapy market size was valued at USD 5.8 billion in 2019 and is projected to witness a CAGR of 5.4% during the forecast period. The development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. In addition, automation in adult stem cell and cord blood processing and storage are the key technological advancements that have supported the growth of the market for cell therapy.

The investment in technological advancements for decentralizing manufacturing of this therapy is anticipated to significantly benefit the market. Miltenyi Biotec is one of the companies that has contributed to the decentralization in manufacturing through its CliniMACS Prodigy device. The device is an all-in-one automated manufacturing system that exhibits the capability of manufacturing various cell types.

An increase in financing and investments in the space to support the launch of new companies is expected to boost the organic revenue growth in the market for cell therapy. For instance, in July 2019, Bayer invested USD 215 million for the launch of Century Therapeutics, a U.S.-based biotechnology startup that aimed at developing therapies for solid tumors and blood cancer. Funding was further increased to USD 250 billion by a USD 35 million contribution from Versant Ventures and Fujifilm Cellular Dynamics.

The biomanufacturing companies are working in collaboration with customers and other stakeholders to enhance the clinical development and commercial manufacturing of these therapies. Biomanufacturers and OEMs such as GE healthcare are providing end-to-end flexible technology solutions to accelerate the rapid launch of therapies in the market for cell therapy.

The expanding stem cells arena has also triggered the entry of new players in the market for cell therapy. Celularity, Century Therapeutics, Rubius Therapeutics, ViaCyte, Fate Therapeutics, ReNeuron, Magenta Therapeutics, Frequency Therapeutics, Promethera Biosciences, and Cellular Dynamics are some startups that have begun their business in this arena lately.

Use-type Insights

The clinical-use segment is expected to grow lucratively during the forecast period owing to the expanding pipeline for therapies. The number of cancer cellular therapies in the pipeline rose from 753 in 2018 to 1,011 in 2019, as per Cancer Research Institute (CRI). The major application of stem cell treatment is hematopoietic stem cell transplantation for the treatment of the immune system and blood disorders for cancer patients.

In Europe, blood stem cells are used for the treatment of more than 26,000 patients each year. These factors have driven the revenue for malignancies and autoimmune disorders segment. Currently, most of the stem cells used are derived from bone marrow, blood, and umbilical cord resulting in the larger revenue share in this segment.

On the other hand, cell lines, such as Induced Pluripotent Stem Cells (iPSC) and human Embryonic Stem Cells (hESC) are recognized to possess high growth potential. As a result, a several research entities and companies are making significant investments in R&D pertaining to iPSC- and hESC-derived products.

Therapy Type Insights of Cell Therapy Market

An inclination of physicians towards therapeutic use of autologous and allogeneic cord blood coupled with rising awareness about the use of cord cells and tissues across various therapeutic areas is driving revenue generation. Currently, the allogeneic therapies segment accounted for the largest share in 2019 in the cell therapy market. The presence of a substantial number of approved products for clinical use has led to the large revenue share of this segment.

Furthermore, the practice of autologous tissue transplantation is restricted by the limited availability of healthy tissue in the patient. Moreover, this type of tissue transplantation is not recommended for young patients wherein tissues are in the growth and development phase. Allogeneic tissue transplantation has effectively addressed the above-mentioned challenges associated with the use of autologous transplantation.

However, autologous therapies are growing at the fastest growth rate owing to various advantages over allogeneic therapies, which are expected to boost adoption in this segment. Various advantages include easy availability, no need for HLA-matched donor identification, lower risk of life-threatening complications, a rare occurrence of graft failure, and low mortality rate.

Regional Insights of Cell Therapy Market

The presence of leading universities such as the Institute for Stem Cell Biology and Regenerative Medicine, Stanford University, and Yale Stem Cell Center that support research activities in U.S. is one of the key factor driving the market for cell therapy in North America. Moreover, strong regulatory and financing support from the federal bodies for expansion of this arena in U.S. as well as Canada is driving the market.In Asia Pacific, the market is anticipated to emerge as a lucrative source of revenue owing to the availability of therapies at lower prices coupled with growing awareness among the healthcare entities and patients pertaining the potential of these therapies in chronic disease management. Japan is leading the Asian market for cell therapy, which can be attributed to its fast growth as a hub for research on regenerative medicine.

Moreover, the Japan government has recognized regenerative medicine and cell therapy as a key contributor to the countrys economic growth. This has positively influenced the attention of global players towards the Asian market, thereby driving marketing operations in the region.

Market Share Insights of Cell Therapy Market

Some key companies operating in this market for cell therapy are Fibrocell Science, Inc.; JCR Pharmaceuticals Co. Ltd.; Kolon TissueGene, Inc.; PHARMICELL Co., Ltd.; Osiris Therapeutics, Inc.; MEDIPOST; Cells for Cells; NuVasive, Inc.; Stemedica Cell Technologies, Inc.; Vericel Corporation; and ANTEROGEN.CO.,LTD. These companies are collaborating with the blood centers and plasma collection centers in order to obtain cells for use in therapeutics development.

Several companies have marked their presence in the market by acquiring small and emerging therapy developers. For instance, in August 2019, Bayer acquired BlueRock Therapeutics to establish its position in the market for cell therapy. BlueRock Therapeutics is a U.S. company that relies on a proprietary induced pluripotent stem cell (iPSC) platform for cell therapy development.

Several companies are making an entry in the space through the Contract Development and Manufacturing Organization (CDMO) business model. For example, in April 2019, Hitachi Chemical Co. Ltd. acquired apceth Biopharma GmbH to expand its global footprint in the CDMO market for cell and gene therapy manufacturing.

In September 2020, Takeda Pharmaceutical Company Limited announced the expansion of its cell therapy manufacturing capabilities with the opening of a new 24,000 square-foot R&D cell therapy manufacturing facility at its R&D headquarters in Boston, Massachusetts. The facility provides end-to-end research and development capabilities and will accelerate Takedas efforts to develop next-generation cell therapies, initially focused on oncology with the potential to expand into other therapeutic areas.

The R&D cell therapy manufacturing facility will produce cell therapies for clinical evaluation from discovery through pivotal Phase 2b trials. The current Good Manufacturing Practices (cGMP) facility is designed to meet all U.S., E.U., and Japanese regulatory requirements for cell therapy manufacturing to support Takeda clinical trials around the world.

The proximity and structure of Takedas cell therapy teams allow them to quickly apply what they learn across a diverse portfolio of next-generation cell therapies including CAR NKs, armored CAR-Ts, and gamma delta T cells. Insights gained in manufacturing and clinical development can be quickly shared across global research, manufacturing, and quality teams, a critical ability in their effort to deliver potentially transformative treatments to patients as fast as possible.

Takeda and MD Anderson are developing a potential best-in-class allogeneic cell therapy product (TAK-007), a Phase 1/2 CD19-targeted chimeric antigen receptor-directed natural killer (CAR-NK) cell therapy with the potential for off-the-shelf use being studied in patients with relapsed or refractory non-Hodgkins lymphoma (NHL) and chronic lymphocytic leukemia (CLL). Two additional Phase 1 studies of Takeda cell therapy programs were also recently initiated: 19(T2)28z1xx CAR T cells (TAK-940), a next-generation CAR-T signaling domain developed in partnership with Memorial Sloan Kettering Cancer Center (MSK) to treat relapsed/refractory B-cell cancers, and a cytokine and chemokine armored CAR-T (TAK-102) developed in partnership with Noile-Immune Biotech to treat GPC3-expressing previously treated solid tumors.

Takedas Cell Therapy Translational Engine (CTTE) connects clinical translational science, product design, development, and manufacturing through each phase of research, development, and commercialization. It provides bioengineering, chemistry, manufacturing and control (CMC), data management, analytical and clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Segmentations, Sub Segmentations, CAGR, & High-Level Analysis overview of Cell Therapy Market Research ReportThis report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2019 to 2030. For the purpose of this study, this market research report has segmented the global cell therapy market on the basis of use-type, therapy-type, and region:

Use-Type Outlook (Revenue, USD Million, 2019 2030)

Clinical-use

By Therapeutic Area

By Cell Type

Non-stem Cell Therapies

Therapy Type Outlook (Revenue, USD Million, 2019 2030)

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Cell Therapy Market Size, Share, Market Research and Industry Forecast Report, 2020-2027 (Includes Business Impact of COVID-19) - Eurowire

In simple words, apoptosis assays refers to programmed cell deaths, which are able to regulate genetically cell ablation over the period of normal development. It is utilized for the purpose of elimination of unhealthy, unnecessary, and old cells sans any release of harmful materials into the adjoining areas. The morphological attributes of apoptotic cells comprise production of membrane-bound apoptotic bodies, cytoplasm contraction, and chromatin compaction. The growing importance the assay in biotechnology sector is expected to foster development of the global apoptosis assay market in the years to come.

The rising incidences of chronic and infectious diseases across the globe play an important growth factor for the global apoptosis assay market. In addition to that, a rise in the number of cell-based research projects together with increased allocation of funding for cancer research projects is likely to pave way for the development of the global apoptosis assay market over the tenure of analysis, from 2019 to 2029.

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Product, assay type, technique, end use, and region are the five key parameters based on which the global Apoptosis Assay market has been divided. The objective of such segmentation is to offer a clearer, 360-degree view of the market.

Global Apoptosis Assay Market: Notable Developments

The global apoptosis assay market has witnessed significant developments in the recent years. One of such developments pertaining to the market is mentioned below:

Some of the key players in the global apoptosis assay market comprise the below-mentioned:

Global Apoptosis Assay Market: Key Trends

The following drivers, restraints, and opportunities characterize the global apoptosis assay market over the assessment period, from 2019 to 2029.

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There have been on-going developments pertaining to the apoptosis-modulating drugs, which is likely to widen the scope of growth of the global apoptosis assay market over the forecast timeframe, from 2019 to 2029. Extensive use of molecular targeting and apoptosis assays for the purpose of treatment of various chronic diseases and cancer is anticipated to fuel growth of the market in the years to come.

In addition, increased prevalence of autoimmune diseases, such as multiple sclerosis, systemic lupus erythematosus are likely to work in favor of the market over the period of analysis, from 2019 to 2029. According to the findings of American Autoimmune Related Disease Association (AARDA), nearly 50 million Americans were suffering from one or more autoimmune disease in 2018. Presence of such huge base of patients in America only indicates towards vast pool of such patients worldwide, which is likely to augur well for the global apoptosis assay market in the near future.

Global Apoptosis Assay Market: Geographical Analysis

North America region is expected to account for most of the revenue contribution of the global apoptosis assay market and the region is expected to dominate the market throughout the period of analysis, from 2019 to 2029. The US is estimated to be one of the major contributors of the global apoptosis assay market.

The global apoptosis assay market is segmented as:

Product

Technique

Assay Type

End Use

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Apoptosis Assay Market - Rising Incidences of Chronic and Infectious Diseases Drive the Market Growth - BioSpace

Oct. 13, 2020 12:01 UTC

Organizations to work together to integrate Be The Match BioTherapies existing cell therapy infrastructure to manage the supply chain for potential commercialization of omidubicel

BOSTON & MINNEAPOLIS--(BUSINESS WIRE)-- Gamida Cell Ltd.. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, today announced an expansion of their existing strategic collaboration for omidubicel, Gamida Cells advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of an allogeneic hematopoietic stem cell (bone marrow) transplant. The broadened agreement represents an important step in both organizations patient access efforts and in Gamida Cells preparation for potential approval by the U.S. Food and Drug Administration (FDA).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201013005030/en/

The original partnership agreement between the organizations focused on the omidubicel development program and leveraged a wide range of Be The Match BioTherapies capabilities and services. This included providing cellular source material from the Be The Match Registry, which offers the most ethnically diverse listing of potential donors and umbilical cord blood units in the world, with 22 million potential donors and more than 300,000 umbilical cord blood units, as well as cell therapy supply chain and logistics management services. In building upon the existing collaboration, Gamida Cell will work through Be The Match BioTherapies for the ordering and supply of cord blood units, which serve as the starting material for omidubicel. The expanded agreement is designed to provide a smooth process throughout the omidubicel therapy supply chain.

Gamida Cells work to bring a new stem cell graft source to patients aligns with our core mission to help organizations deliver cellular therapies that can save more lives and improve the quality of life for patients, said Amy Ronneberg, chief executive officer of the National Marrow Donor Program/Be The Match and Be The Match BioTherapies. Were delighted to expand upon our collaboration to more fully leverage our infrastructure and technology to support the advancement of Gamida Cells efforts to seamlessly bring omidubicel to patients in clinical and commercial settings. Gamida Cells efforts to make stem cell transplant more accessible to patients could be particularly impactful for patients who do not have a matched donor of suitable age.

Be The Match BioTherapies is a respected leader in cell therapy and has an extensive history of assuring broad transplant access through the delivery of source material, globally, for patients in need of a transplant, stated Michele Korfin, chief operating and commercial officer of Gamida Cell. Deepening our collaboration represents an important step for Gamida Cell as the company increases its focus on potentially bringing omidubicel to patients in the commercial setting after reporting that omidubicel met its primary endpoint and all three secondary endpoints in our randomized, multi-center Phase 3 study. We look forward to our continued collaboration with Be The Match BioTherapies to ensure that we have an efficient and reliable cell therapy supply chain that can provide a positive experience for transplant teams and their patients.

In May, Gamida Cell reported that its Phase 3 study of omidubicel met its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a stem cell transplant. Additionally, in October, Gamida Cell reported that all three secondary endpoints for the study related to platelet engraftment, infections and hospitalizations demonstrated statistical significance. Gamida Cell expects to begin submitting the biologics license application for omidubicel to the FDA on a rolling basis in the fourth quarter of 2020.

Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including difficulty in finding a matched donor. Omidubicel is designed to potentially serve as a universal alternative to existing donor sources for bone marrow transplant.

About Omidubicel Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit http://www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.

About Gamida Cell Gamida Cell is an advanced cell therapy company committed to cures for patients with blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.

About Be The Match BioTherapies Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program (NMDP)/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the worlds largest and most diverse registry of more than 22 million potential blood stem cell donors and more than 300,000 umbilical cord blood units. Through established relationships with apheresis, marrow collection and transplant centers worldwide, the organization develops, onboards, trains and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven integrated model of both cell therapy supply chain and logistics managers, complimented by regulatory compliance experts to successfully transport and deliver life-saving therapies across the globe. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter at @BTMBioTherapies.

Gamida Cell Forward Looking Statements This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the effect on any cell therapy supply chain or Gamida Cells anticipated timing regulatory filing submissions for omidubicel, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the ongoing global COVID-19 pandemic and manufacturing, clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Annual Report on Form 20-F, filed with the Securities and Exchange Commission (SEC) on February 26, 2020, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to Gamida Cell as of the date of this release.

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Gamida Cell and Be The Match BioTherapies Expand Strategic Collaboration - BioSpace

Find out which biopharma companies are raking in the cash this week, as companies from around the globe provide updates on their financing rounds and IPOs.

Evotec

The UAE dropped a load of cash to become a 5.6% stakeholder into Germanys Evotec with $236 million worth of shares purchased by their sovereign wealth fund, Mubadala Investment Company. Not one to be left out, existing investor Novo increased its stake to 11% by throwing in another $59 million, bringing Evotecs haul to about $295 million. Evotec is a drug discovery alliance and development partnership company out of Hamburg, Germany. With revenues nearly quadrupling over the last five years and a goal of 10% yearly growth in the future, its no surprise these investors want in big. Evotec plans to use the cash to reach its goals by growing, particularly in the U.S. and Europe as they ramp up global ops.

SQZ Biotech

Allied with Roche to develop new cancer cell therapies, SQZ has turned its focus to the NYSE. With a preliminary goal of $75 million for its IPO, theyve applied for listing as SQZ. While traditional cell therapies require a depletion of the immune cells to improve efficacy, SQZ claims to avoid those limitations with a proprietary technology that, as its name touts, squeezes cells through a microfluidic chip to open the cell membrane and allow the therapeutic load inside. They also tout a huge leg up in manufacturing 24-hour turnaround in comparison with a month or more for current therapies. Plus, without the pre-conditioning to weaken immune systems, SQZs technology aims to shorten hospital stays. The IPO earnings are intended to finance their groundbreaking cancer and infectious disease research.

Galecto Biotech

Just two weeks after completing a $64 million Series D round, Galecto Biotech rounds the corner and goes after the public market, hoping to raise $100 million in its IPO. If successful, the Copenhagen-based company will have totaled over $250 million in financing in just the last two years. Galectos focus is on a wide range of fibrotic disease, with its lead project, GB0139 for idiopathic pulmonary fibrosis, currently in a Phase IIb trial. The Series D and the new funding from this IPO will go toward getting the program through to approval and commercialization.

Codiak BioSciences

After filing to go public for a second time after withdrawing in 2019, Codiak finally hit the market with an $83 million IPO, falling short of its $100 million originally sought when filed in September. The bulk of the funds will be used to advance its lead program ExoSTING through a phase study in advanced or metastatic, recurrent solid tumors, support discover and preclinical R&Dand expand its engEx technology that supports its programs. Another $10 million will go into its second program, exoIL-12, through a Phase I trial in patients with cutaneous T-cell lymphoma.

EdiGene

A Series B of $67 million takes EdiGenes track record up to $100 million raised in the last two years. The Beijing-based biotech is currently leading the gene-editing wave in China with four platforms steadily advancing. The company's top candidate is a treatment for hereditary blood disease, with the next in line being a CAR-T treatment for cancer. CEO Dong Wei hopes their T cell therapeutics can help make a higher quality, lower cost option for patients and their families.

Cedilla Therapeutics

Small molecule-focused Cedilla wraps up a $57.6 million Series B round to drug the undruggable. The funding will go into preclinical work on its first two oncology candidates, which are being kept hush hush for now. They also have about five or six oncology programs running that are years away from the clinic. In addition to the Series B, Cedilla is bringing Casdin CIO and founder Eli Casdin and Boxer senior VP Dominik Naczynski onto its board of directors.

RayzeBio

Debuting with $45 million in Series A money, biotech newbie RayzeBio is ready to defeat cancer with radiopharmaceuticals. With a vision to be the first radiopharma platform in the market, RayzeBio has seven active programs and would like to see one development candidate by the second half of 2021. Radiopharmaceuticals have intrigued the biotech sphere lately, but securing a reliable supply of therapeutic radioisotopes has been a hang up. But recently the industry has devised alternate ways to generate Actinium-225, which is the radioisotope RayzeBio is working with. This new development spurred the drive to launch RayzeBio with the intent to penetrate specific tumor targets. The fledgling biotech is now rolling up its sleeves to get to work with the goal of being first.

Priothera Limited

To get more clinical data on its highly-promising therapy for high risk AML patients, Priothera closed on a $35 million USD Series A. The company's drug mocravimod should enhance the curative potential of allogeneic hematopoietic stem cell transplantation for treating AML. Allogeneic stem cell transplant is currently the only potentially curative approach for AML patients, but has a high mortality rate. This therapy appears promising for improving survival outcomes. Priothera acquired mocravimod from KYORIN Pharmaceutical.

Ori Biotech

Ori Biotech wants to speed up the innovation of cell and gene therapies via its manufacturing platform, and this weeks $30 million Series A is certainly a step in reaching that goal. Typically, a drug discovery pipeline can take an average of a decade to get from lab to patient. Oris platform closes, automates and standardizes manufacturing for cell and gene therapy developers so the company can move its treatments from pre-clinical to scale commercially. This novel automation will reduce cost of goods and the footprint. In addition to taking its platform to the market, Ori is also expecting to double its 8-head employee count in four months, and double that again by next year.

Kanaph Therapeutics

Kanaph beefs up its initial $8 million start in 2019 with a $21 million Series B in South Korea. This round of funding will go toward expediting the clinical development of Kanaphs pipelines, chiefly its TMEkine molecules platform for immuno-oncology and bi-specific Fc fusions for the treatment of retinal disease. Preclinical studies are anticipated to be completed at the end of this year or beginning of next year, and are ready for the next steps.

Rappta Therapeutics

Novo Seeds plants its stake in emerging biotech Rappta Therapeutics in a $10.5 million Series A round. Rapptas primary focus is developing first-in-class anti-cancer drugs that work by activating protein phosphatase 2A (PP2A). The PP2A enzyme is a key tumor suppressor which has historically been tricky to target with drugs. Rappta has derived a unique understanding of the protein along with propriety tools to allow therapeutic reactivation of PP2A, which offers the potential of multiple therapies with this as the platform for a new class of anti-cancer drugs. Jeroen Bakker, Principal at Novo Seeds, will join Rapptas board. Novartis Venture Fund, Advent Life Sciences and one family office also participated in the round.

Lixte Biotechnology

Previously listed on the OTCQB, Lixte is ready to take it to Nasdaq with a $9 million offering of 1.5 million shares at a price range of $5.75 to $6.75. The NY-based biotech has developed two active series LB-100 and LB-200. The current focus is on the LB-100, which targets several types of cancer and has potential for vascular and metabolic diseases. A Phase I trial has already been completed and demonstrated antitumor activity in humans. LB-100 is now in Phase Ib/II.

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Biopharma Money on the Move: October 7-14 - BioSpace

If I had to place money on a neurotech that will win the Nobel Prize, its optogenetics.

The technology uses light of different frequencies to control the brain. Its a brilliant mind-meld of basic neurobiology and engineering that hijacks the mechanism behind how neurons naturally activateor are silencedin the brain.

Thanks to optogenetics, in just ten years weve been able to artificially incept memories in mice, decipher brain signals that lead to pain, untangle the neural code for addiction, reverse depression, restore rudimentary sight in blinded mice, and overwrite terrible memories with happy ones. Optogenetics is akin to a universal programming language for the brain.

But its got two serious downfalls: it requires gene therapy, and it needs brain surgery to implant optical fibers into the brain.

This week, the original mind behind optogenetics is back with an update that cuts the cord. Dr. Karl Deisseroths team at Stanford University, in collaboration with the University of Minnesota, unveiled an upgraded version of optogenetics that controls behavior without the need for surgery. Rather, the system shines light through the skulls of mice, and it penetrates deep into the brain. With light pulses, the team was able to change how likely a mouse was to have seizures, or reprogram its brain so it preferred social company.

To be clear: were far off from scientists controlling your brain with flashlights. The key to optogenetics is genetic engineeringwithout it, neurons (including yours) dont naturally respond to light.

However, looking ahead, the study is a sure-footed step towards transforming a powerful research technology into a clinical therapy that could potentially help people with neurological problems, such as depression or epilepsy. We are still far from that visionbut the study suggests its science fiction potentially within reach.

To understand optogenetics, we need to dig a little deeper into how brains work.

Essentially, neurons operate on electricity with an additional dash of chemistry. A brain cell is like a living storage container with doorscalled ion channelsthat separate its internal environment from the outside. When a neuron receives input and that input is sufficiently strong, the cells open their doors. This process generates an electrical current, which then gallops down a neurons output brancha biological highway of sorts. At the terminal, the electrical data transforms into dozens of chemical ships, which float across a gap between neurons to deliver the message to its neighbors. This is how neurons in a network communicate, and how that network in turn produces memories, emotions, and behaviors.

Optogenetics hijacks this process.

Using viruses, scientists can add a gene for opsins, a special family of proteins from algae, into living neurons. Opsins are specialized doors that open under certain frequencies of light pulses, something mammalian brain cells cant do. Adding opsins into mouse neurons (or ours) essentially gives them the superpower to respond to light. In classic optogenetics, scientists implant optical fibers near opsin-dotted neurons to deliver the light stimulation. Computer-programmed light pulses can then target these newly light-sensitive neurons in a particular region of the brain and control their activity like puppets on a string.

It gets cooler. Using genetic engineering, scientists can also fine-tune which populations of neurons get that extra powerfor example, only those that encode a recent memory, or those involved in depression or epilepsy. This makes it possible to play with those neural circuits using light, while the rest of the brain hums along.

This selectivity is partially why optogenetics is so powerful. But its not all ponies and rainbows. As you can imagine, mice dont particularly enjoy being tethered by optical fibers sprouting from their brains. Humans dont either, hence the hiccup in adopting the tool for clinical use. Since its introduction, a main goal for next-generation optogenetics has been to cut the cord.

In the new study, the Deisseroth team started with a main goal: lets ditch the need for surgical implants altogether. Immediately, this presents a tough problem. It means that bioengineered neurons, inside a brain, need to have a sensitive and powerful enough opsin door that responds to lighteven when light pulses are diffused by the skull and brain tissue. Its like a game of telephone where one person yells a message from ten blocks away, through multiple walls and city noise, yet you still have to be able to decipher it and pass it on.

Luckily, the team already had a candidate, one so good its a ChRmine (bad joke cringe). Developed last year, ChRmine stands out in its shockingly fast reaction times to light and its ability to generate a large electrical current in neuronsabout a 100-fold improvement over any of its predecessors. Because its so sensitive, it means that even a spark of light, at its preferred wavelength, can cause it to open its doors and in turn control neural activity. Whats more, ChRmine rapidly shuts down after it opens, meaning that it doesnt overstimulate neurons but rather follows their natural activation trajectory.

As a first test, the team used viruses to add ChRmine to an area deep inside the brainthe ventral tegmental area (VTA), which is critical to how we process reward and addiction, and is also implicated in depression. As of now, the only way to reach the area in a clinical setting is with an implanted electrode. With ChRmine, however, the team found that a light source, placed right outside the mices scalp, was able to reliably spark neural activity in the region.

Randomly activating neurons with light, while impressive, may not be all that useful. The next test is whether its possible to control a mouses behavior using light from outside the brain. Here, the team added ChRmine to dopamine neurons in a mouse, which in this case provides a feeling of pleasure. Compared to their peers, the light-enhanced mice were far more eager to press a lever to deliver light to their scalpsmeaning that the light is stimulating the neurons enough for the mice to feel pleasure and work for it.

As a more complicated test, the team then used light to control a population of brain cells, called serotonergic cells, in the base of the brain, called the brainstem. These cells are known to influence social behaviorthat is, how much an individual enjoys social interaction. It gets slightly disturbing: mice with ChRmine-enhanced cells, specifically in the brainstem, preferred spending time in their test chambers social zone versus their siblings who didnt have ChRmine. In other words, without any open-brain surgery and just a few light beams, the team was able to change a socially ambivalent mouse into a friendship-craving social butterfly.

If youre thinking creepy, youre not alone. The study suggests that with an injection of a virus carrying the ChRmine geneeither through the eye socket or through veinsits potentially possible to control something as integral to a personality as sociability with nothing but light.

To stress my point: this is only possible in mice for now. Our brains are far larger, which means light scattering through the skull and penetrating sufficiently deep becomes far more complicated. And again, our brain cells dont normally respond to light. Youd have to volunteer for what amounts to gene therapywhich comes with its own slew of problemsbefore this could potentially work. So keep those tin-foil hats off; scientists cant yet change an introvert (like me) into an extrovert with lasers.

But for unraveling the inner workings of the brain, its an amazing leap into the future. So far, efforts at cutting the optical cord for optogenetics have come with the knee-capped ability to go deep into the brain, limiting control to only surface brain regions such as the cortex. Other methods overheat sensitive brain tissue and culminate in damage. Yet others act as 1990s DOS systems, with significant delay between a command (activate!) and the neurons response.

This brain-control OS, though not yet perfect, resolves those problems. Unlike Neuralink and other neural implants, the study suggests its possible to control the brain without surgery or implants. All you need is light.

Image Credit: othebo from Pixabay

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Scientists Found a New Way to Control the Brain With LightNo Surgery Required - Singularity Hub

NEW YORK, Oct. 12, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced Stacy Lindborg, Ph.D., Executive Vice President and Head of Global Clinical Research, will deliver a presentation at the 2020 Cell & Gene Meeting on the Mesa, being held virtually October 12-16, 2020.

Dr. Lindborg's presentation will be in the form of an on-demand webinar that will be available beginning today. Those who wish to listen to the presentation are required to register here. At the conclusion of the 2020 Cell & Gene Meeting on the Mesa, a copy of the presentation will also be available in the "Investors and Media" section of the BrainStorm website under Events and Presentations.

About the 2020 Cell & Gene Meeting on the Mesa

The conference will feature 80+ on-demand company presentations by leading public and private companies, highlighting their technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, and tissue engineering. Registrants will have access to 15+ expert-led panels and workshops including a mix of both live and on-demand sessions. The conference will be delivered in a virtual format over the course of five days October 12-16. There is also a premier partnering system, partneringONE, allowing registrants to plan 11 meetings with other attendees. For a list of presenting companies, refer to https://www.meetingonthemesa.com/company-presentations/.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com.

Contacts Investor Relations:Corey Davis, Ph.D.LifeSci Advisors, LLCPhone: +1 646-465-1138cdavis@lifesciadvisors.com

Media:Paul TyahlaSmithSolvePhone: + 1.973.713.3768Paul.tyahla@smithsolve.com

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SOURCE Brainstorm Cell Therapeutics Inc

Company Codes: NASDAQ-SMALL:BCLI

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BrainStorm to Present at the 2020 Cell & Gene Meeting on the Mesa - BioSpace

The recent surge in interest in genetically-modified therapies has resulted in a steep rise in demand for different vectors for fundamental and pharmacological research, opening up opportunities for companies with expertise in targeted gene delivery

Roots Analysis has announced the addition of Viral Vectors, Non-Viral Vectors and Gene Therapy Manufacturing Market (3rd Edition), 2019-2030 (Focus on AAV, Adenoviral, Lentiviral, Retroviral, Plasmid DNA and Other Vectors) report to its list of offerings.

Currently, biopharmaceutical developers are plagued by high development costs, complex production protocols and the need for specialized equipment, in order to ensure the safety and quality of complex biological interventions, such as cell and gene therapies. Consequently, a number of innovator companies have demonstrated a preference for outsourcing vector manufacturing, a key aspect of advanced, genetically-modified product development, to contract service providers.

To order this 430+ page report, which features 140+ figures and 180+ tables, please visit this link

Key Market Insights

The market is fragmented, with over 180 industry players and non-industry playersOver 50% of industry players are large or mid-sized firms. In recent years, the growing demand for vectors has spurred the establishment of several start-ups, as well. Further, more than 80 non-industry players, including universities, research institutes and hospitals, are also currently involved in producing viral vectors or plasmid DNA for use in genetically modified therapies

The demand for vectors for research / clinical use is presently more than that for commercial applicationsApproximately 80% of industry stakeholders presently claim to manufacture vectors at the laboratory and / or clinical scale. However, some firms (around 40, as per our research) have developed / are developing commercial scale capacity for the production of viral vectors or plasmid DNA.

The US and EU have emerged as major vector manufacturing hubsHigh volume of active clinical studies, requiring vectors, being conducted in these regions makes the US and EU the major vector manufacturing hubs. Approximately 50% of the vector manufacturing facilities are located in North America. This is followed by the EU, where approximately 45% of the worlds vector manufacturing facilities are located.

The current installed vector manufacturing capacity in the world is estimated to be over 60,000 LThe major share (70%) of the global vector manufacturing capacity belongs to companies that are manufacturing vectors at both clinical and commercial scales. Across the major global regions, 50% of the total installed vector manufacturing capacity is in the US. This can be attributed to the large number of small-sized and mid-sized companies that are presently situated in this region.

Around 140 partnerships were inked during the last three yearsThe maximum number of deals (37) were reported in 2016, followed by 27 partnerships established in 2018. Around 30% of the deals were related to the manufacturing of vectors across different scales of operation; this was followed by technology licensing agreements (23%).

90% of the market share is captured by viral vectors intended for use in oncological disordersDriven by the rapidly evolving pipeline of genetically modified therapies, including T-cell therapies and vector-based vaccines, and the increasing adoption of advanced production technologies, the vector manufacturing market is projected to grow at an accelerated pace. Specifically, revenues generated from the sales of lentiviral vectors currently represent the largest share of the market, followed by retroviral vectors.

To request a sample copy / brochure of this report, please visit this link

Key Questions Answered

Close to USD 2 billion (by 2030) financial opportunity within the vector manufacturing market has been analyzed across the following segments:

The report features inputs from eminent industry stakeholders, according to whom there is an evident need for industry stakeholders to modify operational models and expand manufacturing capabilities in order to ensure uninterrupted growth within the market. The report includes detailed transcripts of discussions held with the following experts:

The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), vector manufacturing technology, manufacturing facilities, vector manufacturing experience and an informed future outlook of the company.

For additional details, please visithttps://www.rootsanalysis.com/reports/view_document/viral-vectors-non-viral-vectors-and-gene-therapy-manufacturing-market-3rd-edition-2019-2030-focus-on-aav-adenoviral-lentiviral-retroviral-plasmid-dna-and-other-vectors/274.html or email [emailprotected]

You may also be interested in the following titles:

Contact:Gaurav Chaudhary+1 (415) 800 3415+44 (122) 391 1091[emailprotected]

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Viral and Non-Viral Vector Manufacturing Market is anticipated to grow at an annualized rate of over 20%, claims Roots Analysis - The Think Curiouser