Archive for the ‘Gene Therapy Research’ Category

Janssen is seeking approval of amivantamab for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

This morning, the company submitted a Biologics License Application to the U.S. Food and Drug Administration for this indication. Amivantamab is an investigational fully-human EGFR and mesenchymal epithelial transition factor (MET) bispecific antibody that targets both adriver mutation as well as a resistance mechanism. Exon 20 insertion mutations account for at least 9% of EGFR mutations. However, there are no FDA-approved targeted therapies for patients with these mutations, which means chemotherapy remains the standard of care, Janssen said this morning.

EGFR mutations, can lead to uncontrolled cancer cell growth and division. Those mutations are some of the most common mutations in NSCLC.EGFR exon 20 insertion mutations are the third most prevalent primary EGFR mutation, but often go undetected, the company said.

The BLA submission for amivantamab is based on data from the monotherapy arm of the Phase I CHRYSALIS study, a multi-center, open-label, multi-cohort study evaluating the safety and efficacy of amivantamab as a monotherapy and in combination with lazertinib, a novel third-generation EGFR tyrosine kinase inhibitor (TKI), in adult patients with advanced NSCLC. The BLA for amivantamab marks the first regulatory submission for patients with exon 20 insertion mutations and also marks Janssens first filing for the treatment of patients with lung cancer, the company said.

Elsewhere today:

Taysha Gene Therapies Texas-based Taysha Gene Therapies, Inc. secured both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration for TSHA-103, an AAV-9-based gene therapy in development for SLC6A1-related epilepsy, the company announced this morning.

RA Session II, founder and chief executive officer of Taysha, said the company is pleased with the designations awarded by the FDA and encouraged by the results of the ongoing research.

SLC6A1 epilepsy is an autosomal dominant genetic disorder characterized by the loss of function of one copy of theSLC6A1gene, with clinical manifestations of seizures, epilepsy, language impairment and intellectual disability. Steven Gray, chief scientific advisor to the company and an associate professor in the Department of Pediatrics at UT Southwestern, said haploinsufficiency in theSLC6A1gene has been identified as a cause of genetic epilepsy. However, as of yet, there are no approved disease-modifying therapies for this indication, he said. Gray touted the FDA designations and said they highlight the importance of developing a treatment for patients.

BioAge Labs California-based BioAge Labs raised $90 million in an oversubscribed Series C financing found. Funds will be used to support the development of BioAges platform that maps key pathways that drive human aging, as well as its pipeline of treatments that targets those pathways, BioAge CEO Kristen Fortney said in a statement. BioAge anticipates taking its first platform-derived therapies, BGE-117 and BGE-175 into clinical trials in the first half of 2021.

The financing round was co-led by Andreessen Horowitz and serial entrepreneur, Elad Gil. New investors include Kaiser Foundation Hospitals, AARP Foundation and Phi-X Capital. Current investors in the company, including Caffeinated Capital, Redpoint Ventures, PEAR Ventures, AME Cloud Ventures and Felicis Ventures, also participated.

Kinnate Biopharma Bay Area-based Kinnate Biopharma announced the pricing of a $20 per share price for its initial public offering of 12,000,000 shares of common stock. Gross proceeds are expected to be $240 million. The stock will be available for trading starting today on the Nasdaq exchange under the ticker symbol KNTE. Kinnate Biopharma is focused on the discovery and development of small molecule kinase inhibitors for difficult-to-treat, genomically defined cancers. Proceeds from the IPO will be used to fund clinical trials, for R&D and other general corporate purposes, the company said.

Valneva Frances Valneva SE said it will accelerate pediatric development of its Lyme vaccine candidate, VLA15, in collaboration with Pfizer. The company intends to begin dosing in the first quarter of 2021. The Phase II VLA15-221 study will include approximately 600 healthy participants between the ages of 5 and 65. If greenlit by health authorities, the study will be the first clinical trial of VLA15 to enroll a pediatric population aged 5-17 years and will compare the three-dose vaccination schedule Month 0-2-6 with a reduced two-dose schedule of Month 0-6. This study will complement the two ongoing Phase II studies VLA15-201 and VLA15-202, both of which posted positive data earlier this year. All three Phase II trials are anticipated to support a Phase III pivotal efficacy trial including all main target populations for the Lyme vaccine candidate starting in 2022.

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Daily Rundown: Who's Gunning For and Gaining Approval and Other Industry News - BioSpace

SEATTLE--(BUSINESS WIRE)--Shape Therapeutics, Inc. (ShapeTX), a next-generation gene therapy company with an industry-leading RNA targeting technology platform, announces today the unveiling of the AAVidTM capsid discovery platform and results from its first AAV5 variant library in a non-human primate selection campaign.

The AAVidTM capsid discovery platform uses non-random mutational fitness to create massive capsid libraries of billions of unique AAV variants for direct-to-NHP in vivo biological selection. By combining cutting-edge DNA synthesis, advanced synthetic biology, next-generation sequence barcoding and machine learning algorithms, ShapeTX generates industry-leading library size and diversity to enable the development of best-in-class human therapeutics.

Wildtype first-generation AAVs are enabling the recent advances in gene therapy, but they have been plagued by toxicities in the clinic due in part to a lack of tissue specificity, resulting in the need for high doses. Our AAVidTM platform solves the issue by creating novel capsid variants with specific tissue-tropism, said Francois Vigneault Ph.D., President and CEO at ShapeTX. Weve stayed quiet for the past three years while developing a superior AAV platform technology and are excited to announce that we have best-in-class AAV variants in hand. Today, we are announcing our novel liver-tropic AAV5 variants stay tuned for more to come.

David J. Huss, Ph.D., Vice President and Head of Research added, The vast structural space for exploration at the AAV capsid/target cell interface necessitates enormous library size and diversity, which until now, has only been probed with capsid library sizes in the tens of thousands to millions. At ShapeTX, we set out to create a superior AAV capsid discovery platform with library sizes in the billions of unique variants, thereby maximizing the opportunity for novel virus/target cell interactions. Dr. Huss presented the details of the platform at the 2nd RNA Editing Summit on Dec. 2, 2020.

About Shape Therapeutics, Inc

Shape Therapeutics is a biotechnology company developing next-generation RNA-targeted therapies to treat the worlds most challenging diseases. The ShapeTX technology platform includes RNAskip, a proprietary suppressor tRNA technology that enables premature stop codon readthrough; RNAfixTM, a precision RNA editing technology using endogenous Adenosine Deaminase Acting on RNA (ADAR); and AAVidTM, a next-generation engineered adeno-associated virus (AAV) platform producing highly specific, tissue-tropic AAVs. The power of the ShapeTX platforms resides in redirecting the cellular machinery already present in our cells, thereby bypassing the risks of immunogenicity and DNA damage seen with other contemporary editing technologies. ShapeTX is committed to data-driven scientific advancement, passionate people and a mission of providing life-long cures to patients. Shape Life!

Originally posted here:
Shape Therapeutics Unveils AAVid Capsid Discovery Platform and Identification of Novel Tissue-Specific AAV Variants, Solving a Fundamental Delivery...

Fort Collins, Colorado Global Cancer Gene Therapy Market report on in-depth research, sales estimates, and growth forecast details by segments, regions and research scope, historical data, key players, and growth value.

The Cancer Gene Therapy Market 2020 analysis provides a basic summary of the trade along with definitions, classifications, uses, and trade chain structure. Global Cancer Gene Therapy market study is provided for the international markets, along with development trends, competitive landscape analysis, and key regions development status. Development policies and plans are mentioned in the same way as production processes and value structures are analyzed. This report also provides information on import/export consumption, supply and demand, costs, prices, sales, and gross margins.

Global Cancer Gene Therapy Market industry valued approximately USD 0.2 billion in 2016 is anticipated to grow with a healthy growth rate of more than 35.1% over the forecast period 2017-2025.

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The Major Players Profiled in this Report include

Impact of Covid-19 on this Market:

Cancer Gene Therapy Market report analyses the impact of Coronavirus (COVID-19) on the Cancer Gene Therapy industry. Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 180+ countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Cancer Gene Therapy market in 2020.

The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; emergency declared in many countries; massive slowing of the supply chain; stock market unpredictability; falling business assurance, growing panic among the population, and uncertainty about future.

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Market Segments and Sub-segments Covered in the Report are as per below:

By End-User:

By Therapy:

This Market Study covers the Cancer Gene Therapy Market Size across segments. It aims at estimating the market size and the growth potential of the market across segments by component, data type, deployment type, organization size, vertical, and region. This Cancer Gene Therapy study also includes an in-depth competitive analysis of the key market players, along with their company profiles, key observations related to product and business offerings, recent developments, and key market strategies.

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Cancer Gene Therapy Market Size, Opportunities, Dynamic, Outlook and Forecast To 2027 - Cheshire Media

Professor Laurence D Hurst explains why understanding the nucleotide mutations in viruses, including SARS-CoV-2, can have significant implications for vaccine design.

With 61 codons specifying 20 amino acids, some can be encoded by more than one codon and it is often presumed that it does not matter which one a gene uses. When I first studied genetics, some books I read taught that mutations between such alternative codons (eg, GGA->GGC, both giving glycine) were called synonymous mutations, while others referred to them as silent mutations. However, are synonymous mutations really silent meaning they are identical in terms of fitness and function? Although they may specify the same amino acid, does that mean they are all the same?

Figure 1: Intronless GFP transgene expression is higher for variants of GFP with higher GC content at synonymous sites5

Perhaps one of the biggest surprises over recent years has been the discovery that versions of the same gene, differing only at synonymous sites, can not only have different properties, but effects that are not modest.1-5 For example, two versions of green fluorescent protein (GFP) differing only at synonymous sites can have orders of magnitude differences in their expression level.4 We similarly recently discovered that for an intronless transgene to express in human cell lines it needs to be GC rich, which can be achieved by altering the synonymous sites,5 as seen in Figure 1. It is no accident, we suggest, that the well-expressed endogenous intronless genes in humans (such as histones) are all GC rich and that our functional retrogenes tend to be richer in GC content than their parental genes.

The realisation that synonymous sites matter has clear relevance to the design of transgenes or other artificial genes, be these for experiments, gene therapy, protein production (eg, in bacteria) or for vaccine design. In the case of vaccines, we might wish to modulate a viral protein to be effectively expressed in human cells to illicit a strong and robust immune response.6 Conversely to the design of attenuated vaccines, we seek to produce a tuned down version of the virus that can function but is weak.7

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered. One approach is mass randomisation try many alternatives and see what works.4,8,9 In principle this is fine, but this approach requires many randomisations, which is still technically difficult for long attenuated viruses. An alternative strategy that we have been exploring is to let nature tell us; we can apply tools and ideas from population genetics to better understand what natural selection favours and disfavours and in turn to estimate the strength of selection.

it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus

Estimation of the strength of selection is possible from knowledge of the site frequency spectrum, (ie, how common variants are) from which we can infer the distribution of fitness effects (DFE). If a site is under strong purifying selection, then mutations may occur in the population but these are rapidly eliminated, so variants are always rare. By contrast, if they are selectively neutral, we expect some variants to be quite common. We recently applied this methodology to show that synonymous mutations in human genes that disrupt exonic splice enhancer motifs are often under strong selection and affect many synonymous sites in our genes.10 This has implications for both diagnostics and for transgene design for gene therapy, as we often remove introns in heterologous genes, so freeing up these residues from their role in specifying exons ceases.11

The same DFE methodology cannot easily be applied to viruses, as the methods assume free recombination (ie, we assume one mutation does not impact the fate of others in the same genome). However, other population genetical tools can still be applied. Recently, we examined SARS-CoV-2 and identified the profile of mutations that we see at four-fold degenerate sites.12 From this profile we could estimate what the synonymous site composition would be, assuming that the only forces are mutational biases and neutral evolution (ie, no selection). We observed that in this genome there is a strikingly strong C->U mutation bias and a G->U one. In the raw data this is not so obvious as G and C are quite rare. However, the mutability of the sites per occurrence of the site reveals the underlying patterns.

Figure 2: The rate of mutational flux from one dinucleotide to another in the coding sequence of SARS-CoV-2. The direction of flux is indicated by the indentation of the connecting links: the inner layer represents flux out while the outermost layer represents flux into the node. The frequency of the flux exchange is represented by the width of any given link where it meets the outer axis. Dinucleotide nodes are coloured according to their GC-content. Hence, it is evident that there is high flux away from GC-rich dinucleotides whereas AU-rich dinucleotides are largely conserved.12

With knowledge of the mutational bias we then asked what the equilibrium frequency of the four nucleotides would be using four simultaneous equations. This is the nucleotide content at which for every mutation changing a particular base there is an equal and opposite one creating the same base somewhere else in the genome, ensuring overall unchanged nucleotide content. Given the strong C->U and G->U mutational biases, it is no surprise that the equilibrium content is very U rich (we estimate equilibrium U content should be about 65 percent). However, while the four-fold sites are indeed U rich, they are not that U rich, being closer to 50 percent. A clue as to why the mutation bias is so skewed to generating U comes from analysis of equilibrium UU content: UU residues are predicted to be very common, with CU residues being particularly mutable generating UU (Figure 2) this is expected due to human APOBEC proteins attacking and mutating/editing the virus.13

One probable explanation for this difference between predicted and observed nucleotide content is selection against U content. There may be many U residues appearing in the population, but many are pushed out of the population owing to purification selection, ie, because of the deleterious effects of the mutations. That such selection is happening in the SARS-CoV-2 genome is also clear from the sequence data. We estimate that for every 10 mutations that appear in the sequence databases, another six are lost because of selection prior to genome sequencing. Indeed, UU content is about a quarter of that predicted (Figure 3).

Figure 3: The predicted (under neutral mutational equilibrium) and observed dinucleotide content of SARS-CoV-2. Note the very high predicted levels of UU given the strong mutational flux to UU residues (see Figure 2) and the net underrepresentation in actual sequence.9

This leaves two problems: why is selection operating on SARS-CoV-2 and what can we do with this information? In some cases, we have a good idea as to why: many mutations to U at codon sites generate stop codons. However, we have observed that U destabilises the transcripts and is associated with lower-reported transcript levels;12 a full explanation of the causes of selection on nucleotide content therefore requires manipulation of the sequences.

The second question, what to do with this information, is perhaps more urgent. It has previously been noted that nucleotide content manipulation is a viable means to attenuate viruses.7 Currently there are three groups investigating this route to make a vaccine for SARS-CoV-2: Indian Immunologicals Ltd/Griffith University, Codagenix/Serum Institute of India and Acbadem Labmed Health Services/Mehmet Ali Aydinlar University. In prior attempts, attention has been paid to CpG levels and UpA levels (which we find to be correlated between SARS genes and between different viruses).12 CpGs attract the attention of zinc antiviral protein (ZAP) and UpA attracts an RNAase L. Not surprisingly, some viruses, including SARS-CoV-2, therefore have low levels of both dinucleotide pairs given the levels of the underlying nucleotides.

The challenge is knowing not just which synonymous sites can be altered but knowing how they should be altered

In the past, attenuation strategies have focused on modulating synonymous sites to increase CpG and UpA, making the virus more visible to antiviral proteins.14 We in turn suggest a general strategy to utilise this method and to increase U content as well.12 Given the evidence that selection on the virus is to reduce U content, while our antiviral proteins are mutating it to increase U content, it will be interesting to see if we can learn a lesson from nature as to how to weaken a virus. This is an unusual circumstance in which we predict that we should build in more of the already most common synonymous site nucleotides (U in this case) to degrade the virus. More generally, it is assumed that the most used codons are those that tend to increase the fitness of the organism. In the face of such a severe mutation bias, however, this simpler logic no longer holds.

Laurence D Hurst is Professor of Evolutionary Genetics and Director of the Milner Centre for Evolution at the University of Bath. He is currently also the President of the Genetics Society. He completed his D.Phil in Oxford, after which he won a research fellowship and then moved to Cambridge University as a Royal Society Research Fellow. While on the fellowship he assumed his current Chair at Bath University. In 2015 he was elected a Fellow of the Academy of Medical Sciences and a Fellow of the Royal Society. He is a recipient of the Genetics Society Medal and the Scientific Medal of the Zoological Society of London.

Related topicsDisease research, DNA, Gene Therapy, Genetic analysis, Genomics, Protein, Proteogenomics, Proteomics, Research & Development, RNAs, Vaccine

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Tweaking synonymous sites for gene therapy and vaccines - Drug Target Review

FREDERICK, Md., Dec. 02, 2020 (GLOBE NEWSWIRE) -- RoosterBio, Inc, a leading supplier of innovative cellular therapy bioprocess tools and scalable manufacturing solutions for regenerative medicine, today announced it entered into an exclusive agency agreement with Sartorius Korea Biotech, a subsidiary of the Sartorius Group, a leading international partner of the biopharmaceutical and life science research sectors. This strategic partnership combines Sartorius proven expertise in providing high-caliber bioprocess testing, technical and customer services to the local SouthKorean market, as well as its global reach, and RoosterBios advanced platform of RUO and cGMP-grade human mesenchymal stem/stromal cell (hMSC) working cell banks, optimized paired media, and hMSC bioprocess systems. This winning combination sets the stage for accelerating the development of new, effective medical treatments and expands RoosterBios footprint into Asia, especially in South Korea.

South Korea is a preeminent innovator in hMSCs, and the South Korean biotech ecosystem has been flourishing according to every measure of growth. One of the first MSC therapies was developed in Korea, clearing an entirely new path to treat previously incurable diseases, said RoosterBio CEO Margot Connor. We are delighted to be working with Sartorius Korea Biotech in our global effort to industrialize the supply chain for next-gen treatments, and we are committed to accelerating the commercialization of cell- and gene-based therapies. We believe the talented team at Sartorius is uniquely suited to provide our mutual customers with scalable hMSC systems to expedite their cell-based therapeutic programs.

Under the terms of the agreement, Sartorius Korea Biotech will engage with its customers as an exclusive agent for RoosterBio to facilitate sales operations and increase the market potential for RoosterBio products on an exclusive basis in South Korea.

RoosterBios commercialized product systems standardize hMSC manufacturing to remove years of time and millions of dollars from the timeline of traditional mesenchymal cell therapeutic product development and clinical translation efforts, said Duck Sang Kim, Managing Director of Sartorius Korea Biotech. We look forward to introducing this synergistic biotechnology relationship to South Korea.

About RoosterBio, Inc

RoosterBio, Inc. is a privately held cell manufacturing platform technology company focused on accelerating the development of a sustainable Regenerative Medicine industry, one customer at a time. RoosterBio's products are high-volume, affordable, and well-characterized adult human mesenchymal stem/stromal cells (hMSCs) paired with highly engineered media systems. RoosterBio has simplified and standardized how living cells are purchased, expanded, and used in development, leading to marked time and costs savings for customers. RoosterBio's innovative products are ushering in a new era of productivity and standardization into the field. Visit http://www.roosterbio.com.

About Sartorius Korea Biotech, Ltd.

Sartorius Korea Biotech based in Pangyo, South Korea, addresses the evolving needs of the biomanufacturing industry to produce biotech medications and vaccines safely and efficiently. The company is a subsidiary of the Sartorius Group, a leading international partner of life science research and the biopharmaceutical industry, and offers a diversified business portfolio reflecting the products and services of the two Group divisions, Bioprocess Solutions and Lab Products & Services. Sartorius Korea Biotech provides pharmaceutical and laboratory equipment, validation service, training, engineering and consulting, and KOLAS calibration, among other products and services.

Visit http://www.sartorius.com

Contact: Carrie Zhang, Director of Marketing, RoosterBioczhang@roosterbio.comAndre Hofmann, Head of Public Relations, Corporate Communications, Sartoriusandre.hofmann@sartorius.com

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RoosterBio Collaborates with Sartorius to Expand Cell and Gene Therapy Manufacturing Platform Technology into South Korea - GlobeNewswire

Blood vessels are supposed to act like trees, pumping in oxygen tissues need to grow and immune cells required to clear out pathogens. But in tumors, the forest can go a bit haywire. Vessels grow prodigiously and bulge and twist at abrupt points, making it difficult to even tell whats a vein and whats an artery. It starts to look less like a forest and more like a gnarled root floor. A disorganized labyrinth, one oncologist has called it.

For cancer, chaos is a virtue. That gnarled root floor insulates solid tumors from immune cells and, in recent years, has flustered drug developers best attempts at developing therapies meant to rev up the immune system and direct it toward the tumors.

Researchers at the University of Pennsylvania, however, think they may have stumbled onto a solution, a way of whipping the blood vessels back into proper shape. If it works, experts say, it could pave the way for CAR-T treatments that attack solid tumors and potentially improve the effectiveness for more traditional approaches, such as radiation and chemotherapy.

Its a really novel and potentially important approach, Patrick Wen, a neuro-oncologist at Dana-Farber who was not involved in the work, told Endpoints News. They really did good work. This is a very different way of improving immunotherapy.

Yi Fan, a radiation oncologist and neurosurgeon at Penns School of Medicine, has been working for the last few years to understand why the labyrinth appears in the first place. Researchers had previously circled in on the so-called growth factors that stimulate blood vessel formation. Attempts to block these factors, though, disappointed; Avastin, an antibody against the factor VEGF, became a blockbuster but has continually failed to improve survival on a range of malignancies.

Scientists would have to go more fundamental. In a pair of 2018 papers, Fan showed that part of the problem is a process called endothelial cell transformation. Cells lining the blood vessels around the tumor acquire stem cell-like properties that allow them to reproduce and expand rapidly, as stem cells do.

Theres a genetic reprogramming, Fan told Endpoints. Theyll become really aggressive.

But how did that reprogramming happen? If Fan could pin down the pathway, he figured he could then devise a way to block it. He started knocking out kinases the cellular engines that can drive epigenetic change, or reprogramming one by one in endothelial cells isolated from patients with an aggressive brain cancer called glioblastoma. Out of 518, 35 prevented transformation and one did so particularly well: PAK4.

Then they injected tumors into mice, some who had PAK4 and some who had the kinase genetically removed: Eighty percent of the mice who had PAK4 removed lived for 60 days, while all of the wild-type mice died within 40. Fans team also showed that T cells infiltrated the tumors more easily in the PAK4-less mice.

It was a fortuitous finding: Drug companies had developed several PAK inhibitors a decade ago, when kinase inhibitors were the flashiest thing in pharma. Many had been abandoned, but Karyopharm had recently brought a PAK4 blocker into Phase I.

To see whether drug developers could exploit this finding, Fan and his team removed T cells from mice and developed a CAR-T therapy to attack the tumors.

They gave mice three different regimens. The CAR-T therapy on its own failed to reduce tumor size, apparently unable to reach through the vessels. The Karyopharm drug also had little effect on its own. But combined, they managed to reduce tumor size by 80% after five days. They published the results in Nature Cancer this week.

It is a really eye-opening result, Fan said. I think we see something really dramatic.

That, of course, is just in mice, but Fan already has strong supporting evidence for PAK4s role in cancer. Last December, while Fan was still completing his experiment, Nature Cancer published a paper from Antoni Ribas UCLA lab suggesting that PAK4 inhibitors can help T cells infiltrate around various solid tumors. They showed that the same Karyopharm inhibitor could boost the effects of PD-1 inhibitors in mice, allowing activated T cells to better reach tumors.

That work has already translated into the clinic; weeks after it came out, Karyopharm added an arm to their Phase I study of the drug that will look at the PAK4 inhibitor in combination with the PD-1 blocker Opdivo.

Ribas said that Fans work is compelling and helps confirm the role of PAK4, but he said a CAR-T therapy would face a much longer path to the clinic. Its simply much easier to combine an approved drug with an experimental one than to devise a new CAR-T therapy, mix it with the unapproved inhibitor (and all the other things, such as bone marrow-clearing chemotherapy, CAR-T recipients receive) and then deduce what effect each is having.

It will a take a while, Ribas told Endpoints. But I hope this is right and its developed clinically.

There are also other unresolved obstacles for CAR-T in solid tumors, Wen said. Developers still struggle to find targets that wont also send the super-charged T cells after healthy tissue. And tangled blood vessels are just one of several mechanisms tumors have of defending themselves. They can, for example, turn tumor-eating immune cells into tumor-defending ones.

Still, Wen said, in the short term, the approach offered a path toward boosting the efficacy of radiation, chemotherapy and other small molecule drugs. Although Fan focused on glioblastoma, researchers agreed PAK4 likely plays the same vessel-warping role in many other solid tumors.

Theres a lot of things you could look at, he said.

In a January review, Jessica Fessler and Thomas Gajewski at the University of Chicago said Ribas paper pointed towards a path for improving PD-1 and overcoming resistance in some tumors. But they also raised questions about the Karyopharm drug, noting that it hits other proteins besides PAK4. That could mean other mechanisms are also at play and that the drug could affect other tissues in humans.

Ribas agreed that Karyopharms drug might not be the perfect molecule but said others could be on their way. He serves as a scientific advisor to Arcus, the Terry Rosen startup that is now working on developing its own PAK4 inhibitor.

If they can develop a very selective PAK4 inhibitor, he said, it may be a more direct way of testing the role of PAK4.

Tests with that drug, in turn, could help clear up a biological mystery that emerged out of Fans and Ribas papers. Although both investigators zeroed in on PAK4, each of them suggested very different mechanisms by which PAK4 kept immune cells out of the tumor. Ribas suggested it directly suppresses T cells, while Fan found it led to those transformations inside the blood vessels near the tumor.

Kinases are versatile proteins and both researchers said its possible that PAK4 is doing both. Its also possible, they said, that one is more important than the other, or simply that one of them is just wrong.

When you start with completely new biology, its hard to get it right the first time, Ribas said.

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Penn researchers find a way through the labyrinth keeping CAR-T from solid tumors - Endpoints News

A little more than 2 years after Mercks Roger Perlmutter signed off on a deal that would pay Dragonfly up to $695 million for each drug program it picked off for solid tumors, the pharma giant is stepping up with their first opt-in.

We dont know exactly how much this deal costs Merck in the upfront, or which immunotherapy theyre getting, but its a major step forward for Bill Haneys Waltham, MA-based biotech, which built its TriNKET technology platform with the help of Tyler Jacks, an MIT professor, HHMI investigator and director of the David H. Koch Institute for Integrative Cancer Research as well as Berkeleys David Raulet, whose background as an expert in NK cells and tumor immunology helped spotlight some of the big ideas Dragonfly is pursuing.

This latest pact marks the latest in a flurry of BD deals for the pharma giant, just one last step before Perlmutter hangs it up as head of R&D and passes the reins to Dean Li. John Carroll

A little over a year since announcing its Series A, gene therapy biotech ViGeneron has entered into a new deal.

The German company is partnering with WuXi Advanced Therapies, a contract testing, development and manufacturing organization under WuXi AppTec based out of Philadelphia, to ramp up production of ViGenerons lead candidate VG901 for ophthalmic disorders. VG901s current target is for retinitis pigmentosa, also known as rod cone dystrophy, a degenerative eye disease that causes severe vision impairment as early as childhood.

Manufacturing for the candidate should begin before the year is out, ViGeneron said in a statement. The company added that the program came out of its proprietary vgAAV vector platform, which allows for better transduction of retinal cells as well as a less invasive treatment administration.

There is currently no cure for the disease, though there are some methods that can help manage symptoms like the use of low vision aids and portable lighting. Patients often experience worsening peripheral vision and trouble seeing at night. Max Gelman

Lentiviral vector manufacturer iVexSol has raised $13 million in Series A financing, bringing the total haul to $15.2 million from Casdin Capital and BioLife Solutions and a third undisclosed lead investor.

Founded on the promise to change the way this critical raw material is made using next-generation manufacturing tech, the company said it can produce LVVs at significantly greater quantities than traditional transient transfection processes. The companys name is short for intelligent vector solutions.

Much like adeno-associated viral vectors, or AAV, these delivery vehicles are crucial for cell and gene therapies such as CAR-T, iVexSol added, and their shortage means developers often have to wait 12 to 18 months for production slots.

Details on exactly how it plans to revolutionize the space are scant, but CEO Rod Rietze and CSO Mike Greene both bring technical experience from shops like Novartis and Pfizer.

Its new funding will help establish a facility in Lexington, MA housing stable LVV producer cell line master banks and commercial-grade LVV. Amber Tong

Continue reading here:
News briefing: Merck's Roger Perlmutter buys his first solid tumor TriNKET from Dragonfly; ViGeneron to expand production of eye gene therapy -...

Gene therapy has shown promise for the first time to help treatbowelcancerthat has spread to the liver.

Adelaide researchers showed the novel approach, which uses a modified virus to infect the liver, was able to shrink tumours in mice.

"We're very excited by these results," Dr Susan Woods, one of the investigators in the study that was published in the Gastroenterology journal, said.

Further testing will be carried out to see whether the therapy could work for othercancers that travel to the liver such as tumours of the breast, lung and pancreas.

The modified virus exclusively targets the liver and introduces a copy of a gene that instructs the organ to make more cells called fibroblasts which are known to be good and slowcancergrowth.

The team from SAHMRI and the University of Adelaide have been investigating why normal cells that surround thecancerare good while others are corrupted and promote tumour growth.

"Inbowelcancer, we know that patients with the poorest prognosis have a lot of these corrupted or bad tumour supporting fibroblasts," Dr Susan Woods said.

This type of gene therapy that uses a modified virus to enter the liver is currently being used on patients with blood disorders.

"This is the first sign that we could use this to treatcancerthat has spread to the liver," Dr Woods said.

Bowelcancersurvivor Hannah Devereux is heartened by the research and said there needs to be more treatment options for people who are diagnosed with the disease late, when it has already spread.

Hannah was only 34 when she was diagnosed withbowelcancer, soon after her second child was born.

"Had the baby, he was 10 days old, and they found two tumours. My world came crashing down," she said.

Hannah had complained about digestive symptoms during the pregnancy.

"The doctor just thought it was pregnancy related," she said.

Hannah required intensive treatment for a year including six months of chemotherapy, radiotherapy and two major surgeries.

She has now reached the five-yearcancer-free milestone and is the ambassador of the Jodi Lee Foundation to preventbowelcancer.

Bowelcanceraffects more than 15,000 Australians each year and fewer than 50 per cent of cases are detected early.

More than 100 Australians each week die frombowelcancer.

More:
New gene therapy could help treat bowel cancer that reaches the liver - 9News

~ Strong Presence at ASH Featuring Five Presentations, Including Late-BreakingOral Presentation on HOPE-B Pivotal Trial ~

~ uniQure to Host Investor Webcast Tuesday, December 8, 2020 at 5:00 p.m. ET ~

LEXINGTON, Mass. and AMSTERDAM, Nov. 30, 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 Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, will present clinical data from the HOPE-B pivotal trial of etranacogene dezaparvovec in hemophilia B at the virtual 62nd American Society of Hematology (ASH) Annual Meeting.

uniQure management along with Dr. Pipe will host an investor webcast on Tuesday, December 8, 2020, at 5:00 p.m. ET. To access the live webcast with presentation slides, please visit the Investor Relations section of uniQures website at http://www.uniQure.com. The webcast will be archived for 90 days. The event also may be accessed by dialing (877) 870 9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode is 3164585. Please specify to the operator that you would like to join the uniQure Conference Call.

The conference abstracts are available and can be accessed through this link: ASH abstracts.

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 we will present long-term follow-up data from our hemophilia B gene therapy studies, including two years of follow-up on the Phase IIb clinical trial of etranacogene dezaparvovec (AMT-061) and up to five years of follow-up from the Phase I/II clinical trial of AMT-060, and whether we will announce top-line data from the pivotal HOPE-B study of etranacogene dezaparvovec before the end of this year. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, 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 Quarterly Report on Form 10-Q filed onOctober 27, 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:

Link:
uniQure Announces Multiple Presentations and Investor Webcast on Hemophilia B Gene Therapy Program at the 62nd American Society of Hematology (ASH)...

A new study on the CRISPR and Cas Genes market provides a detailed overview of the demands and consumptions of various products/services associated with the growth dynamics of the market. The in-depth market estimation of various opportunities in the segments is expressed in volumes and revenues during the forecast period 2020 to 2026. The insights and analytics on the CRISPR and Cas Genes market span several pages. These are covered in numerous sections, including, drivers and restraints, challenges and opportunities, regional segmentation and opportunity assessment, end-use/application prospects analysis, and competitive landscape assessment.The global revenues in CRISPR and Cas Genes market are projected to garner a CAGR of 21.2% from during 2020 to 2026.

Key stakeholders in the CRISPR and Cas Genes market including industry players, policymakers, and investors in various countries have been continuously realigning their strategies and approaches to implement them in order to tap into new opportunities. Many in recent months have overhauled their strategies to remain agile in the backdrop of worldwide disruptions caused by the COVID-19 pandemic.

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The study also offers scrutiny of the changing government policies amid COVID-19 disruptions. Policymakers in developing and developed nations are framing new regulations to meet the continuing macrocosmic shocks by COVID-19 pandemic. The authors of the report have taken into account the impact analysis of the pandemic, and have elaborated on the trends that will be crucial to the upcoming competitive landscape. New entrants, as well as established players who want to emerge as leaders in the post-COVID era, are taking the impact analysis seriously.

The following insights and evaluations are worth knowing for any market participant, helping them in ascertaining the prevailing dynamics and the future trajectories of the CRISPR and Cas Genes market. They are a part of the estimations of the opportunities in various segments.

Some of the insights and market estimations that make this study unique in approach and effective in guiding stakeholders in understanding the growth dynamics. The study provides:

The report offers detailed regional segmentation of the CRISPR and Cas Genes market which includes shares of each key region in the global landscape during the forecast period. The segmentation is done as follows:

The CRISPR and Cas Genes market report offers a critical quantitative and qualitative assessment of the current and future avenues in the following product segments:

The global CRISPR and Cas Genes market report comes with the following end uses:

A detailed assessment of the growth dynamics includes opportunities and growth trends. Some of the questions pertain to these are answered in the study:

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The report on the CRISPR and Cas Genes market is unique in its approach and insights-gathering initiatives. Some of the aspects that the study highlight are:

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Fact.MR Report: What is the impact of Coronavirus on CRISPR and Cas Genes Market Growth? - The Cloud Tribune

New York, Nov. 27, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989496/?utm_source=GNW However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The use of instruments is essential for handling cell therapies such as NSC, PSC, MSC, T cells, and HSC.These cell therapy products are derived from animals or human cells and thus need to be protected from contamination.

The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation. Companies such as Hitachi Chemical Advanced Therapeutics Solutions Corning Incorporated; Thermo Fisher Scientific Inc., MiltenyiBiotec, LLC; Invetech; and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

The global COVID-19 emergency has been particularly affecting the supply chain worldwide.The supply chain disruptions, along with the enormous demand for effective therapies for the treatment of COVID-19, have put the healthcare research industry in a crucial situation in the Asia Pacific region.

However, many medical companies have realized the importance of cell therapy in the treatment of COVID 19, which would raise its demand in the coming years.

The Asia Pacific cell therapy instruments market, by product, is segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

On the basis of cell type, the cell therapy instruments market is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during the forecast period.

On the basis of process, the Asia Pacific cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The Asia Pacific cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment accounted for the largest share of the market in 2019 and is anticipated to register the highest CAGR during the forecast period.

A few of the major primary and secondary sources associated with the Asia Pacific cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); Medical Research Future Fund (MRFF); Asia-Pacific Economic Corporation (APEC); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989496/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User,...

DUBLIN--(BUSINESS WIRE)--The "Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms" report has been added to ResearchAndMarkets.com's offering.

The research provides technological insights across inflammation, infectious diseases, and microbiomics.

The Life Science, Health & Wellness TOE will feature disruptive technology advances in the global life sciences industry. The technologies and innovations profiled will encompass developments across genetic engineering, drug discovery and development, biomarkers, tissue engineering, synthetic biology, microbiome, disease management, as well as health and wellness among several other platforms.

The Health & Wellness cluster tracks developments in a myriad of areas including genetic engineering, regenerative medicine, drug discovery and development, nanomedicine, nutrition, cosmetic procedures, pain and disease management and therapies, drug delivery, personalized medicine, and smart healthcare.

Innovations in Life Sciences, Health & Wellness from:

For more information about this report visit https://www.researchandmarkets.com/r/tkufmb

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2020 Report: Growth Opportunities in Gene Therapy, Automated Bioanalytics, and Biomarker Platforms - ResearchAndMarkets.com - Business Wire

A team of Irish scientists have developed a gene therapy that could potentially lead to a new treatment for an eye disease causing progressive loss of vision, which affects many thousands of people across the globe.

The breakthrough therapy, which offers hope for people with dominant optic atrophy (DOA), was devised by researchers at Trinity College Dublin in collaboration with clinicians at the Royal Victoria Eye and Ear Hospital and the Mater hospital in Dublin.

It also has implications for a much wider suite of neurological disorders associated with ageing which collectively affect millions of people worldwide.

DOA is an inherited disorder characterised by degeneration of optic nerves. It typically starts during the first decade of life. Affected people usually develop moderate visual loss and colour vision defects but severity varies, symptoms can worsen over time and some people become blind. At present, there is no way to prevent or cure DOA.

The scientists published their results in Frontiers in Neuroscience on Thursday.

A gene (OPA1) provides instructions for making a protein found in cells and tissues throughout the body that is pivotal to the proper function of mitochondria, the energy generators in cells.

Without the protein, mitochondrial function is sub-optimal; the normally well-interconnected mitochondrial network in healthy cells becomes highly disrupted. For those with DOA, it is mutations in OPA1 and the dysfunctional mitochondria that are responsible for onset and progression of the disorder.

Led by research fellow Dr Daniel Maloney and Prof Jane Farrar from TCD School of Genetics and Microbiology, the gene therapy successfully protected the visual function of mice who were treated with a chemical targeting the mitochondria and were consequently living with dysfunctional mitochondria.

The scientists also found the gene therapy improved mitochondrial performance in human cells that contained mutations in the OPA1 gene.

We used a clever lab technique that allows scientists to provide a specific gene to cells that need it using specially engineered non-harmful viruses, Dr Maloney said.

This allowed us to directly alter the functioning of the mitochondria in the cells we treated, boosting their ability to produce energy which, in turn, helps protect them from cell damage.

Excitingly, our results demonstrate that this OPA1-based gene therapy can potentially provide benefit for diseases like DOA, which are due to OPA1 mutations, and also possibly for a wider array of diseases involving mitochondrial dysfunction.

Mitochondrial dysfunction causes problems in neurological disorders such as Alzheimers and Parkinsons disease. The impacts gradually build up over time, which is why many may associate such disorders with ageing.

Prof Farrar added: We are very excited by the prospect of this new gene therapy strategy, although it is important to highlight that there is still a long journey to complete from a research and development perspective before this therapeutic approach may one day be available as a treatment.

Because mitochondrial dysfunction was implicated in so many neurological disorders, there was great potential for this type of therapeutic strategy to make a major societal impact, she said.

The research was supported by Science Foundation Ireland, the Health Research Board of Ireland, Fighting Blindness Ireland, and Health Research Charities Ireland.

Original post:
Irish scientists develop gene therapy for inherited vision loss disorder - The Irish Times

Nov. 24, 2020 12:00 UTC

Appoints former Chief Medical Officer of Spark Therapeutics, Kathy Reape, M.D., and Chief Scientific Officer of Intellia Therapeutics, Laura Sepp-Lorenzino, Ph.D., to board of directors

Directors bring significant gene therapy translational and development expertise ahead of GM2 gangliosidosis clinical trial initiation in 2020 and submission of four INDs by the end of 2021

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 the appointment of Kathy Reape, M.D., and Laura Sepp-Lorenzino, Ph.D., to the companys board of directors.

Drs. Reape and Sepp-Lorenzino bring significant gene therapy translational and development expertise to our board, said RA Session II, President, Founder and CEO of Taysha Gene Therapies. Their combined gene therapy experience across preclinical and clinical development will be invaluable as we continue to advance our broad portfolio into the clinic. Across all levels of the organization, we are building a team that has the passion, experience and talent to execute on our mission of eradicating monogenic CNS disease.

Dr. Reape was most recently Chief Medical Officer at Spark Therapeutics where she oversaw clinical development, pharmacovigilance and medical affairs activities and was a key member of the team responsible for the development and commercialization of the first FDA-approved in vivo gene therapy, LUXTURNA, for an inherited retinal disease caused by mutations in both copies of the RPE65 gene. She also oversaw the development of Sparks pipeline of gene therapies addressing CNS disease, hemophilia, metabolic disorders and inherited retinal dystrophies. She has over 18 years of experience in the pharmaceutical industry in clinical research and development and has been involved with approximately two dozen product approvals including small molecules, biologics, biosimilars and therapeutic devices. She received both her undergraduate and M.D. degrees from the University of Pennsylvania and completed her internship and residency at the University of Florida and University of Medicine and Dentistry of New Jersey.

Taysha has built a deep pipeline of potentially transformative gene therapies for patients with life-threatening CNS diseases, said Dr. Reape. Many of the conditions that Taysha is addressing have no therapeutic alternatives, are associated with a poor quality of life and often result in a shortened life expectancy. It is important that we rapidly advance these gene therapies into the clinic to serve patients so desperately in need.

Dr. Sepp-Lorenzino is currently the Chief Scientific Officer at Intellia Therapeutics and has held several senior positions over her extensive career. Most recently, she was Vice President and Head of Nucleic Acid Therapies at Vertex Pharmaceuticals. She previously served as Alnylams Vice President and Entrepreneur-in-Residence, where she led the hepatic infectious disease strategies therapeutic area and was a key figure in partnering and in-licensing activities. She spent 14 years at Merck & Co., including as Executive Director and Department Head of the RNA therapeutics discovery biology unit. Dr. Sepp-Lorenzino received her degree in biochemistry at the University of Buenos Aires, Argentina and her M.S. and Ph.D. in biochemistry from New York University.

Joining the Taysha board is a truly exceptional opportunity to contribute to the development of multiple innovative gene therapies, commented Dr. Sepp-Lorenzino. Taysha is taking a leadership position in the industry by combining decades of gene therapy experience with a portfolio of programs that have the potential to address the underlying biology of various CNS disorders in order create an engine for potential new treatments.

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.

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 conduct or timing of our clinical trials and our research, development and regulatory plans for our product candidates, the potential of our product candidates to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for 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 (SEC) filings, including in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, 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/20201124005342/en/

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Taysha Gene Therapies Adds Industry-Leading Gene Therapy Executives to Board of Directors - BioSpace

AUSTIN, Texas--(BUSINESS WIRE)--Genprex, Inc. (Genprex or the Company) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that the Company will participate at the Diamond Equity Research Emerging Growth Invitational taking place virtually on December 1, 2020. Genprexs Executive Vice President and Chief Operating Officer, Michael Redman, will virtually deliver a company overview to investors followed by a guided question and answer session.

Event: Diamond Equity Research Emerging Growth InvitationalDate: Tuesday, December 1Time: 11:40 a.m. ESTRegistration Link: https://bit.ly/2UJS3Si

A live audio webcast and archive of the conference presentation will be available for a period of time using the registration link above. For more information on the Diamond Equity Research Emerging Growth Invitational, please contact your Diamond Equity Research representative.

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprexs technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Companys lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance. In January 2020, the U.S. Food and Drug Administration granted Fast Track Designation for REQORSA for NSCLC in combination therapy with osimertinib (AstraZenecas Tagrisso) for patients with EFGR mutations whose tumors progressed after treatment with osimertinib alone.

For more information, please visit the Companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effect of Genprexs product candidates, alone and in combination with other therapies, on cancer and diabetes, regarding potential, current and planned clinical trials, regarding the Companys future growth and financial status and regarding our commercial partnerships and intellectual property licenses. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of the effect of our product candidates, alone and in combination with other therapies, on cancer; the timing and success of our clinical trials and planned clinical trials of REQORSA immunogene therapy drug, alone and in combination with targeted therapies and/or immunotherapies, and whether our other potential product candidates, including GPX-002, our gene therapy in diabetes, advance into clinical trials; the success of our strategic partnerships, including those relating to manufacturing of our product candidates; the timing and success at all of obtaining FDA approval of REQORSA and our other potential product candidates including whether we receive or benefit from fast track or similar regulatory designations; costs associated with developing our product candidates, whether we identify and succeed in acquiring other technologies and whether patents will ever be issued under patent applications that are the subject of our license agreements or otherwise. These and other risks and uncertainties are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex to Participate at the Diamond Equity Research Emerging Growth Invitational on December 1 - Business Wire

"Despite the projected growth in market applications andabundant investment capital, there is a danger that legal andethical concerns related to genetic research could put the brakeson gene editing technologies and product programs emanatingtherefrom."

There are thousands of diseases occurring in humans, animals,and plants caused by aberrant DNA sequences. Traditional smallmolecule and biologic therapies have only had minimal success intreating many of these diseases because they mitigate symptomswhile failing to address the underlying genetic causes. While humanunderstanding of genetic diseases has increased tremendously sincethe mapping of the human genome in the late 1990s, our ability totreat them effectively has been limited by our historical inabilityto alter genetic sequences.

The science of gene editing was born in the 1990s, as scientistsdeveloped tools such as zinc-finger nucleases (ZFNs) and TALEnucleases (TALENs) to study the genome and attempt to altersequences that caused disease. While these systems were anessential first step to demonstrate the potential of gene editing,their development was challenging in practice due to the complexityof engineering protein-DNA interactions.

Then, in 2011, Dr. Emmanuelle Charpentier, a French professor ofmicrobiology, genetics, and biochemistry, and Jennifer Doudna, anAmerican professor of biochemistry, pioneered a revolutionary newgene-editing technology called CRISPR/Cas9. Clustered Regularly InterspacedShort Palindromic Repeats (CRISPR) and Cas9 stands forCRISPR-associated protein 9. In 2020, the revolutionary work ofDrs. Charpentier and Doudna developing CRISPR/Cas9 were recognizedwith the Nobel Prize for Chemistry. The technology was also thesource of a long-running and high-profile patent battle between two groups ofscientsists.

CRISPR/Cas9 for gene editing came about from a naturallyoccurring viral defense mechanism in bacteria. The system ischeaper and easier to use than previous technologies. It deliversthe Cas9 nuclease complexed with a synthetic guide RNA (gRNA) intoa cell, cutting the 'cell's genome at the desired location,allowing existing genes to be removed and new ones added to aliving organism's genome. The technique is essential inbiotechnology and medicine as it provides for the genomes to beedited in vivo with extremely high precision, efficiently, and withcomparative ease. It can create new drugs, agricultural products,and genetically modified organisms or control pathogens and pests.More possibilities include the treatment of inherited geneticdiseases and diseases arising from somatic mutations such ascancer. However, its use in human germline genetic modification ishighly controversial.

The following diagram from CRISPR Therapeutics AG, a Swisscompany, illustrates how it functions:

In the 1990s, nanotechnology and gene editing were necessaryplot points for science fiction films. In 2020, developments likenano-sensors and CRISPR gene editing technology have moved thesetechnologies directly into the mainstream, opening a new frontierof novel market applications. According to The Business ResearchCompany, the global CRISPR technology market reached a value ofnearly $700 million in 2019, is expected to more than double in2020, and reach $6.7 billion by 2030. Market applications targetall forms of life, from animals to plants to humans.

Gene editing's primary market applications are for thetreatment of genetically-defined diseases. CRISPR/Cas9 gene editingpromises to enable the engineering of genomes of cell-basedtherapies and make them safer and available to a broader group ofpatients. Cell therapies have already begun to make a meaningfulimpact on specific diseases, and gene editing helps to acceleratethat progress across diverse disease areas, including oncology anddiabetes.

In the area of human therapy, millions of people worldwidesuffer from genetic conditions. Gene-editing technologies likeCRISPR-Cas9 have introduced a way to address the cause ofdebilitating illnesses like cystic fibrosis and create betterinterventions and therapies. They also have promising marketapplications for agriculture, food safety, supply, anddistribution. For example, grocery retailers are even looking athow gene editing could impact the products they sell. Scientistshave created gene-edited crops like non-browning mushrooms andmildew-resistant grapes - experiments that are part of an effort toprevent spoilage, which could ultimately change the way food issold.

Despite the inability to travel and conduct face-to-facemeetings, attend industry conferences or conduct business otherthan remotely or with social distance, the investment markets forventure, growth, and private equity capital, as well as corporateR&D budgets, have remained buoyant through 2020 to date.Indeed, the third quarter of 2020 was the second strongest quarterever for VC-backed companies, with 88 companies raising roundsworth $100 million or more according to the latest PwC/Moneytreereport. Healthcare startups raised over $8 billion in the quarterin the United States alone. Gene-editing company MammouthBiosciences raised a $45 million round of Series B capital in thesecond quarter of 2020. CRISPR Therapeutics AG raised more in thepublic markets in primary and secondary capital.

Bayer, Humboldt Fund and Leaps are co-leading a $65 million Series A round for Metagenomi, abiotech startup launched by UC Berkeley scientists. Metagenomi,which will be run by Berkeley's Brian Thomas, is developing atoolbox of CRISPR- and non-CRISPR-based gene-editing systems beyondthe Cas9 protein. The goal is to apply machine learning to searchthrough the genomes of these microorganisms, finding new nucleasesthat can be used in gene therapies. Other investors in the Series Ainclude Sozo Ventures, Agent Capital, InCube Ventures and HOFCapital. Given the focus on new therapies and vaccines to treat thenovel coronavirus, we expect continued wind in the sails forgene-editing companies, particularly those with strong productportfolios that leverage the technology.

Despite the projected growth in market applications and abundantinvestment capital, there is a danger that legal and ethicalconcerns related to genetic research could put the brakes ongene-editing technologies and product programs emanating therefrom.The possibility of off-target effects, lack of informed consent forgermline therapy, and other ethical concerns could cause governmentregulators to put a stop on important research and developmentrequired to cure disease and regenerate human health.

Gene-editing companies can only make money by developingproducts that involve editing the human genome. The clinical andcommercial success of these product candidates depends on publicacceptance of gene-editing therapies for the treatment of humandiseases. Public attitudes could be influenced by claims that geneediting is unsafe, unethical, or immoral. Consequently, productscreated through gene editing may not gain the acceptance of thegovernment, the public, or the medical community. Adverse publicreaction to gene therapy, in general, could result in greatergovernment regulation and stricter labeling requirements ofgene-editing products. Stakeholders in government, third-partypayors, the medical community, and private industry must work tocreate standards that are both safe and comply with prevailingethical norms.

The most significant danger to growth in gene-editingtechnologies lies in ethical concerns about their application tohuman embryos or the human germline. In 2016, a group of scientistsedited the genome of human embryos to modify the gene forhemoglobin beta, the gene in which a mutation occurs in patientswith the inherited blood disorder beta thalassemia. Althoughconducted in non-viable embryos, it shocked the public thatscientists could be experimenting with human eggs, sperm, andembryos to alter human life at creation. Then, in 2018, abiophysics researcher in China created the first human geneticallyedited babies, twin girls, causing public outcry (and triggeringgovernment sanctioning of the researcher). In response, the WorldHealth Organization established a committee to advise on thecreation of standards for gene editing oversight and governancestandards on a global basis.

Some influential non-governmental agencies have called for amoratorium on gene editing, particularly as applied to altering thecreation or editing of human life. Other have set forth guidelineson how to use gene-editing technologies in therapeuticapplications. In the United States, the National Institute ofHealth has stated that it will not fund gene-editing studies inhuman embryos. A U.S. statute called "The Dickey-WickerAmendment" prohibits the use of federal funds for researchprojects that would create or destroy human life. Laws in theUnited Kingdom prohibit genetically modified embryos from beingimplanted into women. Still, embryos can be altered in researchlabs under license from the Human Fertilisation and EmbryologyAuthority.

Regulations must keep pace with the change that CRISPR-Cas9 hasbrought to research labs worldwide. Developing international guidelines could be a steptowards establishing cohesive national frameworks. The U.S.National Academy of Sciences recommended seven principles for thegovernance of human genome editing, including promoting well-being,transparency, due care, responsible science, respect for persons,fairness, and transnational co-operation. In the United Kingdom, anon-governmental organization formed in 1991 called The NuffieldCouncil has proposed two principles for the ethical acceptabilityof genome editing in the context of reproduction. First, theintervention intends to secure the welfare of the individual borndue to such technology. Second, social justice and solidarityprinciples are upheld, and the intervention should not result in anintensifying of social divides or marginalizing of disadvantagedgroups in society. In 2016, in application of the same, the CrickInstitute in London was approved to use CRISPR-Cas9 in humanembryos to study early development. In response to a cacophony ofconflicting national frameworks, the International Summit on HumanGene Editing was formed in 2015 by NGOs in the United States, theUnited Kingdom and China, and is working to harmonize regulationsglobal from both the ethical and safety perspectives. As CRISPRco-inventor Jennifer Doudna has written in a now infamous editorialin SCIENCE, "stakeholders must engage in thoughtfullycrafting regulations of the technology without stiflingit."

The COVID-19 pandemic has forced us to rely more on newtechnologies to keep us healthy, adapt to working from home, andmore. The pandemic makes us more reliant on innovative digital,biological, and physical solutions. It has created a united senseof urgency among the public and private industry (together withgovernment and academia) to be more creative about using technologyto regenerate health. With continued advances in computing power, networkarchitecture, communications bandwidths, artificial intelligence,machine learning, and gene editing, society will undoubtedly findmore cures for debilitating disease and succeed in regeneratinghuman health. As science advances, it inevitably intersects withlegal and ethical norms, both for individuals and civil society,and there are new externalities to consider. Legal and ethicalnorms will adapt, rebalancing the interests of each. The fourthindustrial revolution is accelerating, and hopefully towards curingdisease.

Originally published by IPWatchdog.com, November 24,2020.

The content of this article is intended to provide a generalguide to the subject matter. Specialist advice should be soughtabout your specific circumstances.

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Future Visioning The Role Of CRISPR Gene Editing: Navigating Law And Ethics To Regenerate Health And Cure Disease - Technology - United States -...

Market Study Report, LLC, has recently added a report on the Gene Therapy market which presents substantial inputs about the market size, market share, regional trends, and profit projection of this business sphere. The report also enlightens users regarding the foremost challenges and existing growth tactics implemented by the leading organizations that constitute the dynamic competitive gamut of this industry.

The Gene Therapy market is projected to accomplish a very enviable valuation portfolio by the end of the estimated duration, claims this report. The research study also enumerates that this vertical will register a highly commendable growth rate over the forecast timeframe, while simultaneously elucidating a pivotal overview of this business space. Inclusive of highly significant details pertaining to the overall valuation presently held by this industry, the report also lists down, in meticulous detail, the segmentation of the Gene Therapy market and the growth opportunities prevailing across this vertical.

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Enumerating a basic coverage of the Gene Therapy market report:

What pointers are covered in the report with respect to the geographical spectrum of Gene Therapy market?

How meticulously is the Gene Therapy market segmented?

. The report comprises details about the market share that each product holds and the projected valuation of the segment.

. The market share which each application holds alongside the projected valuation that each application will account for is also incorporated in the study.

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What are the drivers & challenges of the Gene Therapy market?

An analysis of the important competitors in the Gene Therapy market:

The Gene Therapy market report is also comprised of some details such as market concentration ratio, spanning the concentration classes CR3, CR10, and CR5 over the projected timeline.

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Global Gene Therapy Market Size, Analytical Overview, Growth Factors, Demand, Trends and Forecast to 2025 - Cheshire Media

The Global Gene Therapy Market Research Report Forecast 2020 2024 provides a comprehensive analysis of the market segments, including their dynamics, size, growth, regulatory requirements, competitive landscape, and emerging opportunities of the global industry. It provides an in-depth study of the Gene Therapy market by using SWOT analysis. The research analysts provide an elaborate description of the value chain and its distributor analysis. This Market study provides comprehensive data that enhances the understanding, scope, and application of this report.

The global gene therapy market is expected to reach US$6.42 billion in 2024, witnessing growth at a CAGR of 19.29%, over the period 2020-2024.

(Exclusive Offer: Up to 10% discount on this report)

Get a Sample PDF Copy of the Latest Research on Gene Therapy Market 2020 Before the purchase:

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Segmentation:

Top Key Players in the Global Gene Therapy Market: Roche Holding AG, Pfizer Inc., Novartis International AG, GlaxoSmithKline PLC, Bristol Myers Squibb Co. (Celgene Corporation) and Gilead Sciences, Inc.

Growth in the gene therapy market has accrued due to the increasing prevalence of chronic diseases, rising healthcare expenditure, expanding urbanization, growth of gene therapy clinical trials and upsurge in economic growth. The market is anticipated to experience certain trends like rapid adoption of personalized medicine, growing occurrence of genetic disorders, advancements in gene therapy and increasing R&D funding. The growth of the market would be challenged by side effects of gene therapy and ethical and safety concerns and high cost of the treatment.

The global gene therapy market has been segmented on the basis of cell type, vector type, application, end-user and region. Depending on the cell type, the market can be bifurcated into somatic cell gene therapy and germ cell gene therapy. According to the vector type, the global gene therapy market can be categorized into retrovirus & gammaretrovirus, adeno-associated viruses (AAV), lentivirus, adenovirus, modified herpes simplex virus and non-viral plasmid vector. Whereas, on the basis of application, the market can be split into oncological disorders, neurological disorders, infectious diseases, cardiovascular diseases, rare diseases and others. Further, in terms of end-user, the global gene therapy market can broadly be segmented into hospitals, specialty treatment centers and other end-users.

The fastest growing regional market is North America due to the rising incidence of cancer and other target diseases, increasing favorable reimbursement scenario in the region and improvements in healthcare infrastructure. Further, the sudden outbreak of COVID-19 is causing an adverse disruption on the overall economy and society, affecting the rate of gene therapy procedures and clinical trials, which is expected to negatively impact the growth of the global gene therapy market during the forecasted period.

Scope of the report:

The report provides a comprehensive analysis of the global gene therapy market segmented on the basis of cell type, vector type, application, end-user and region. The major regional and country markets (North America, Europe, Asia Pacific and Rest of the World) have been analyzed. The market dynamics such as growth drivers, market trends and challenges are analyzed in-depth. The competitive landscape of the market, along with the company profiles of leading players (Roche Holding AG, Pfizer Inc., Novartis International AG, GlaxoSmithKline PLC, Bristol Myers Squibb Co. (Celgene Corporation) and Gilead Sciences, Inc.) are also presented in detail.

Regional Analysis for Market:

For a comprehensive understanding of market dynamics, the global Gene Therapy market is analyzed across key geographies namely: United States, China, Europe, Japan, South-east Asia, India, and others. Each of these regions is analyzed on the basis of market findings across major countries in these regions for a macro-level understanding of the market.

This Gene Therapy Market statistical surveying report underlines the leading merchants in this market everywhere throughout the world. This sector of the report includes market depictions, requirements, and product portrayals, manufacture, competence, contact figures, cost, and revenue. In a comparable way, automated gathering, upstream raw materials, and downstream demand studies are administered.

Browse Complete Report details with Table of Content:

https://www.marketinsightsreports.com/reports/08312273097/global-gene-therapy-market-by-cell-type-vector-type-application-end-user-region-insights-forecast-with-potential-impact-of-covid-19-2020-2024?mode=69

Table Of Content:

Overview: This segment offers an overview of the report to provide an idea regarding the contents and nature of the research report along with a wide synopsis of the global Gene Therapy Market.

Analysis of Leading Players Strategies: Market top players can utilize this analysis to increase the upper hand over their rivals in the market.

Study on Major Market Trends: This segment of the report delivers a broad analysis of the most recent and future market trends.

Forecasts of the Market: The report gives production, consumption, sales, and other market forecasts. Report Buyers will approach exact and approved evaluations of the total market size in terms of value and volume.

Analysis of Regional Growth: This report covered all major regions and countries. The regional analysis will assist market players to formulate strategies specific to target regions, tap into unexplained regional markets, and compare the growth of all regional markets.

The research includes historic data from 2015 to 2020 and forecasts until 2024 which makes the reports an invaluable resource for industry executives, marketing, sales, and product managers, consultants, analysts, and other people looking for key industry data in readily accessible documents with clearly presented tables and graphs.

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Gene Therapy Market Insights and Global Research and Clinical Survey Report 2020-2024 - The Market Feed

The latest report published by Polaris Market Research, titled Global Gene Therapy Market provides key information about the current status and prospects of the market. The report focuses on the market size, share, growth, emerging trends, and regional analysis of the market. The study also includes a comprehensive analysis of the various market factors, including market drivers, restraints, trends, risk, and opportunities prevailing within the market.

The report provides an in-depth analysis of the global Gene Therapy market that could help market players to devise their strategies and leads to the profitability of their business. The study also profiles key companies present in the market along with their market shares, growth rate, and product launches. The rapidly changing market scenario covering the initial and future assessment of the impact is covered in the report. Further, the report provides niche insights for every possible segment to help market players in the strategic decision-making process and market size estimation of the global Gene Therapy market on a regional and global basis.

Get a Sample Copy of the Report: https://www.polarismarketresearch.com/industry-analysis/gene-therapy-market/request-for-sample

The report produced by Polaris Market Research is widely known for its accuracy as it consists of precise graphs, tables, and figures that give a clear picture of the developments of the products and their market performance in the past and predicts the future trend. It uses statistical surveying for SWOT analysis, PESTLE analysis, predictive analysis, and real-time analysis.

Manufacturers covered in this report are:

Novartis Pharmaceuticals Corporation, Kite Pharma, Incorporated, Spark Therapeutics, Inc, GlaxoSmithKline, Adaptimmune, Bluebird bio

*Note: Additional companies can be included on request

Geographical Overview

The report provides an extensive analysis of the key geographical regions of the market. The regional analysis focused on North America, Latin America, Europe, Asia-Pacific, and Middle East & Africa regions. The report offers insightful information such as demand and supply, production and consumption ratio, import and export, and demand trends in each region. The report also includes covers a country-specific analysis of the each segment and sub-segment of the Gene Therapy market.

Impact of COVID-19

The report also takes into account the impact of the ongoing global crisis of the COVID-19 pandemic, on the global Gene Therapy market. It covers the key information current status of the industries and predicts the post-COVID-19 impact on the global market. The report also sheds light on the financial impact on businesses at the domestic level as well as global level. Polaris Market Research has gathered insights from various industry delegates and has been involved in primary and secondary research to provide customers with data and strategies to address market challenges during COVID-19 and post the COVID-19 pandemic.

Key questions answered in the report

The global Gene Therapy market report will help clients to gain an in-depth understanding of the global Gene Therapy market. The report identifies the ongoing market trend and anticipated growth in the near future to help industry vendors, manufacturers, consultants, and suppliers to align their market-centric strategies. The analytical studies are conducted to identify ensure customer requirements with a thorough understanding of the market capacities.

If you have any special requirements, please let us know and we will offer you the report as you want. Speak to Analyst to know more @ https://www.polarismarketresearch.com/industry-analysis/gene-therapy-market/speak-to-analyst

About Polaris Market Research

Polaris Market Research is a global market research and consulting company. We provide unmatched quality of offerings to our clients present globally. The company specializes in providing exceptional market intelligence and in-depth business research services for our clientele spread across different enterprises.

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Gene Therapy Market Potential Growth, Share and Demand-Analysis of Key Players- Research Forecasts - Cheshire Media

Bridging the gap: SYNGAP1 protein is located mostly at synapses, the junctions between neurons (green). Editors Note

This article was originally published on 24 November, based on preliminary data presented at a conference. We have updated the article to provide additional context.

Spectrum is covering the 2020 International SYNGAP1 Scientific Conference, which took place virtually because of the coronavirus pandemic. Here were highlighting researchers reactions to noteworthy presentations.

Drug test: A new assay allows researchers to test thousands of candidate drugs for their ability to boost expression of the autism gene SYNGAP1. The tool may help researchers identify and screen potential treatments for people with mutations that silence the gene. Gavin Rumbaugh, professor of neuroscience at Scripps Research in Jupiter, Florida, presented the unpublished results on 18 November.

The assay uses neurons from mice with one intact and one mutated copy of SYNGAP1. The researchers genetically engineer the mice so that SYNGAP1 protein made from the intact copy is tagged with luciferase the enzyme that gives fireflies their glow.

They then grow these neurons in tiny wells and add a different candidate drug to each well. The amount of SYNGAP1 protein in the dish gives a proportionate amount of light in your well, Rumbaugh says.

Rumbaugh and his team plan to use the platform to run through more than 100,000 different experimental compounds in 2021, he says.

Thats going to be really exciting for drug discovery efforts for SYNGAP1. I think thats going to be a game changer, says Karun Singh, senior scientist at the University Health Network in Toronto, Canada, who was not involved in the work.

It will be very exciting to see if they are able to uncover any useful hits with their novel approach, says Helen Bateup, associate professor of neurobiology at the University of California, Berkeley, who was not involved in the work.

Treatment across ages: A leading theory of autism is that the condition is characterized by a signaling imbalance: too much excitation or too little inhibition in the brain. One of the key players in creating this imbalance is thought to be inhibitory interneurons, which employ the neurotransmitter gamma-aminobutyric acid (GABA). And mutations to SYNGAP1 may disrupt GABAs function, said James Clement, assistant professor of neuroscience at the Jawaharlal Nehru Centre for Advanced Scientific Research in Bangalore, India, in a presentation on 18 November.

GABA is excitatory early on in brain development and inhibitory later on a switch that seems to be impaired in mice with SYNGAP1 mutations, he says. He and his team have tested a new compound that restores the GABA switch in mice and eases almost all SYNGAP1-related traits including seizures, learning issues and motor impairment in the mice. It works in newborn and adolescent mice. Due to a pending patent application, Clement and his lab are not revealing the compounds name.

I think its important to test efficacy at multiple ages, as they have done, to understand which phenotypes or problems can be improved with early treatment and which might still be responsive to treatment even if its administered later in life, says Bateup, who was not involved in the work. The idea that GABA may remain depolarizing for longer in SYNGAP1 mutant mice is quite interesting.

Clements lab was the only other lab that was presenting at this meeting that presented data from a very early age, says Shilpa Kadam, associate professor of neurology at the Kennedy Krieger Institute in Baltimore, Maryland, who was not involved in the work. Kadam also presented results on mice with SYNGAP1 mutations, showing that from an early age, theseanimalshave seizures thatcan be treated by blocking one type of GABA receptor.

Motor coordination: For mice, the loss of SYNGAP1 function in the striatum impairs their goal-directed learning and seems to lead to inflexible behavior, Bateup said in a presentation on 18 November.

Helen Bateups work looking at striatal function as it relates to motor coordination and motor learning is also pretty exciting and may shed light not only on the motor-coordination difficulties but also the repetitive or habitual motor behaviors, says Constance Smith-Hicks, child neurologist and research scientist at the Kennedy Krieger Institute, who was not involved in the work.

Bateups presentation also demonstrated that SYNGAP1 deletion seems to affect neurons differently depending on which type of dopamine receptor they express.

We know SYNGAP1 is at most excitatory synapses, so why shes seeing some functional effects in one type of cell and not the other, I find that interesting, says Richard Huganir, professor of neuroscience and psychological and brain sciences at Johns Hopkins University in Baltimore, Maryland, who was not involved in the work.

Its exciting to be able to kind of pinpoint which pathway might be involved and get a better understanding of the circuits that are disrupted, says Singh, who was not involved in the work.

Protein levels: People with a nonfunctional copy of SYNGAP1 have about half the typical amount of SYNGAP1 protein. Increasing the activity of the intact copy of the gene could help restore typical functioning, Huganir said in a presentation on 18 November.

He and his team tested this idea on two unique mouse models in unpublished work. Instead ofhaving one intact and one missing copyof the SYNGAP1 gene, as is typical for SYNGAP1 mouse models, each mouse model carriesone intact copy of the gene and one with a mutation seen in people. Both mice produce about half the typical amount of SYNGAP1 protein and show the same behaviors as the classical knockout mouse, despite having different types of mutations.

These new mouse models are crucial because they can directly correlate to what is happening in the humans, says Clement, who was not involved in the work.

Huganir and his team are testing different types of gene therapies to increase SYNGAP1 protein up to the typical levels, and have found that there are two SYNGAP1 protein isoforms, or slight variations of the protein.

One of the isoforms can restore synaptic plasticity in the animal model for SYNGAP1, so I think thats really exciting because even though theres multiple isoforms, it seems that one might be more important from a gene therapy point of view, says Singh, who was not involved in the work. Its pretty exciting to have a specific target now.

Read more reports from the2020 International SYNGAP1 Scientific Conference.

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Reactions from the 2020 SYNGAP1 Scientific Conference - Spectrum

New York, Nov. 25, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "North America Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989572/?utm_source=GNW

The surge in the number of cell therapy transplantation procedures, growing research and development activities, and rising investments in building production facilities for cell and gene therapy products drive the growth of the North America cell therapy instruments market. However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The cell therapy products are derived from animals or human cells and thus need to be protected from contamination.The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation.

Companies such as Hitachi Chemical Advanced Therapeutics Solutions; Corning Incorporated; Thermo Fisher Scientific Inc.; Miltenyi Biotec, LLC; Invetech, and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

In North America, various healthcare research centers are focused specifically on COVID 19 therapeutics.During the initial phase of COVID-19 crisis, the demand for cell therapy instruments was disrupted owing to change in customer behaviors due to lockdown and supply chain disruptions.

However, the lack of definitive therapy offers significant opportunities for cell therapy instrumentation market players as US FDA has recently approved use of plasma therapy for critically ill COVID 19 patients. Further, the cell therapy instruments market also has significant growth prospects during the post-pandemic period.

On the basis of product, the North America cell therapy instruments market is further segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

The cell therapy instruments market, based on cell type, is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during 20202027.

On the basis of process, the North America cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The North America cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment grasped the greatest share of the market in 2019 and is anticipated to register the highest CAGR from 2020 to 2027.

A few of the major primary and secondary sources associated with this report on the North America cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); American Society of Gene & Cell Therapy (ASGCT); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989572/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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North America Cell Therapy Instruments Market Forecast to 2027 - COVID-19 Impact and Regional Analysis By Product ; Cell Type ; Process ; End User,...

The report provides revenue of the global Gene Therapy in Oncology market for the period 20162026, considering 2019 as the base year and 2026 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global market during the forecast period. The global Gene Therapy in Oncology market studies past as well as current growth trends and opportunities to gain valuable insights of these indicators of the market during the forecast period from 2020 to 2026.

With the advancement in technology, the gene therapy market has transformed during the recent few years. Cancer gene therapy is a technique used for the treatment of cancer where therapeutic DNA is being introduced into the gene of the patient with cancer.

Due to the high success rate during the preclinical and clinical trial, cancer gene therapy is gaining popularity. There are many techniques used for cancer gene therapy, for example, a procedure where the mutated gene is being replaced with a healthy gene or inactivation of gene whose function is abnormal. Recently, a new technique has been developed, where new genes are introduced into the body to help fight against cancer cells.

The study offers a comprehensive analysis on diverse features, including production capacities, demand, product developments, revenue generation, and sales in the Gene Therapy in Oncology market across the globe.

Request Coronavirus Impact Analysis on This [emailprotected] https://www.qyresearchmedical.com/sample/57077

A comprehensive estimate on the Gene Therapy in Oncology market has been provided through an optimistic scenario as well as a conservative scenario, taking into account the sales of Gene Therapy in Oncology during the forecast period. Price point comparison by region with global average price is also considered in the study.

It is pertinent to consider that in a volatile global economy, we havent just conducted Gene Therapy in Oncology market forecasts in terms of CAGR, but also studied the market based on key parameters, including Year-on-Year (Y-o-Y) growth, to comprehend the certainty of the market and to find and present the lucrative opportunities in market.

Download Full Report https://www.qyresearchmedical.com/report/checkout/57077/3500

Drivers and Restraints

This section covers the various factors driving the global Advanced Driver Assistance Systems (ADAS) market. To understand the growth of the market it is important to analyze the various drivers present the market. It provides data by value and volume of different regions and their respective manufacturers. This data will elaborate on the market share occupied by them, predict their revenue concerning strategies, and how they will grow in the future. After explaining the drivers, the report further evaluates the new opportunities and current trends in the market.

Market restraints are factors hampering market growth. Studying these factors is equally pivotal as they help a reader need understand the weaknesses of the market.

Market Segmentation:

The report is divided into major categories comprising product, application, regions and others. Every segment is further sub-segmented into several sub-segmented that are deeply analyzed by experts to offer valuable information to the buyers and market players. Every segment is studied thoroughly in order to offer a better picture to the buyers and stakeholders to benefit from. Information like highest prevailing product, highly demanded product by the application segment and end users are rightly mentioned in the Gene Therapy in Oncology report.

The report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments included in the scope of the study. Furthermore, the report sheds light on the changing competitive dynamics in the global Gene Therapy in Oncology market. These indices serve as valuable tools for existing market players as well as for entities interested in entering the global Gene Therapy in Oncology market.

Gene Therapy in Oncology Breakdown Data by TypeEx VivoIn VivoGene Therapy in Oncology Breakdown Data by ApplicationHospitalsDiagnostics CentersResearch Institutes

In this study, the years considered to estimate the market size of Gene Therapy in Oncology are as follows:

Major Players Covered in this Report

The updated market research report on Gene Therapy in Oncology market allows the buyers and manufacturers to stay updated with the current market trends, ongoing happenings, and a clear picture on the market scenario. List of key players included in the research report will help the market vendors to know their market position and plan more operational strategies to gain topmost position among other players. The report offers crucial company information on each market player, such as company profile, financial information, and recently adopted growth strategies. This will help other existing players and the new entrants to plan strategies and establish their presence in the market.

Major manufacturers & their revenues, percentage splits, market shares, growth rates and breakdowns of the product markets are determined through secondary sources and verified through the primary sources.

Bristol-Myers SquibbCold GenesysAdvantageneAmgenAstraZenecaBio-Path HoldingsCRISPR TherapeuticsEditas MedicineGeron CorpIdera PharmaceuticalsIntellia TherapeuticsJohnson & JohnsonMarsala BiotechMerckMologen AGOncolytics BiotechOncosecOncotelicShenzhen SiBiono GeneTechSillajen BiotherapeuticsTocagenUniQureZiopharm Oncology

Regional Insights:

The Gene Therapy in Oncology market is segmented as North America, South America, Europe, Asia Pacific, and Middle East and Africa. Researchers have thoroughly studied about the historical market. With extensive research, experts have offered details on the current and the forecast demand made by these regions. The Gene Therapy in Oncology report also includes highlights on the prevailing product demanded by end users and end customers for better understanding of product demand by producers. This will help the producers and the marketing executives to plan their production quantity and plan effective marketing strategies to more buyers. Businesses can hence, increase their product portfolio and expand their global presence. Gene Therapy in Oncology market research report further offers information on the unexplored areas in these regions to help the producers to plan promotional strategies and create demand for their new and updated products. This will again help the manufacturers to increase their customers and emerge as leaders in the near future.

TABLE OF CONTENT

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered: Ranking by Gene Therapy in Oncology Revenue1.4 Market Analysis by Type1.4.1 Global Gene Therapy in Oncology Market Size Growth Rate by Type: 2020 VS 20261.4.2 Ex Vivo1.4.3 In Vivo1.5 Market by Application1.5.1 Global Gene Therapy in Oncology Market Share by Application: 2020 VS 20261.5.2 Hospitals1.5.3 Diagnostics Centers1.5.4 Research Institutes1.6 Coronavirus Disease 2019 (Covid-19): Gene Therapy in Oncology Industry Impact1.6.1 How the Covid-19 is Affecting the Gene Therapy in Oncology Industry1.6.1.1 Gene Therapy in Oncology Business Impact Assessment Covid-191.6.1.2 Supply Chain Challenges1.6.1.3 COVID-19s Impact On Crude Oil and Refined Products1.6.2 Market Trends and Gene Therapy in Oncology Potential Opportunities in the COVID-19 Landscape1.6.3 Measures / Proposal against Covid-191.6.3.1 Government Measures to Combat Covid-19 Impact1.6.3.2 Proposal for Gene Therapy in Oncology Players to Combat Covid-19 Impact1.7 Study Objectives1.8 Years Considered

2 Global Growth Trends by Regions2.1 Gene Therapy in Oncology Market Perspective (2015-2026)2.2 Gene Therapy in Oncology Growth Trends by Regions2.2.1 Gene Therapy in Oncology Market Size by Regions: 2015 VS 2020 VS 20262.2.2 Gene Therapy in Oncology Historic Market Share by Regions (2015-2020)2.2.3 Gene Therapy in Oncology Forecasted Market Size by Regions (2021-2026)2.3 Industry Trends and Growth Strategy2.3.1 Market Top Trends2.3.2 Market Drivers2.3.3 Market Challenges2.3.4 Porters Five Forces Analysis2.3.5 Gene Therapy in Oncology Market Growth Strategy2.3.6 Primary Interviews with Key Gene Therapy in Oncology Players (Opinion Leaders)

3 Competition Landscape by Key Players3.1 Global Top Gene Therapy in Oncology Players by Market Size3.1.1 Global Top Gene Therapy in Oncology Players by Revenue (2015-2020)3.1.2 Global Gene Therapy in Oncology Revenue Market Share by Players (2015-2020)3.1.3 Global Gene Therapy in Oncology Market Share by Company Type (Tier 1, Tier 2 and Tier 3)3.2 Global Gene Therapy in Oncology Market Concentration Ratio3.2.1 Global Gene Therapy in Oncology Market Concentration Ratio (CR5 and HHI)3.2.2 Global Top 10 and Top 5 Companies by Gene Therapy in Oncology Revenue in 20193.3 Gene Therapy in Oncology Key Players Head office and Area Served3.4 Key Players Gene Therapy in Oncology Product Solution and Service3.5 Date of Enter into Gene Therapy in Oncology Market3.6 Mergers & Acquisitions, Expansion Plans

4 Breakdown Data by Type (2015-2026)4.1 Global Gene Therapy in Oncology Historic Market Size by Type (2015-2020)4.2 Global Gene Therapy in Oncology Forecasted Market Size by Type (2021-2026)

5 Gene Therapy in Oncology Breakdown Data by Application (2015-2026)5.1 Global Gene Therapy in Oncology Market Size by Application (2015-2020)5.2 Global Gene Therapy in Oncology Forecasted Market Size by Application (2021-2026)

6 North America6.1 North America Gene Therapy in Oncology Market Size (2015-2020)6.2 Gene Therapy in Oncology Key Players in North America (2019-2020)6.3 North America Gene Therapy in Oncology Market Size by Type (2015-2020)6.4 North America Gene Therapy in Oncology Market Size by Application (2015-2020)

7 Europe7.1 Europe Gene Therapy in Oncology Market Size (2015-2020)7.2 Gene Therapy in Oncology Key Players in Europe (2019-2020)7.3 Europe Gene Therapy in Oncology Market Size by Type (2015-2020)7.4 Europe Gene Therapy in Oncology Market Size by Application (2015-2020)

8 China8.1 China Gene Therapy in Oncology Market Size (2015-2020)8.2 Gene Therapy in Oncology Key Players in China (2019-2020)8.3 China Gene Therapy in Oncology Market Size by Type (2015-2020)8.4 China Gene Therapy in Oncology Market Size by Application (2015-2020)

9 Japan9.1 Japan Gene Therapy in Oncology Market Size (2015-2020)9.2 Gene Therapy in Oncology Key Players in Japan (2019-2020)9.3 Japan Gene Therapy in Oncology Market Size by Type (2015-2020)9.4 Japan Gene Therapy in Oncology Market Size by Application (2015-2020)

10 Southeast Asia10.1 Southeast Asia Gene Therapy in Oncology Market Size (2015-2020)10.2 Gene Therapy in Oncology Key Players in Southeast Asia (2019-2020)10.3 Southeast Asia Gene Therapy in Oncology Market Size by Type (2015-2020)10.4 Southeast Asia Gene Therapy in Oncology Market Size by Application (2015-2020)

11 India11.1 India Gene Therapy in Oncology Market Size (2015-2020)11.2 Gene Therapy in Oncology Key Players in India (2019-2020)11.3 India Gene Therapy in Oncology Market Size by Type (2015-2020)11.4 India Gene Therapy in Oncology Market Size by Application (2015-2020)

12 Central & South America12.1 Central & South America Gene Therapy in Oncology Market Size (2015-2020)12.2 Gene Therapy in Oncology Key Players in Central & South America (2019-2020)12.3 Central & South America Gene Therapy in Oncology Market Size by Type (2015-2020)12.4 Central & South America Gene Therapy in Oncology Market Size by Application (2015-2020)

13 Key Players Profiles13.1 Bristol-Myers Squibb13.1.1 Bristol-Myers Squibb Company Details13.1.2 Bristol-Myers Squibb Business Overview and Its Total Revenue13.1.3 Bristol-Myers Squibb Gene Therapy in Oncology Introduction13.1.4 Bristol-Myers Squibb Revenue in Gene Therapy in Oncology Business (2015-2020))13.1.5 Bristol-Myers Squibb Recent Development13.2 Cold Genesys13.2.1 Cold Genesys Company Details13.2.2 Cold Genesys Business Overview and Its Total Revenue13.2.3 Cold Genesys Gene Therapy in Oncology Introduction13.2.4 Cold Genesys Revenue in Gene Therapy in Oncology Business (2015-2020)13.2.5 Cold Genesys Recent Development13.3 Advantagene13.3.1 Advantagene Company Details13.3.2 Advantagene Business Overview and Its Total Revenue13.3.3 Advantagene Gene Therapy in Oncology Introduction13.3.4 Advantagene Revenue in Gene Therapy in Oncology Business (2015-2020)13.3.5 Advantagene Recent Development13.4 Amgen13.4.1 Amgen Company Details13.4.2 Amgen Business Overview and Its Total Revenue13.4.3 Amgen Gene Therapy in Oncology Introduction13.4.4 Amgen Revenue in Gene Therapy in Oncology Business (2015-2020)13.4.5 Amgen Recent Development13.5 AstraZeneca13.5.1 AstraZeneca Company Details13.5.2 AstraZeneca Business Overview and Its Total Revenue13.5.3 AstraZeneca Gene Therapy in Oncology Introduction13.5.4 AstraZeneca Revenue in Gene Therapy in Oncology Business (2015-2020)13.5.5 AstraZeneca Recent Development13.6 Bio-Path Holdings13.6.1 Bio-Path Holdings Company Details13.6.2 Bio-Path Holdings Business Overview and Its Total Revenue13.6.3 Bio-Path Holdings Gene Therapy in Oncology Introduction13.6.4 Bio-Path Holdings Revenue in Gene Therapy in Oncology Business (2015-2020)13.6.5 Bio-Path Holdings Recent Development13.7 CRISPR Therapeutics13.7.1 CRISPR Therapeutics Company Details13.7.2 CRISPR Therapeutics Business Overview and Its Total Revenue13.7.3 CRISPR Therapeutics Gene Therapy in Oncology Introduction13.7.4 CRISPR Therapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)13.7.5 CRISPR Therapeutics Recent Development13.8 Editas Medicine13.8.1 Editas Medicine Company Details13.8.2 Editas Medicine Business Overview and Its Total Revenue13.8.3 Editas Medicine Gene Therapy in Oncology Introduction13.8.4 Editas Medicine Revenue in Gene Therapy in Oncology Business (2015-2020)13.8.5 Editas Medicine Recent Development13.9 Geron Corp13.9.1 Geron Corp Company Details13.9.2 Geron Corp Business Overview and Its Total Revenue13.9.3 Geron Corp Gene Therapy in Oncology Introduction13.9.4 Geron Corp Revenue in Gene Therapy in Oncology Business (2015-2020)13.9.5 Geron Corp Recent Development13.10 Idera Pharmaceuticals13.10.1 Idera Pharmaceuticals Company Details13.10.2 Idera Pharmaceuticals Business Overview and Its Total Revenue13.10.3 Idera Pharmaceuticals Gene Therapy in Oncology Introduction13.10.4 Idera Pharmaceuticals Revenue in Gene Therapy in Oncology Business (2015-2020)13.10.5 Idera Pharmaceuticals Recent Development13.11 Intellia Therapeutics10.11.1 Intellia Therapeutics Company Details10.11.2 Intellia Therapeutics Business Overview and Its Total Revenue10.11.3 Intellia Therapeutics Gene Therapy in Oncology Introduction10.11.4 Intellia Therapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)10.11.5 Intellia Therapeutics Recent Development13.12 Johnson & Johnson10.12.1 Johnson & Johnson Company Details10.12.2 Johnson & Johnson Business Overview and Its Total Revenue10.12.3 Johnson & Johnson Gene Therapy in Oncology Introduction10.12.4 Johnson & Johnson Revenue in Gene Therapy in Oncology Business (2015-2020)10.12.5 Johnson & Johnson Recent Development13.13 Marsala Biotech10.13.1 Marsala Biotech Company Details10.13.2 Marsala Biotech Business Overview and Its Total Revenue10.13.3 Marsala Biotech Gene Therapy in Oncology Introduction10.13.4 Marsala Biotech Revenue in Gene Therapy in Oncology Business (2015-2020)10.13.5 Marsala Biotech Recent Development13.14 Merck10.14.1 Merck Company Details10.14.2 Merck Business Overview and Its Total Revenue10.14.3 Merck Gene Therapy in Oncology Introduction10.14.4 Merck Revenue in Gene Therapy in Oncology Business (2015-2020)10.14.5 Merck Recent Development13.15 Mologen AG10.15.1 Mologen AG Company Details10.15.2 Mologen AG Business Overview and Its Total Revenue10.15.3 Mologen AG Gene Therapy in Oncology Introduction10.15.4 Mologen AG Revenue in Gene Therapy in Oncology Business (2015-2020)10.15.5 Mologen AG Recent Development13.16 Oncolytics Biotech10.16.1 Oncolytics Biotech Company Details10.16.2 Oncolytics Biotech Business Overview and Its Total Revenue10.16.3 Oncolytics Biotech Gene Therapy in Oncology Introduction10.16.4 Oncolytics Biotech Revenue in Gene Therapy in Oncology Business (2015-2020)10.16.5 Oncolytics Biotech Recent Development13.17 Oncosec10.17.1 Oncosec Company Details10.17.2 Oncosec Business Overview and Its Total Revenue10.17.3 Oncosec Gene Therapy in Oncology Introduction10.17.4 Oncosec Revenue in Gene Therapy in Oncology Business (2015-2020)10.17.5 Oncosec Recent Development13.18 Oncotelic10.18.1 Oncotelic Company Details10.18.2 Oncotelic Business Overview and Its Total Revenue10.18.3 Oncotelic Gene Therapy in Oncology Introduction10.18.4 Oncotelic Revenue in Gene Therapy in Oncology Business (2015-2020)10.18.5 Oncotelic Recent Development13.19 Shenzhen SiBiono GeneTech10.19.1 Shenzhen SiBiono GeneTech Company Details10.19.2 Shenzhen SiBiono GeneTech Business Overview and Its Total Revenue10.19.3 Shenzhen SiBiono GeneTech Gene Therapy in Oncology Introduction10.19.4 Shenzhen SiBiono GeneTech Revenue in Gene Therapy in Oncology Business (2015-2020)10.19.5 Shenzhen SiBiono GeneTech Recent Development13.20 Sillajen Biotherapeutics10.20.1 Sillajen Biotherapeutics Company Details10.20.2 Sillajen Biotherapeutics Business Overview and Its Total Revenue10.20.3 Sillajen Biotherapeutics Gene Therapy in Oncology Introduction10.20.4 Sillajen Biotherapeutics Revenue in Gene Therapy in Oncology Business (2015-2020)10.20.5 Sillajen Biotherapeutics Recent Development13.21 Tocagen10.21.1 Tocagen Company Details10.21.2 Tocagen Business Overview and Its Total Revenue10.21.3 Tocagen Gene Therapy in Oncology Introduction10.21.4 Tocagen Revenue in Gene Therapy in Oncology Business (2015-2020)10.21.5 Tocagen Recent Development13.22 UniQure10.22.1 UniQure Company Details10.22.2 UniQure Business Overview and Its Total Revenue10.22.3 UniQure Gene Therapy in Oncology Introduction10.22.4 UniQure Revenue in Gene Therapy in Oncology Business (2015-2020)10.22.5 UniQure Recent Development13.23 Ziopharm Oncology10.23.1 Ziopharm Oncology Company Details10.23.2 Ziopharm Oncology Business Overview and Its Total Revenue10.23.3 Ziopharm Oncology Gene Therapy in Oncology Introduction10.23.4 Ziopharm Oncology Revenue in Gene Therapy in Oncology Business (2015-2020)10.23.5 Ziopharm Oncology Recent Development

14 Analysts Viewpoints/Conclusions

15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Disclaimer15.3 Author Details

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Gene Therapy in Oncology Market New Research Study Report with Size, Share, Trends, Emerging Applications, Opportunities and Worldwide Analysis with...

Gene Therapy Market report would come handy to understand the competitors in the market and give an insight into sales, volumes, revenues in the Gene Therapy Industry & will also assists in making strategic decisions. The report also helps to decide corporate product & marketing strategies. It reduces the risks involved in making decisions as well as strategies for companies and individuals interested in the Gene Therapy industry. Both established and new players in Gene Therapy industries can use the report to understand the Gene Therapy market.

In Global Market, the Following Companies Are Covered:

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Analysis of the Market:

Gene therapy is an experimental treatment that involves introducing genetic material into a persons cells to fight or prevent disease. Researchers are studying gene therapy for a number of diseases, such as severe combined immuno-deficiencies, hemophilia, Parkinsons disease, cancer and even HIV, through a number of different approaches. A gene can be delivered to a cell using a carrier known as a vector. The most common types of vectors used in gene therapy are viruses. The viruses used in gene therapy are altered to make them safe, although some risks still exist with gene therapy. The technology is still in its infancy.

Gene therapy is a method that involves introducing genetic material into a persons cells to fight or prevent diseases. In recent decades of years, researchers are paying more and more attention on it. Until today, it is still in infancy. There are many bio-technology companies entered into this market and carry on related research and development.

These research companies are including Bluebird Bio, Sangamom, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical and Advantagene. Among them, the top 5 research companies occupied more than 85% market.

Generally, the research fees of companies are come from their collaboration partners. For example, Bayer Healthcare is the partner of Dimension Therapeutics. General, some products of these companies are in the period 3 of their pipeline.

In general, cancer diseases are still the largest application with market share over 65% in 2015. In addition, there are some companies are focus on rare diseases.

Market Analysis and Insights: Global Gene Therapy Market

In 2019, the global Gene Therapy market is valued at considerable rate by the end of 2026, growing at a steady rate of CAGR during 2021-2026.

Global Gene Therapy Scope and Market Size

Gene Therapy market is segmented by Type, and by Application. Players, stakeholders, and other participants in the global Gene Therapy market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2015-2026.

Segment by Type, the Gene Therapy market is segmented into Ex vivo, In Vivo, etc.

Segment by Application, the Gene Therapy market is segmented into Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other, etc.

Regional and Country-level Analysis

The Gene Therapy market is analysed and market size information is provided by regions (countries).

The key regions covered in the Gene Therapy market report are North America, Europe, China, Japan, Southeast Asia, India and Central & South America, etc.

The report includes country-wise and region-wise market size for the period 2015-2026. It also includes market size and forecast by Type, and by Application segment in terms of revenue for the period 2015-2026.

Competitive Landscape and Gene Therapy Market Share Analysis

Gene Therapy market competitive landscape provides details and data information by vendors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on revenue (global and regional level) by player for the period 2015-2020. Details included are company description, major business, company total revenue and the revenue generated in Gene Therapy business, the date to enter into the Gene Therapy market, Gene Therapy product introduction, recent developments, etc.

The major vendors include Bluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene, etc.

This report focuses on the global Gene Therapy status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Gene Therapy development in North America, Europe, China, Japan, Southeast Asia, India and Central & South America.

Gene Therapy Market Breakdown by Types:

s

Gene Therapy Market Breakdown by Application:

Critical highlights covered in the Global Gene Therapy market include:

The information available in the Gene Therapy Market report is segmented for proper understanding. The Table of contents contains Market outline, Market characteristics, Market segmentation analysis, Market sizing, customer landscape & Regional landscape. For further improving the understand ability various exhibits (Tabular Data & Pie Charts) has also been used in the Gene Therapy Market report.

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Reasons for Buy Gene Therapy Market Report:

In the end, Gene Therapy Industry report provides the main region, market conditions with the product price,profit, capacity, production, supply, demand and market growth rateand forecast etc. This report also Present newproject SWOT analysis,investment feasibility analysis, andinvestment return analysis.

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Gene Therapy Market 2020 to Flourish with an Impressive CAGR of XX% in the year 2026, Predicts Market Research Future According to Current Market...

TipRanks

Expectations of good news on the near horizon are buoying markets right now. Over the past month, both the S&P 500 and the NASDAQ are up 11% to new record highs.Investors are excited at the prospect of a COVID vaccine coming before the winter is out. And the electoral results, that Democrat Joe Biden will ascend to the Presidency while the Republicans will emerge strengthened in Congress, promise the avoidance of extremes typical of divided government. In short, investors are looking forward to return to normal environment over the next several months. And that has them seeking stocks that are primed for gains. Against this backdrop, Goldman Sachs analysts are pounding the table on three stocks in particular, noting that each could surge over 40% in the year ahead. After running both tickers through TipRanks database, we found out that the rest of the Street is also standing squarely in the bull camp.Codiack BioSciences (CDAK)As we have all learned from coronavirus pandemic, some new thing in medical science can make huge impact on our world. Codiack aims to turn that principle to good. This research-oriented pharmaceutical aims to turn exosome therapeutics into a whole new class of medicines. Exosomes are the degradation mechanism RNA, and can transfer genetic material around a body.And therein lies the potential. Codiack has developed a design platform for the engineering of exosome proteins capable of carrying and protecting drug molecules through cell walls. In effect, the proteins will mimic the pathways used by viruses but are non-viral, and are designed to carry a payload of therapeutic agents. If successful, exosome therapy offers doctors the ability to design a drug that will deliver specific agents to specific cells to fight specific disease.Codiack is involved in all aspects of exosome therapeutics, from design to manufacturing, and currently has an active pipeline of agents seven, in all in various stages of discovery, preclinical testing, and the beginnings of Phase 1 trials.In the biosciences, success or failure is all about that pipeline, and in its diverse, active pipeline of agents in a new sector of biotechnological pharmaceuticals, Codiack has a fine resource to attract investors. To get those investors, the company went public this past October, selling 5.5 million shares at an opening price of $14.10 per share.Among the healthcare name's fans is Goldman Sachs analyst Graig Suvannavejh. The analyst wrote, Biopharma industry interest in exosomes has long been high, but engineering them for a specific function and manufacturing at scale have both proven challenging. Among a field of multiple competitors, CDAK has made the most significant progress on both fronts, and as such we view their technology platform as best-in-class."Given share underperformance (-37%) since the IPO, we find risk/reward highly compelling at current levels, and with key 2021 data sets to provide potential de-risking and positive share inflection," the analyst concluded.Suvannavejh rates CDAK a Buy, and his $29 price target shows the extent of his confidence it implies a 222% upside for the coming year. (To watch Suvannavejhs track record, click here)Overall, Codiack has a Strong Buy from the analyst consensus 3 reviewers have put up Buy ratings in recent weeks. The stock is selling for $8.90, and its $24 average price target implies a 166% one-year upside potential. (See CDAK stock analysis on TipRanks)Arcutis Biotherapeutics (ARQT)Acrutis is a pioneering researcher in the treatment of dermatological disease. Arcutis is involved in discovering the next generation of dermatological treatments an important niche, especially when one realizes that one common ailment, psoriasis, has not seen an FDA approval for a novel treatment in over two decades.The company is leveraging recent advances in immunology and inflammation to find new approaches to skin treatment. The goal is to make it easier for patients and doctors together to manage conditions like psoriasis, alopecia, atopic dermatitis, seborrheic dermatitis, and vitiligo, to name just a few.The company's lead candidate, ARQ-151 (roflumilast cream), is about to enter a phase 3 trial for atopic dermatitis, and is in an advanced phase 3 stage in Plaque Psoriasis. Arcutis has recently issued an update on positive data from the Phase 2 trials of ARQ-151 in atopic dermatitis. The drug is a once-daily treatment, and has demonstrated significant patient relief from symptoms, especially itching and itching-related sleep problems. This is another stock in Suvannavejhs coverage universe. The Goldman analyst is impressed by developments in the companys pipeline work, noting: ARQT provided an update on the outcome of its end-of-Phase 2 meetings with the FDA, following their Phase 2a trial of ARQ-151 in atopic dermatitis (AtD). Feedback from regulators was broadly encouraging, in particular, acknowledging the robust long-term safety data being generated by ARQT for ARQ-151 in plaque psoriasisAccordingly, Suvannavejh rates ARQT a Buy, and sets a $36 price target that indicates room for 40% upside growth in 2021. (To watch Suvannavejhs track record, click here)Arcutis has 2 recent Buy reviews, making the consensus rating a Moderate Buy. The stocks average price target is $37, suggesting a 44% upside from current levels. (See ARQT stock analysis on TipRanks)Oak Street Health (OSH)With the last stock, we move from medical research to medical care. Specifically, Oak Street Health is a primary care clinic operator, and part of the Medicare Network. The company has operations and clinics in Illinois, Indiana, Michigan, Pennsylvania, and Ohio, along with New York, North Carolina, Rhode Island, Tennessee, and Texas. It has been in operation for eight years, and went public this past summer, holding the IPO in August.In the third quarter, the companys first as a publicly traded entity, OSH brought in $217.9 million in revenue. The revenue number was up 56% from the year-ago quarter. Earnings per share matched expectations, at 15 cents.The companys expansion proceeds apace, and in October, Oak Street entered New York by opening, in Brooklyn, its 70th location. A planned expansion in Texas, involving a partnership with Walmart, is also proceeding as planned, and Oak Street has opened its first Walmart Community Clinic the Dallas-Fort Worth area city of Carrollton.Robert Jones, covering this stock for Goldman, set a $74 price target to back his Buy rating. At currently levels, this target implies an upside of ~58% in the next 12 months. (To watch Jones track record, click here)Results suggest operations are still on track, with few incremental updates since the 2Q call, where management noted a resumption of center openings, (pivoted) marketing efforts, and in-person visits despite COVID. In 3Q, OSH opened 13 new centers and is on track for 73-75 by end of year The company maintained that it is continuing to operate at a high level in places with elevated COVID case counts like Chicago and Detroit, Jones noted.All in all, the Strong Buy analyst consensus rating OSH is based on 8 reviews, breaking down to 7 Buys and just a single Hold. The stock is selling for $46.94, and its $61.29 average price target suggests it has a ~31% upside for the coming year. (See OSH stock analysis on TipRanks)To find good ideas for healthcare stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Global Contract Cell and Gene Therapy Manufacturing Market Report 2020-2026: CDMO Categorization - Primed for Business Model Disruption - Yahoo...

The Most Recent study on the Hemophilia Gene Therapy Market Research provides a profound comprehension of the various market dynamics like trends, drivers, the challenges, and opportunities. The report further elaborates on the micro and macro-economic elements that are predicted to shape the increase of the Hemophilia Gene Therapy market throughout the forecast period (2019-2029).

The introduced study elucidates the crucial indicators of Market growth which comes with a thorough analysis of this value chain, CAGR development, and Porters Five Forces Analysis. This data may enable readers to understand the quantitative growth parameters of this international industry that is Hemophilia Gene Therapy .

Analytical Insights Included from the Hemophilia Gene Therapy Market Report

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Hemophilia Gene Therapy Market Segmentation Assessment

The increase prospects of this market in various Regions are studied in the report together with details like the regulatory framework, political, and financial outlook of each region.

Competitive landscape

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The COVID-19 pandemic has changed narratives related to growth and expansion across several key industries. Therefore, theHemophilia Gene Therapy market is also battling the cons of supply chain disruptions and procurement issues. Over the course of the next quarter, market players could be investing in new technologies to recover from the shocks of the pandemic.

The report provides a comprehensive account of the following key points:

The report also answers some of the burning questions pertaining to market expansion:

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The Report intends to eliminate the subsequent doubts regarding the Hemophilia Gene Therapy market:

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Hemophilia Gene Therapy Market to Develop Rapidly by 2018 to 2028 - The Haitian-Caribbean News Network