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Archive for the ‘Gene Therapy Research’ Category

Gene therapy restores immune function in children with rare immunodeficiency – National Institutes of Health

News Release

Tuesday, May 11, 2021

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The findings were published today inthe New England Journal of Medicine.

ADA-SCID, which is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide, is caused by mutations in theADAgene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function. This impairment leaves children with the condition highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first two years of life.

These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA-SCID, said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life.

People with ADA-SCID can be treated with enzyme replacement therapy, but this treatment does not fully reconstitute immune function and must be taken for life, usually once or twice weekly. Transplants of blood-forming stem cells, ideally from a genetically matched sibling donor, can provide a more lasting solution. However, most people lack such a donor. Additionally, stem cell transplants carry risks such asgraft-versus-host disease and side effects from chemotherapy medications given to help the donor stem cells establish themselves in the patients bone marrow.

The new research evaluated an experimental lentiviral gene therapy designed to be safer and more effective than previously tested gene-therapy strategies for ADA-SCID. This gene therapy involves inserting a normal copy of theADAgene into the patients own blood-forming stem cells. First, stem cells are collected from the patients bone marrow or peripheral blood. Next, a harmless virus is used as a vector, or carrier, to deliver the normalADAgene to these cells in the laboratory. The genetically corrected stem cells then are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the cells establish themselves in the bone marrow and begin producing new immune cells.

The experimental gene therapy, developed by researchers from the University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London, uses a modified lentivirus to deliver the ADA gene to cells. Previous gene-therapy approaches for ADA-SCID have relied on a different type of virus called a gamma retrovirus. Some people who have received gamma retroviral gene therapies have later developed leukemia, which scientists suspect is due to the vector causing activation of genes that control cell growth.The lentiviral vector is designed to avoid this outcome and to enhance the effectiveness of gene delivery into cells.

The results come from three separate Phase 1/2 clinical trials, two conducted in the United States and one in the United Kingdom. The U.S. trials, led by principal investigator Donald Kohn, M.D., of UCLA, enrolled 30 participants with ADA-SCID ranging in age from 4 months to 4 years at UCLA Mattel Childrens Hospital and the NIH Clinical Center in Bethesda, Maryland. The U.K. study, conducted at GOSH and led by principal investigator Claire Booth, M.B.B.S., Ph.D., enrolled 20 participants ranging in age from 4 months to 16 years. Most participants acquired and retained robust immune function following gene therapy 96.7% after two years in the U.S. studies and 95% after three years in the U.K. study and were able to stop enzyme replacement therapy and other medications. Of the two participants for whom gene therapy did not restore lasting immune function, one restarted enzyme replacement therapy and later received a successful stem cell transplant from a donor, and the other restarted enzyme replacement therapy. The lentiviral gene therapy appeared safe overall, although all participants experienced some side effects. Most of these were mild or moderate and attributable to the chemotherapy that the participants received.

Researchers observed similar outcomes in all three trials, although there were some differences between the studies. Stem cells were collected from bone marrow in the U.S. trials and from peripheral blood in the U.K. trial. In one of the U.S. trials, 10 children were treated with genetically corrected stem cells that had been frozen and later thawed. The two other trials used fresh stem cell preparations. In the future, the freezing procedure known as cryopreservation may allow stem cells to be more easily transported and processed at a manufacturing facility far from the patients home and shipped back to a local hospital, reducing the need for patients to travel long distances to specialized medical centers to receive gene therapy. A trial of the cryopreserved treatment is now underway at the Zayed Centre for Research into Rare Diseases in Children in London, in partnership with GOSH.

For more information about the trials described in the New England Journal of Medicine paper, visit ClinicalTrials.gov under identifiers NCT01852071, NCT02999984 and NCT01380990. The investigational lentiviral gene therapy, which is licensed to Orchard Therapeutics, has not been approved for use by any regulatory authority.

The research was funded in part by three NIH Institutes: NIAID; the National Heart, Lung and Blood Institute; and the National Human Genome Research Institute. Additional funding was provided by the California Institute for Regenerative Medicine, the Medical Research Council, the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children National Health Service Foundation Trust and University College London, and Orchard Therapeutics.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

DB Kohn, C Boothet al. Autologousex vivolentiviral gene therapy for adenosine deaminase deficiency.New England Journal of MedicineDOI: 10.1056/NEJMoa2027675 (2021).

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Gene therapy restores immune function in children with rare immunodeficiency - National Institutes of Health

$2.3M boost puts Hawaii gene therapy research on the map | University of Hawaii System News – UH System Current News

Jesse Owens

Making waves in the fight against disease at the University of Hawaii is the Institute for Biogenesis Researchs (IBR) youngest full-time faculty member, 36-year-old Jesse Owens. Owens, who also earned his PhD in cell and molecular biology from UH Mnoa, was awarded $2.3 million from the National Institutes of Health (NIH) over the next five years to develop a new, safer and more efficient approach to gene therapy. He leads a team of collaborators from UH Mnoas John A. Burns School of Medicine (JABSOM), MIT and UC Davis.

The $2.3 million grant is significant because it was a first-time submission and received a score in the 1st percentile, higher than 99% of grants submitted from institutions across all 50 states in the U.S.

Directed evolution of a sequence-specific targeting technology for therapeutic gene delivery to the human genome, Owens grant addresses drawbacks to current genome editing technologies that randomly insert a therapeutic gene, which can disrupt important host genes and potentially cause cancer. Methods like CRISPR (clustered regularly interspaced short palindromic repeats), a family of DNA sequences in genomes of organisms, are inefficient in non-dividing cells and delivering large DNA cargos. However, the technology that Owens is developing can direct large pieces of DNA to a safe location in the genome that can be used in all body tissues, both dividing and non-dividing, which will allow us to cure more types of diseases.

This is an R01, which is the coveted personal grant that people get to do researchits bringing absolutely state-of-the-art technology that hes inventing to do new gene therapy in Hawaii and this is why were very proud of him, said IBR Director Steven Ward.

I think the schools here in Hawaii are underestimated Owens

Not only is he faculty here, he got his PhD here, he was trained here, he was raised on the Big Island, added Ward. Hes the product of that and it just shows you that Hawaii can do some of the worlds greatest biomedical research.

In addition to the NIH funding, Owens recently signed a sponsored-research agreement with a private company specializing in non-viral gene therapies called SalioGen Therapeutics. The goal of this collaboration is to advance the tools he is developing in the lab into clinical-stage gene therapy candidates.

Born on Hawaii Island, Owens shares his Hilo High School alma mater with renowned Nobel Prize winner Jennifer Doudna, who developed CRISPR gene therapy. He hopes to inspire other children from Hawaii to pursue a career in science.

I think the schools here in Hawaii are underestimated really, but you can succeed coming out of Hawaii, for sure, Owens said.

Read more on the JABSOM website.

This research is an example of UH Mnoas goal of Excellence in Research: Advancing the Research and Creative Work Enterprise (PDF), one of four goals identified in the 201525 Strategic Plan (PDF), updated in December 2020.

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$2.3M boost puts Hawaii gene therapy research on the map | University of Hawaii System News - UH System Current News

How a genetic ‘CopyCatcher’ could open the door to CRISPR-based precision gene therapy – FierceBiotech

Gene therapies such as Novartis spinal muscular atrophy treatment Zolgensma deliver a surrogate copy of a gene to replace a dysfunctional one in the body. The CRISPR gene editing tool could offer an alternative strategyone that could allow for a mutated gene to be fixed on site. But scientists arent sure whether such a technique, known as gene drive, would work at scale.

Researchers at the University of California, San Diego (UCSD) say they may have found a way to make CRISPR efficient in gene therapy. They have developed a technology called CopyCatcher, which candetect and quantify events in which a genetic element is copied precisely from one chromosome to another during CRISPR-based gene editing.

In fruit flies, CopyCatcher revealed unexpectedly high rates of gene conversion, according to results published in Nature Communications. With help of the new tool and DNA screening, the team also identified the c-MYC gene as an inhibitor of genetic copying in human embryonic cells. The researchers said the findings lay the groundwork for developing CRISPR-based gene therapy for humans.

The promise of CRISPR-based gene drives is that one chromosome bearing the drive can cutthe other chromosome, which uses the drive as a template torepair the damage. UCSD used CopyCatcher to measure the efficiency of such a system in living animals.

CopyCatcher carries a highly sensitive detectorgene that produces fluorescent proteins only if the target genetic element copies itself onto a sister chromosome, allowing investigators to detect and quantify gene conversion.

In fruit flies, the frequency of gene conversion was unexpectedly high, at 30% to 50% of cells in the targeted tissues of the eye and thorax, the team reported.

RELATED: MIT and UCSF researchers create CRISPR 'on-off switch' that controls gene expression without changing DNA

But the rates of chromosome copying dropped sharply to just 4% to 8% of cells in human cells. That may be because mammalian chromosomes dont typically engage in chromosome pairing but rely instead on a different mechanism to fix DNA cuts. The researchers showed they could use certain techniques to improve copying, suggesting that human cells might be induced to perform efficient gene conversion.

By using a genetic screen, the team identified several factors affecting how DNA selects a repair pathway. The gene c-MYC emerged as a prime inhibitor of templated gene conversion. Cutting the expression of c-MYC by half increased the production of fluorescence marker expression by 2.5-fold in human embryonic cells compared with control cells, the team showed.

If high-efficiency gene editing could be achieved in human cells, CRISPR-based gene therapies could be developed to treat a variety of genetic disorders including blood diseases, hearing loss, spinal muscular atrophy, congenital heart defects and others, the researchers suggested.

These studies provide a clear proof of principle for a new type of gene therapy in which one copy of a mutated gene could be repaired from a partially intact second copy of the gene, Ethan Bier, Ph.D., the studys senior author, said in a statement. The need for such a design occurs in genetic situations with patients with inherited genetic disorders, if their parents were carriers for two different mutations in the same gene.

For future studies, the researchers plan to use CopyCatcher to identify additional factors that can be manipulated to push the choice of DNA repair toward the chromosome-pairing mechanism. That could improve the efficiency of CRISPR-based editing, they believe.

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How a genetic 'CopyCatcher' could open the door to CRISPR-based precision gene therapy - FierceBiotech

Biogen looks to build better gene therapies through latest deal – BioPharma Dive

Dive Brief:

Biogen's big move into gene therapy came just over two years ago with the $800 million acquisition of Nightstar Therapeutics, a developer of eye medicines.

Since then, the company has inked an agreement with Sangamo Therapeutics to explore gene editing as a way to treat neurological diseases, pushed further into gene therapies for the eye through a deal with Germany's ViGeneron, and just recently announced plans to spend $200 million on a gene therapy manufacturing facility in North Carolina.

These investments have come both as Biogen's peers pour money into gene therapy, and as the company faces investor pressure to diversify its business.

Gene therapy research has made a resurgence, in large part, because the technology and how it interacts with the body are now better understood. The Food and Drug Administration has approved a handful of cell and gene therapies over the last few years, and expects to be reviewing and approving between 10 and 20 annually by 2025.

Yet, there are still major challenges in this cutting-edge field, with one of the biggest being delivery. To be effective, gene therapies must achieve the complicated task of carrying their payloads to the appropriate cells without getting destroyed by the body's defense mechanisms. That task can be especially difficult for therapies going after highly protected areas, such as the brain.

Companies that specialize in gene therapy delivery have therefore been in high demand. Over the last year, for example, a startup called Dyno Therapeutics has caught the attention of heavyweight gene therapy developers like Novartis, Roche and Sarepta Therapeutics.

Dyno's capsid technology is meant to create more targeted gene therapies that are less likely to trigger an immune response. Just last week, the company said it had raised an additional $100 million in fresh funding from tech investor Andreessen Horowitz and several other venture firms.

Also recently, the gene editing startup Beam Therapeutics agreed to buy Guide Therapeutics, which makes tools to deliver genetic medicines into cells, in a deal valued at $120 million.

Capsigen now adds to this flurry of activity with its Biogen collaboration. Per deal terms, Biogen will hold an exclusive license to Capsigen's technology for an undisclosed number of CNS and neuromuscular disease targets.

"One of our priorities for technology innovation is the discovery of AAV capsids with improved delivery profiles," Al Sandrock, Biogen's head of research and development, said in a May 11 statement. "We are investing for the long-term by building platform capabilities and advanced manufacturing technologies with the goal of accelerating our efforts in gene therapy."

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Biogen looks to build better gene therapies through latest deal - BioPharma Dive

Cell And Gene Therapy Clinical Trials Market in 2021 | Expansive Coverage on the Latest Developments in the Market – BioSpace

Cell and gene therapies rapid penetration in clinical trials globally is testimony to the incredible potential these in understanding, treating, and curing diseases. The cell and gene therapy clinical trials market is rapidly evolving, touching numerous frontiers in personalized medicine, especially for chronic diseases. A number of gene therapies approved by the U.S. FDA reinforces the potential. Pharmaceutical companies in clinical trials that test cell and gene therapies have bloomed strikingly, most notably in oncology, eye diseases, and rare hereditary diseases. A partial list of the top diseases that attract massive attention of contract research organizations in cell and gene therapy market are type 1 diabetes, Parkinsons disease, spinal cord injuries, amyotrophic lateral sclerosis, the Alzheimers disease, and osteoarthritis.

The number of cell and gene therapies is seeing marked increase year over year. According to an estimate, there were more than a thousand cell and gene therapy clinical trials by 2019. To complement the trend, investments by pharma companies are also rising by large bounds in those years.

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The study on the cell and gene therapy clinical trials offers deep expounding of current and emerging business strategies, elements of competition, facets of markets attractiveness, and insights into regional growth dynamics across the globe.

Cell And Gene Therapy Clinical Trials Market: Key Trends

Clinical trials pertaining to advanced therapy medicinal product (ATMP) are making consistent increase in some developed nations. A predominant percentage of these in recent years have been viral vector mediated gene therapies. Thus far, some remarkable strides have been witnessed in this direction, enriching the investment scope in the cell and gene therapy market. The effect has been notices in all phases, from Phase I to Phase IV.

A prevalent trend over the past few years is the focus on oncology. Oncology--notably including haematological malignancies and solid tumourshave been at the center of ATMP clinical trials. Metabolic disease trials have also seen a significant increase, cementing revenue growth in the cell and gene therapy market. Advances made in gene therapy trials continue to pave way to new vistas for oncology research, both in vivo and in vitro.

Cell And Gene Therapy Clinical Trials Market: Competitive Dynamics and Key Developments

The profound potential of cell and gene therapies (CGT) notwithstanding, their successful clinical translation is, no doubt, rests on panoply of problems. These also determine the key restraints for the evolution of the cell and gene therapy market. The high degree of personalization that CGT entails, factors affecting their efficacy and safety are difficult to ascertain, if not impossible. For one, obtaining cells from donors is replete with some unique challenges, such as invasiveness of the process to patients. So are the lack of availability of cutting-edge biomarkers and targets anchored on which gene therapies will show their potential. The whole process of delivering CGT in clinical trials is itself associated with some tall challenges for contract research organizations.

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Having put these perspectives, the prospects have limitless potential waiting to be extracted, and researchers are not disheartened by the aforementioned challenges. In oncology alone, a number of new approaches have added liveliness to CGT clinical trials. Biotech companies are testing new waters in allogeneic therapies. T-cell receptor (TCR) are increasingly penetrating safety and feasibility trials, adding momentum to the cell and gene therapy market.

Some of the industry players likely to invade the space of these limitless possibilities are;

Cell And Gene Therapy Clinical Trials Market: Regional Assessment

North America has been at the cynosure of attention for CGT trials. European nations have also been showing substantial potential for generating revenues in the global market. In coming years, Asia Pacific is expected to show high growth potential

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Cell And Gene Therapy Clinical Trials Market in 2021 | Expansive Coverage on the Latest Developments in the Market - BioSpace

Sio Gene Therapies Announces CSF Reductions in GM1 – GlobeNewswire

- GM1 ganglioside in CSF reduced in 4 out of 5 children treated with the lowest dose at 6 months follow-up- Direct evidence of biochemical effect in the CNS suggests intravenous gene therapy may address both the systemic and neurological manifestations of GM1 gangliosidosis- Dr. Cynthia Tifft to deliver oral presentation at the 24th Annual ASGCT conference today at 6:15 PM EDT- On-track to report safety and efficacy data at 12 months follow-up in 2H 2021

NEW YORK and RESEARCH TRIANGLE PARK, N.C., May 13, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, will present new biomarker data from the Companys study of AXO-AAV-GM1, its adeno-associated viral vector (AAV)9-based gene therapy candidate for the treatment of GM1 gangliosidosis, at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), today, May 13, 2021 at 6:15 PM EDT.

Dr. Cynthia Tifft, Deputy Clinical Director of the National Human Genome Research Institute (NHGRI) and Principal Investigator in the study, will review patient-level data on safety and efficacy at six-months follow-up from the low-dose cohort of the Companys ongoing Phase 1/2 clinical study, with a focus on new six-month biomarker data from cerebrospinal fluid (CSF).

Previously reported data from the clinical study demonstrated that AXO-AAV-GM1 was well-tolerated with a favorable safety profile and provided early indications of clinical disease stability. In the low-dose cohort, five patients with late-infantile and juvenile GM1 gangliosidosis received a 1.5x1013 vg/kg intravenous (IV) infusion of AXO-AAV-GM1. At 6 months following gene transfer, serum beta-galactosidase enzyme activity approximately doubled and was restored to 23-57% of the lower limit of the normal reference range in the low-dose cohort. All five children demonstrated signs of clinical stabilization as assessed by measures of development including the Vineland-3 Adaptive Behavior, Upright and Floor Mobility, and Clinical Global Impression scales. To date, there have been no serious adverse events attributed to gene therapy in any patients receiving either the low-dose or the high-dose of AXO-AAV-GM1.

AAV9 is one of the best-studied vector systems currently in development. These new 6-month CSF biomarker data are an important update to the growing body of evidence for AXO-AAV-GM1 where we now provide direct evidence of biodistribution and biochemical effect in the CNS at the lowest dose, similar to what we saw in prior translational studies with naturally occurring animal models, said Gavin Corcoran, M.D., Chief R&D Officer of Sio Gene Therapies. Intravenous administration is likely to impact the disease in the periphery, where the disease burden is substantial. Todays CSF data, indicating a biomarker response in the CNS, provides the first indication that intravenous administration of AXO-AAV-GM1 may be able to treat both the systemic and neurological manifestations of this progressive, multisystem disease. We are proud to continue to lead the way in the development of a potentially transformative treatment for patients and families affected by GM1 gangliosidosis and look forward to the upcoming 12-month data readout later this year to provide further evidence regarding the durability of our AAV9 gene therapy and its potential to slow or halt the progression of GM1 gangliosidosis.

Key findings from the new biomarker analysis:

Dr. Tifft said, These data are highly encouraging and underscore the potential of Sios gene therapy program for GM1 gangliosidosis. I have been involved in the clinical and research aspects of this disease for more than 10 years and have seen first-hand a disease that only gets worse, so I am proud to share this clinical update for a trial where we have seen kids remain stable and most children have shown some overall improvement.

Upcoming Milestones

The clinical study (NCT03952637) is designed to evaluate the safety, tolerability, and potential efficacy of AXO-AAV-GM1 delivered intravenously in children with Type I and Type II GM1 gangliosidosis. Stage 1 is a dose-escalation study in which the low-dose cohort is evaluating 1.5x1013 vg/kg and the high-dose cohort is evaluating a dose of 4.5x1013 vg/kg in early infantile, late infantile, and juvenile children.

GM1 gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene that cause impaired production of the -galactosidase enzyme. Currently, there are no FDA-approved treatment options for GM1 gangliosidosis.

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFood and Drug Administrationand is the only gene therapy in clinical development for all pediatric forms of GM1 gangliosidosis.

In 2018, Sio licensed exclusive worldwide rights from theUniversity of Massachusetts Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

AboutSio Gene TherapiesSio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visitwww.siogtx.com.

Forward-Looking Statements

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as believe, "estimate, " may be, and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing of its upcoming clinical milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for Axo-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on February 9, 2021, as updated by its subsequent filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. MeswaniSio Gene Therapies Inc.Chief Commercial Officerinvestors@siogtx.com

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Sio Gene Therapies Announces CSF Reductions in GM1 - GlobeNewswire

Huntington’s disease setback for Roche shifts the research spotlight to Prilenia – S&P Global

Huntington's disease research suffered a double whammy after Swiss pharmaceutical giant Roche Holding AG halted late-stage trials of an experimental medicine and Wave Life Sciences Ltd. abandoned two drug studies for futility.

Roche stopped dosing patients with tominersen after an independent data monitoring committee questioned whether the drug's benefits outweighed its risks.

"Obviously, it's a very sad situation when we have to stop a pivotal program in a disease like Huntington's where there's such a high unmet need and such a terrible impact on these patients and their families," Bill Anderson, head of Roche's pharmaceuticals business, said on a call with reporters following Roche's first-quarter results. "It's a major setback for the field. But we will do everything we can to learn from the work that's been done. And if there's a way forward, we would certainly pursue it."

The research stumbles, while not uncommon in the notoriously complex world of central nervous system drug development, leave only a single experimental compound in the last stage of trials usually required to seek marketing approval: pridopidine from Naarden, Netherlands-based Prilenia Therapeutics BV.

First developed at Israel's Teva Pharmaceutical Industries Ltd., where Prilenia CEO Michael Hayden was global head of R&D and chief scientific officer until 2017, pridopidine is being studied in phase 3 trials that will probably read out by the third quarter of 2022. That makes it the most advanced experimental compound in development for the hereditary disease, which affects about 70,000 people in the U.S. and Europe, the biggest potential markets for treatments. No medicines have been approved to either delay onset or slow the disease's progression.

Roche is following up with the patients on the trials of tominersen to thoroughly assess the effect of therapy. The Basel, Switzerland-based pharma company intends to continue research into Huntington's in its early-stage neurology research groups, as well as possibly via gene therapy, Anderson said.

Regulators have expressed more support for Huntington's drug developers after the influence and investment brought to bear by Roche, Europe's largest pharmaceutical company by revenue.

"They know all about Huntington's disease," Prilenia's Hayden said in an interview with S&P Global Market Intelligence. "I'm very grateful to Roche in particular, that they brought their muscle and their weight and their resources to raising awareness [of the disease] all over the world."

A number of early studies are still ongoing, including one by Roche's crosstown rival Novartis AG, which is investigating its experimental spinal muscular atrophy treatment, branaplam, as a treatment for Huntington's. The once-a-week oral therapy is in phase 1 trials after securing orphan-drug designation from the U.S. Food and Drug Administration, with the first regulatory submissions planned for 2025.

Gene therapy?

Other early-stage approaches to treating the neurodegenerative disease include one-time gene therapies in trials with companies including uniQure NV and Voyager Therapeutics Inc., which had a clinical hold lifted by the FDA on April 26.

But gene therapy may not be the approach to take, Roche's Anderson said. Huntington's disease is caused by a single mistake in a particular gene that triggers the formation of a toxic protein that kills nerve cells. Gene therapy introduces genetic material into cells to make up for abnormal genes or to produce a necessary protein. The therapy also needs to be distributed throughout the brain, and that requires intracranial surgery, injections into the brain or both.

"There are different types of genetic disorders," Anderson said. "Some of them, you have a case where you're missing a protein. And it's a relatively easier thing to add a protein when you're missing a protein with gene therapy, than if you have a genetic defect that is causing a toxic protein to form. In which case, you have to knock that down. That's a harder task for gene therapy. It doesn't mean it's not possible. It's just a trickier thing to do."

Anderson said he was not referring to any research at Spark Therapeutics, the gene therapy company that Roche acquired in 2019.

Hayden, who is also a professor of medical genetics at the University of British Columbia, hopes to show the safety and tolerability already seen in more than 1,300 people to date. The phase 3 trial of the oral compound known as a sigma-1 receptor agonist is focused on the early Huntington's patient population, with a regulatory accepted endpoint of total functional capacity.

"We've learned deeply from the past. And we think we have it at least as close to right as we can now," Hayden said. "We believe that the study is quite de-risked, because we're not trying to look for new findings."

Pridopidine, which has patent protection until 2038, is easy to produce and available in tablet form. Hayden is keen to make the therapy readily available, should it gain approval, noting that the highest prevalence of the disease in the world was discovered in a remote tribe in Venezuela. Hayden also witnessed Huntington's impact at the start of his medical career in South Africa.

"I travelled the country and recognized that Huntington's disease was alive and well in Africa. These were [many] people who were disenfranchised by apartheid and disenfranchised by this disease," Hayden said.

While not wanting to raise unrealistic hopes, both Hayden and Anderson signaled that Huntington's researchers are not undeterred by recent setbacks.

"There are lots of things that we're doing these days that we didn't think we can do a few years ago," Anderson said. "And so we remain hopeful that we will find a solution."

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Huntington's disease setback for Roche shifts the research spotlight to Prilenia - S&P Global

Taysha Gene Therapies Expands Leadership Team with the Appointment of Chief Development Officer – BioSpace

Mary Newman, former SVP of Regulatory Affairs at Astellas Gene Therapies, joins Taysha with over 30 years of experience in regulatory affairs and research and development in the biotech industry

Extensive experience deeply rooted in gene therapy at Astellas Gene Therapies, Audentes Therapeutics and BioMarin

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer. Ms. Newman will oversee program and portfolio management, as well as translational sciences, and will report to Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Tayshas Chief Medical Officer and Head of Research and Development.

We are pleased to welcome Mary to the Taysha leadership team, said RA Session II, President, Founder and CEO of Taysha. In the past few months, we have significantly expanded our extremely talented R&D team, and Mary is an important addition to support the achievement of our ambitious pipeline advancement goals. Her decades of experience in all key aspects of drug development, particularly in gene therapy and rare disease, will be invaluable as we continue to grow our business.

Ms. Newman joins Taysha with more than 30 years of experience in regulatory affairs and research and development within the biotechnology industry, focusing on rare diseases. Most recently, she served as Senior Vice President of Regulatory Affairs at Astellas Gene Therapies (formerly Audentes Therapeutics), where she oversaw global regulatory strategic development, all primary regulatory agency interactions, and regulatory compliance for Audentes development candidates. Prior to joining Audentes, Ms. Newman served as the Senior Vice President, Regulatory Affairs and Quality Assurance at SARcode Bioscience Inc., and was responsible for the development of Xiidra for the treatment of dry eye disease. The company was acquired by Shire Ltd. She previously held various management positions, with increasing responsibility, in Regulatory Affairs at BioMarin Pharmaceutical, Inc., Berlex Inc. (now Bayer HealthCare Pharmaceuticals Inc.), and Sequus Pharmaceuticals, Inc. (now Johnson & Johnson). While at BioMarin, Ms. Newman oversaw the development and approval of Kuvan for the treatment of phenylketonuria (PKU), Naglazyme for mucopolysaccharidosis (MPS) VI, and supported the final approval of Aldurazyme for MPS I. She has also held various research and development leadership roles in oncology, neurology, and antifungal therapeutic areas. Ms. Newman serves as a member of the board of directors of Vedere Bio and is an advisor to the board of directors of Chameleon Biosciences. Ms. Newman holds a B.S. in Physiology from Oregon State University.

I am excited to join Taysha at such a transformative time in the companys corporate lifecycle, said Ms. Newman. Taysha is well-positioned to be a leader in developing disease-modifying gene therapies for patients with monogenic CNS diseases. I look forward to contributing to the further progression of the companys robust pipeline of promising drug candidates.

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.

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Taysha Gene Therapies Expands Leadership Team with the Appointment of Chief Development Officer - BioSpace

Gene and Cell Therapy Breakthroughs Focus of World Medical – GlobeNewswire

Boston, MA, May 13, 2021 (GLOBE NEWSWIRE) -- Gene and cell therapy (GCT), a transformative approach to develop treatments, and, potentially, cures for congenital and other diseases, is the focus of this years World Medical Innovation Forum May 19-21. Widely recognized as a transformational opportunity in medicine, GCT has the potential to stop or slow the effects of disease by targeting them at the genetic level. When the genetic driver for a disease is known, patients can be molecularly matched to therapies.

Featuring a unique combination of nearly 200 speakers and panelists from Mass General Brighams Harvard-affiliated faculty, industry, venture, manufacturing, and regulatory agencies, the World Medical Innovation Forum, in an all-virtual format, will explore interdependent aspects of advancing GCT for patients worldwide. Topics cover GCT strategy, clinical opportunities, patient access, economic and investment considerations, regulatory frameworks and manufacturing scalability. Nearly 1000 organizations will be represented, including attendees from 43 countries and 40 states of the U.S.

The Forum includes updates on:

GCT Potential for PatientsGene Therapies for Specific Diseases Large Incidence and Rare Trends in Vector Development, including AAV, Lentivirus and Nano MethodsClinical Opportunities, Technology and Market PotentialRegulatory Frameworks for GCTShaping GCT InnovationCancer, Gene Therapy and Oncolytic VirusesCAR-T TherapyGCT Manufacturing, Supply Chain and ScalabilityImpact of mRNA Vaccines

The Forum fosters critical discussions and debates on issues that will determine the pace of expansion of GCT therapies for patients in the coming years, says Chris Coburn, Chief Innovation Officer, Mass General Brigham. In the spirit of collaborative innovation, this gathering of leading investors, entrepreneurs, industry executives, Harvard clinicians and scientists is unique in convening top decision-makers in sharing ideas for advancing these game-changing technologies to the front lines of medicine.

We are honored to be the presenting sponsor of this years World Medical Innovation Forum, said Dave Lennon, President, Novartis Gene Therapies. Having brought one of the first gene therapies to market, we have seen the enormous potential these breakthrough medicines hold to transform lives. We look forward to joining other leaders at the forum to share learnings and our vision for the future one in which gene therapies drive scientific innovation and deliver on their promise for patients and societies.

Showcasing Mass General Brigham BreakthroughsThe Forum showcases GCT innovation breakthroughs from Mass General Brighams Harvard Medical School faculty investigators in 10-minute First Look presentations, and a research poster session being included in the Forum for the first time. More than 40 emerging GCT research projects will be part of the virtual showcase, with topics including gene therapy for large incidence and rare diseases including neurology, cardiology, blood disorders, and oncology, CAR-T breakthroughs, and oncolytic, AAV and non-viral delivery methods.

Recipients of Mass General Brighams Innovation Discovery Grants for GCT research will be announced. The Forum concludes with the announcement of the annual Disruptive Dozen, 12 GCT advances members of the Mass General Brigham faculty believe are the most likely to break through in the next few years.

This years Forum co-chairs are Nino Chiocca, MD, PhD, Chair, Neurosurgery, Brigham and Womens Hospital and Cushing Professor of Neurosurgery, Harvard Medical School (HMS); Sue Slaugenhaupt, PhD, Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute, and Professor, Neurology, HMS; Ravi Thadhani, MD, CAO, Mass General Brigham and Professor, Medicine and Faculty Dean, HMS; and Luk Vandenberghe, PhD, Grousbeck Family Chair, Gene Therapy, Mass Eye and Ear, and Associate Professor, Ophthalmology, HMS.

About the World Medical Innovation Forum

The World Medical Innovation Forum was established seven years ago in response to the intensifying transformation of health care and its impact on innovation. The Forum is rooted in the belief that no matter the magnitude of change, the center of health care needs to be a shared, fundamental commitment to collaborative innovation industry and academia working together to improve patient lives.

To find out more about the Forum, speakers and agenda, and to register, visit https://worldmedicalinnovation.org/###

About Mass General Brigham

Mass General Brigham is an integrated academic healthcare system, uniting great minds in medicine to make life-changing impact for patients in our communities and people around the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a non-profit organization that is committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nations leading biomedical research organizations and a principal teaching affiliate of Harvard Medical School. For more information, please visit massgeneralbrigham.org.

About Mass General Brigham Innovation

Innovation is the 150-person business development unit of Mass General Brigham responsible for the worldwide commercial application of the unique capabilities and discoveries of Mass General Brigham's 74,000 employees. Innovation supports the research requirements of its 6,200 Harvard Medical School faculty and research hospitals. It has responsibility for industry collaborations, venture investing, international consulting, licensing, innovation management, company creation, technology marketing, open innovation alliances, and workforce development.

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Gene and Cell Therapy Breakthroughs Focus of World Medical - GlobeNewswire

4D Molecular Therapeutics Reports Financial Results for the First Quarter of 2021 and Provides Operational Highlights – GlobeNewswire

EMERYVILLE, Calif., May 13, 2021 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT), a clinical-stage gene therapy company harnessing the power of directed evolution for targeted gene therapies, announced financial results for the first quarter of 2021, and provided operational highlights.

We continue to relentlessly execute and innovate as demonstrated by achievements in our first full quarter as a public company, said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. The company remains on track to announce initial clinical data from both our 4D-310 Fabry disease product candidate and our 4D-125 XLRP product candidate in the second half of this year. In addition, we remain on track to initiate clinical trials in the second half of this year for 4D-150, our wet AMD and DME product candidate, and for 4D-710, our cystic fibrosis lung disease product candidate. We also recently expanded our technology platform to include applications of machine learning, and yesterday, at the annual ASGCT conference, we presented preclinical non-human primate data from 4D-150.

Recent Operational Highlights

Expected Upcoming Milestones

Financial Results for the First Quarter Ended March 31, 2021

Cash and Cash Equivalents: Cash and cash equivalents were $259.9 million as of March 31, 2021. We expect cash and cash equivalents to be sufficient to fund operations into mid-2023.

Revenue: Total revenue was $2.0 million for the quarter ended March 31, 2021, as compared to $3.5 million for the quarter ended March 31, 2020. The decrease was primarily driven by decreased revenue recognized under the Roche collaboration agreement.

R&D Expenses: Research and development expenses were $12.8 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020. This decrease was primarily driven by decreased external manufacturing expense, which was partially offset by higher payroll and stock-based compensation expense.

G&A Expenses: General and administrative expenses were $5.5 million for the quarter ended March 31, 2021, as compared to $3.7 million for the quarter ended March 31, 2020. This increase was primarily due to higher payroll and stock-based compensation expense and higher business insurance expense.

Net Loss: Net loss was $16.4 million for the quarter ended March 31, 2021, as compared to $13.2 million for the quarter ended March 31, 2020.

About 4DMT

4DMT is a clinical-stage company harnessing the power of directed evolution for targeted gene therapies. 4DMT seeks to unlock the full potential of gene therapy using its platform, Therapeutic Vector Evolution, which combines the power of directed evolution with approximately one billion synthetic capsid sequences to invent evolved vectors for use in targeted gene therapy products. The company is initially focused in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The 4DMT targeted and evolved vectors are invented with the goal of being delivered through clinically routine, well-tolerated and minimally invasive routes of administration, transducing diseased cells in target tissues efficiently, having reduced immunogenicity and, where relevant, having resistance to pre-existing antibodies. 4DMT is currently conducting three clinical trials: 4D-125 is in a Phase 1/2 clinical trial for XLRP patients, 4D-110 is in a Phase 1 clinical trial for choroideremia patients and 4D-310 is in a Phase 1/2 clinical trial for Fabry disease patients.

4D Molecular Therapeutics, 4DMT, Therapeutic Vector Evolution, and the 4DMT logo are trademarks of 4DMT.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding plans and timelines for the clinical development of 4D-310, 4D-125, 4D-110, 4D-150 and 4D-710, including the therapeutic potential and clinical benefits thereof; the estimated timing of clinical data being available for 4D-125s Phase 1/2 clinical trial and 4D-310s Phase 1/2 clinical trial; the estimated timing of initiating the clinical trials for 4D-150 and 4D-710; expectations on how long our cash and cash equivalents can fund operations; expectations regarding current and future interactions with the U.S. Food and Drug Administration (FDA); and 4D Molecular Therapeutics' strategy, business plans and focus. The words "may," might, "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," expect, "estimate," seek, "predict," future, "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, risks associated with: the impact of COVID-19 on countries or regions in which we have operations or do business, as well as on the timing and anticipated results of our clinical trials, strategy and future operations; the delay of any current or planned clinical trials for the development of 4D Molecular Therapeutics' drug candidates, the risk that the results of our clinical trials may not be predictive of future results in connection with future clinical trials; 4D Molecular Therapeutics' ability to successfully demonstrate the safety and efficacy of its drug candidates; the timing and outcome of our planned interactions with regulatory authorities; and obtaining, maintaining and protecting our intellectual property. These and other risks and uncertainties are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics most recent Quarterly Report on Form 10-Q to be filed on or about the date hereof, as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements.

4D-310, 4D-125 and 4D-110 are our product candidates in clinical trials and have not yet been approved for marketing by the US FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of 4D-310, 4D-125, or 4D-110 for the therapeutic use for which they are being studied.

4D Molecular Therapeutics, Inc.Condensed Statements of Operations and Comprehensive Loss(Unaudited)(in thousands, except share and per share amounts)

4D Molecular Therapeutics, Inc.Condensed Balance Sheet Data(Unaudited)(inthousands)

Contacts:

Media:

Theresa Janketjanke@4dmt.com

Investors:

Mike ZanoniEndurance Advisorsmzanoni@4dmt.com

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4D Molecular Therapeutics Reports Financial Results for the First Quarter of 2021 and Provides Operational Highlights - GlobeNewswire

Global Cell and Gene Therapy Contract Manufacturing Organizations (CMOs) Market Report 2021: Increasing Need for CMOs to Cope with Demand for Cell and…

DUBLIN, May 10, 2021 /PRNewswire/ -- The "The Market for Cell and Gene Therapy Contract Manufacturing Organizations (CMOs)" report has been added to ResearchAndMarkets.com's offering.

Contract manufacturing is an increasing part of the pharmaceutical business. In cell therapy, this need is magnified because of the demand for cell and gene therapy products.

The report the Market for Cell and Gene Therapy Contract Manufacturing Organizations (CMOs) details the following:

There are many companies in this market, and the analyst profiles a select group of companies that are representative of the market or handle a large volume.

These companies include:

Key Topics Covered:

Chapter 1: Executive Summary

Overview

Scope and Methodology

Covid 19

Market Overview and Potential

Chapter 2: Introduction to Cell and Gene Therapy

Introduction

Chapter 3: Cell and Gene Therapy Industry - Product Pipeline, Production & Key Players

Overview

Chapter 4: Cell and Gene Therapy Markets

Overview

Chapter 5: Biopharma Contract Manufacturing

Introduction

Chapter 6: Biopharma Contract Manufacturing Markets

Overview

Chapter 7: Cell & Gene Therapy Contract Manufacturing

Introduction

Chapter 8: The Market for Cell & Gene Therapy Contract Manufacturing

Overview

Chapter 9: Key Players in the Cell & Gene Therapy CMO Market

For more information about this report visit https://www.researchandmarkets.com/r/jih3bw

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Global Cell and Gene Therapy Contract Manufacturing Organizations (CMOs) Market Report 2021: Increasing Need for CMOs to Cope with Demand for Cell and...

Cancer Gene Therapy Market Supply Chain Analysis, Trends and Insights | Key Participants Arimex, Hines Nut Company, Archer Daniels Midland, Olam…

The Cancer Gene Therapy Market is expected to grow at a CAGR of 9.36% and is poised to reach US$XX Billion by 2027 as compared to US$XX Billion in 2020. The factors leading to this extraordinary growth is attributed to various market dynamics discussed in the report. Our experts have examined the market from a 360 degree perspective thereby producing a report which is definitely going to impact your business decisions.

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Cancer Gene Therapy Market components like the business overview, SWOT analysis, inventive projects, services & product specifications, investment feasibility analysis, major development areas, development trends, as well as return analysis are well included in the market research report. However, some of the prominent players that are well involved in the target market are summarized totally in an organized manner. Also, widespread research on market segmentation along with product specifications, market size, and revenue are offered in the study of the Cancer Gene Therapy Market research report. In terms of revenue production capacity over the forecast period, The Global Market scope is evaluated. The major regions along with their revenue generation details are also contained in the market research report.To avail Sample Copy of report, visit @ https://decisivemarketsinsights.com/cancer-gene-therapy-market/06134194/request-sample

Key Companies Operating in this MarketKanegradeArimexHines Nut CompanyArcher Daniels MidlandOlam InternationalH.B.S. FoodsSun-MaidSunbeam FoodsGracelandDiamond Foods

Market by TypeGene Induced ImmunotherapyOncolytic VirotherapyGene Transfer

Market by ApplicationHospitalsDiagnostics CentersResearch Institutes

Competitive landscape of the global market in the industry. The growth rate and the global market size. Detailed analysis of major key players, key regions, and the leading vendors. Analysis of the market drivers, the market restraints, the market opportunities, and the market challenges Major trending factors along with their influence on a global basis. Vital outcomes of the research methodologies and the research techniques such as the quantitative and the qualitative analysis.

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North America US, Mexico, Canada Europe Russia, Ukraine, France, Spain, Sweden, Norway, Germany, Finland, Poland, Italy, United Kingdom, Greece, Austria, Denmark, Switzerland, Netherlands, Belgium, Turkey, Luxembourg Asia-Pacific China, Japan, India, Australia, South Korea, Taiwan, Malaysia, Philippines, Thailand, Singapore South America- Brazil, Argentina, Peru, ChileMiddle East and Africa Bahrain, Egypt, Israel, Kuwait, Qatar, Saudi Arabia, United Arab Emirates, South Africa

All the global governing factors, parent industry patterns, retail sales, macroeconomic indicators, and the businesss market segment attractiveness are well examined in the Cancer Gene Therapy Market research report. Also, the major Key leading Companies and a wide range of industries, as well as the market trends and the factors that have affected the industry largely, are well covered by the research study done in the research report. In the forecasted time frame, a quantitative, as well as qualitative analysis of the demand, is also provided in the Cancer Gene Therapy Market research report as well. Moreover, the industrys core dynamics possess the key market elements like the market drivers, rewards, market constraints, and market risks, etc.

Regarding the key segments and the sub-segmentations, a detailed explanation is offered by the report by describing the pointers on the applications, the product types, the regions, new products launched, innovative technologies, and other key factors as well. Also, the market research report has thoroughly evaluated the performance, the emerging market size, and the scope of each segment of the global market. The Cancer Gene Therapy Market research report includes a lengthy analysis of the various key leading players of the market in the industry and the recent vast activities by these leading players of the market which has helped them to change their market positions in these competitive markets.

Chapter1: Introduction and ScopeChapter2: Key Company ProfilesChapter3: Market Overview Share and ForecastChapter4: Market Overview of Asia Pacific regionChapter5: Market Overview of Europe regionChapter6: Market Overview of Asia Pacific regionChapter7: Market Overview of North America regionChapter8: Market Overview of Middle East and AfricaChapter9: Key Significant features of the marketChapter10: Key trends of the marketChapter11: Developments and Strategies

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An insight into the market size and growth 2020-2027 CAGR: 2020 to 2027, calculating 2019 as the base year Detail information about the dominant players in this segment Demand and supply chain mapped to clearly evaluate the market Apart from primary and secondary research methodology, data triangulation method is used for a clear understanding of the report Analysis by best expert in the industry

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Cancer Gene Therapy Market Supply Chain Analysis, Trends and Insights | Key Participants Arimex, Hines Nut Company, Archer Daniels Midland, Olam...

Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science…

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.

Effective gene therapy targeting MECP2 for the treatment of Rett syndrome has been elusive due to the inability to properly regulate transgene expression, said Steven Gray, Ph.D., Chief Scientific Advisor at Taysha and Associate Professor in the Department of Pediatrics at UT Southwestern. The built-in self-regulatory feedback loop mechanism in TSHA-102, work that was initiated in my laboratory in 2007, is a completely novel approach that allows for regulated expression of MECP2 on a cell-by-cell basis. These results published today are highly encouraging and allow us to conceive additional novel approaches using miRARE in conditions with dose-sensitive genes.

The complexities of developing an efficacious and well-tolerated gene therapy for the treatment of Rett syndrome are highlighted by phenotypic variability, mosaicism and the need to regulate MECP2 such that it does not cause overexpression-related toxicity. Todays data give us confidence that we can achieve appropriate MECP2 expression in all cells in a genotype-dependent manner, which we believe significantly de-risks the developmental program in its translation to humans, said Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. Historically, unregulated gene replacement of MECP2 resulted in overt adverse events, including death in wild type mice due to overexpression of the MECP2 protein. With the built-in regulatory element, miRARE, TSHA-102 provided a statistically significant survival extension by 56% in 4- to 5-week-old knockout Rett mice. We see the potential for broadening the miRARE platform to other CNS diseases requiring regulated gene expression. These positive data support our intent to file an IND/CTA for TSHA-102 in the second half of this year, followed by initiation of a Phase 1/2 trial by year-end 2021. TSHA-102 has the potential to address a significant unmet need for an estimated 25,000 patient with Rett syndrome across the United States and in Europe.

The preclinical study was conducted by the UT Southwestern Medical Center (UT Southwestern) laboratory of Sarah Sinnett, Ph.D., and evaluated the safety and efficacy of regulated miniMECP2 gene transfer, TSHA-102 (AAV9/miniMECP2-miRARE), via intrathecal (IT) administration in adolescent mice between four and five weeks of age. TSHA-102 was compared to unregulated full length MECP2 (AAV9/MECP2) and unregulated miniMECP2 (AAV9/miniMECP2).

TSHA-102 extended knockout survival by 56% via IT delivery. In contrast, the unregulated miniMECP2 gene transfer failed to significantly extend knockout survival at either dose tested. Additionally, the unregulated full-length MECP2 construct did not demonstrate a significant extension in survival and was associated with an unacceptable toxicity profile in wild type mice.

In addition to survival, behavioral side effects were explored. Mice were subjected to phenotypic scoring and a battery of tests including gait, hindlimb clasping, tremor and others to comprise an aggregate behavioral score. miRARE attenuated MECP2-mediated aggravation in wild type aggregate phenotype severity scores. Mice were scored on an aggregate severity scale using an established protocol. AAV9/MECP2- and AAV9/miniMECP2-treated wild type mice had a significantly higher mean (worse) aggregate behavioral severity score versus that observed for saline-treated mice (p <0.05; at 630 and 727 weeks of age, respectively). TSHA-102-treated wild type mice had a significantly lower (better) mean aggregate severity score versus those of AAV9/MECP2- and AAV9/miniMECP2-treated mice at most timepoints from 1119 and 920 weeks of age, respectively. No significant difference was observed between saline- and TSHA-102-treated wild type mice.

Of note, miRARE-mediated genotype-dependent gene regulation was demonstrated by analyzing tissue sections from wild type and knockout mice treated with AAV9 vectors given intrathecally. TSHA-102 demonstrated regulated MECP2 expression in different regions of the brain. In the pons and midbrain, miRARE inhibited mean MECP2 gene expression in a genotype-dependent manner as indicated by significantly fewer myc(+) cells observed in wild type mice compared to knockout mice (p<0.05), thereby demonstrating that TSHA-102 achieved MECP2 expression levels similar to normal physiological parameters.

It has been a challenge finding an approach that can appropriately regulate MECP2 expression in Rett syndrome but the preclinical data for TSHA-102 published today support the miRARE approach, said Sarah Sinnett, Ph.D., Assistant Professor in the Department of Pediatrics at UT Southwestern. It is clear that the disease is reversible, and I am encouraged that this novel strategy may enable us to make a difference in the management of this disease.

The publication is available by clicking on the following link: https://academic.oup.com/brain/advance-article-abstract/doi/10.1093/brain/awab182/6265600.

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.

Drs. Gray and Sinnett have intellectual property interest in Taysha and UTSW has a financial interest in the company.

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 the potential of our product candidates, including TSHA-102, to positively impact quality of life and alter the course of disease in the patients we seek to treat, 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, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements 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 Annual Report on Form 10-K for the full-year ended December 31, 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.

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Taysha Gene Therapies Announces Publication of Preclinical Data for TSHA-102 in Rett Syndrome in Brain, a Highly Esteemed Neurological Science...

Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -…

NEW YORK, and RESEARCH TRIANGLE PARK, N.C., April 28, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced four upcoming oral presentations at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), to be held virtually between May 11th to May 14th, 2021.

The AXO-AAV-GM1 presentation will include a review of patient-level data on safety and efficacy at 6 months follow up from the low-dose cohort of the Companys ongoing clinical study. Additionally, Dr. Cynthia Tifft, the lead investigator for the study, will present 6-month biomarker data from cerebrospinal fluid (CSF) in the 5 children who received intravenous AAV9 gene therapy.

Oral Presentation Details:

Presentation Title: AXO-AAV-GM1 for the Treatment of GM1 Gangliosidosis: Preliminary Results from a Phase I-II trialAbstract Number: 162Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Cynthia Tifft, MD, PhD, Deputy Clinical Director, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 6:15 PM 6:30 PM EDT

Presentation Title: AXO-Lenti-PD gene therapy for Parkinsons disease: efficacy, safety, and tolerability data from the second cohort in open-label dose evaluation study SUNRISE-PD at 6 months post administrationAbstract Number: 163Session: Clinical Trials and Advanced Preclinical Studies for Neurologic DiseasesPresenting Author: Gavin Corcoran, MD, Chief R&D Officer Presentation Date and Time: Thursday, May 13, 2021 6:30 PM 6:45 PM EDT

Presentation Title: Immune Modulation Preceding AAV9-GLB1 Gene Therapy Preserves the Possibility for Re-Dosing in Children with GM1 GangliosidosisAbstract Number: 179Session: Immunotherapy and VaccinesPresenting Author: Precilla DSouza, DNP, MSN, CRNP, National Human Genome Research InstitutePresentation Date and Time: Thursday, May 13, 2021 7:00 PM 7:15 PM EDT

Presentation Title: A GLP Safety and Biodistribution Study of AXO-Lenti-PD Manufactured via Two Processes Delivered at a Higher Volume and Flow RateAbstract Number: 256Session: Pharmacology/Toxicology Studies or Assay DevelopmentPresenting Author: Thomas Pack, PhD, Sio Gene TherapiesPresentation Date and Time: Friday, May 14, 2021 from 1:45 PM 2:00 PM EDT

About AXO-AAV-GM1

AXO-AAV-GM1 delivers a functional copy of theGLB1gene via an adeno-associated viral (AAV) vector, with the goal of restoring -galactosidase enzyme activity for the treatment of GM1 gangliosidosis. The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well. Preclinical studies in murine and a naturally-occurring feline model of GM1 gangliosidosis have supported AXO-AAV-GM1s ability to improve -galactosidase enzyme activity, reduce GM1 ganglioside accumulation, improve neuromuscular function, and extend survival.

AXO-AAV-GM1 has received both Orphan Drug Designation and Rare Pediatric Disease Designation from theFood and Drug Administrationand is the only gene therapy in clinical development for both Type I and Type II GM1 gangliosidosis.

In 2018, Sio licensed exclusive worldwide rights from theUniversity of Massachusetts Medical Schoolfor the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoff diseases.

About AXO-Lenti-PDAXO-Lenti-PD is an investigational gene therapy for the treatment of Parkinsons disease that is designed to deliver three genes (tyrosine hydroxylase, cyclohydrolase 1, and aromatic L-amino acid decarboxylase) via a single lentiviral vector to encode a set of critical enzymes required for dopamine synthesis, with the goal of reducing variability and restoring steady levels of dopamine in the brain. The investigational gene therapy aims to provide patient benefit for years following a single administration.Axovantexpects to dose the first patient in EXPLORE-PD, a randomized, sham controlled study in 2021.

AboutSio Gene TherapiesSio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies. For more information, visitwww.siogtx.com.

Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as believe, "estimate," may be and other similar expressions are intended to identify forward-looking statements. For example, all statements Sio makes regarding costs associated with its operating activities, funding requirements and/or runway to meet its upcoming clinical milestones, and timing of its upcoming clinical milestones are forward-looking. All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected. Such risks and uncertainties include, among others, the impact of the Covid-19 pandemic on our operations; the actual funds and/or runway required for our clinical and product development activities and anticipated upcoming milestones; actual costs related to our clinical and product development activities and our need to access additional capital resources prior to achieving any upcoming milestones; the initiation and conduct of preclinical studies and clinical trials; the availability of data from clinical trials; the development of a suspension-based manufacturing process for Axo-Lenti-PD; the scaling up of manufacturing, the expectations for regulatory submissions and approvals; the continued development of our gene therapy product candidates and platforms; Sios scientific approach and general development progress; and the availability or commercial potential of Sios product candidates. These statements are also subject to a number of material risks and uncertainties that are described in Sios most recent Quarterly Report on Form 10-Q filed with theSecurities and Exchange CommissiononFebruary 9, 2021, as updated by its subsequent filings with theSecurities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Media

Josephine Belluardo, Ph.D. LifeSci Communications(646) 751-4361jo@lifescicomms.cominfo@siogtx.com

Investors and Analysts

Parag V. Meswani, Pharm.D.Sio Gene Therapies Inc.Chief Commercial Officer investors@siogtx.com

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Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

SOLA Biosciences Presented Positive ALS Animal Efficacy Data of SOL-257 at the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular…

BOSTON--(BUSINESS WIRE)--SOLA Biosciences, LLC, today announced positive preclinical data in mice from a study designed to evaluate the efficacy of SOL-257, an innovative gene therapy candidate selectively targeting pathogenic TDP-43, which is found in approximately 97% of ALS patients. The data was presented during the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular Disease on April 29, 2021, by Akinori Hishiya, Ph.D., Founder/CSO of SOLA.

SOLA has developed an innovative chaperone technology (JUMP70) to selectively eliminate disease-causing misfolded proteins using the patients' own chaperones. SOLA has developed nine gene therapy candidates incorporating JUMP70 to address conformational diseases such as Amyotrophic Lateral Sclerosis (ALS), Huntington's disease, Parkinson's disease, and Alzheimer's disease. These candidates demonstrated preclinical activities and safety to prove the mechanism of the JUMP70 technology. SOL-257 is a flagship compound to selectively target misfolded and neurotoxic TDP-43 proteins without affecting healthy TDP-43.

At the conference, SOLA presented unprecedented in vivo efficacy data using the NEFH-hTDP-43NLS mouse model, which expresses a doxycycline-repressible form of human TDP-43 lacking a nuclear localization signal (NLS). The transgenic mice accumulate cytoplasmic insoluble TDP-43 in neurons of the brain and spinal cord soon after pathogenic TDP-43 proteins are expressed. The mice develop many features reminiscent of ALS, including motor deficits, denervation of neuromuscular junctions, motor neuron loss, and rapid loss in viability. The mice treated with control gene therapy (placebo) started dying 3 weeks after pathogenic TDP-43 expression, and by 5 weeks, 100% of male mice died. In contrast, all male mice treated with SOL-257 gene therapy survived.

"Now we know many diseases such as ALS are caused with protein folding issues. Repairing or removing the misfolded disease-causing proteins can be a fundamental treatment for the patients, although the treatment has not been realized yet. We learned the natural protein quality control system and carefully designed our novel therapeutics," says Dr. Akinori Hishiya. "Our designed therapeutic enables highly specific intervention, only misfolded disease-causing proteins can be targeted and removed."

"SOL-257 targeting only pathogenic TDP-43 to repair the specific problem has a significant potential for a broadly applicable ALS treatment," says Keizo Koya, Ph.D., Founder/CEO of SOLA. "I hope this groundbreaking animal data of SOL-257 will bring new hope for patients with ALS.

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SOLA Biosciences Presented Positive ALS Animal Efficacy Data of SOL-257 at the 1st Annual MDA Insight in Research Investor Summit for Neuromuscular...

Cell & Gene Therapy Technologies and Supplies Report 2021: Market Demand and Five-year Forecasts, Segmented by Technique, Region and Function -…

DUBLIN--(BUSINESS WIRE)--The "Cell & Gene Therapy Technologies and Supplies" report has been added to ResearchAndMarkets.com's offering.

Cell and gene therapy (CGT) is a rapidly evolving field producing powerful new treatments for cancers and genetic diseases, and expanding into autoimmune disease, cardiovascular disease, musculoskeletal disease, and many others. The CGT field has generated great interest and hope among researchers, patient groups, and regulators, which has led to huge investments in R&D.

Globally, many governments and regulatory bodies have adopted policy and patent environments supportive of CGT development. Since CGTs often target rare and underserved disease areas for which few other therapeutics exist, there is a sense of urgency within the pharma/bio space to develop CGTs.

As development and market entries of CGTs accelerate, the market for suppliers of laboratory and clinical tools within the CGT R&D and manufacturing spaces will see very rapid growth. The Cell & Gene Therapy Technologies and Supplies examines the global market for analytical technologies and products used throughout the various stages of CGT development and manufacturing, evaluating 21 technologies grouped into six categories.

The goal of this report is to provide demand growth projections by technique, region, and function, while also providing comprehensive views of the competitive landscape for each technology.

Report Overview

Participants include the following:

Key Topics Covered:

1. Introduction

2. Market Insights

3. Market Demand

3.1 Overall Demand

3.2 General Techniques

3.3 Gene Therapy Development

3.4 Transduction and Transfection

3.5 Cell Enrichment

3.6 Cell Culture and Cell Expansion

3.7 Market Demand by Technique (2020)

3.8 Cryopreservation

For more information about this report visit https://www.researchandmarkets.com/r/hrs0we

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Cell & Gene Therapy Technologies and Supplies Report 2021: Market Demand and Five-year Forecasts, Segmented by Technique, Region and Function -...

Lantheus Grants Allegheny Health Network Exclusive Rights for the Use of its Microbubbles in Combination with Ultrasound Assisted Gene Therapy for the…

Xerostomia, a lack of saliva production leading to dry mouth, has a variety of causes, including radiotherapy and chemotherapy, the chronic use of drugs and rheumatic and dysmetabolic diseases. It is also a common side effect of ionizing radiation used to treat head and neck cancer.1

A proof of concept Phase 1 clinical trial sponsored by a third party showed that Aquaporin-1 (AQP1), a trans-membrane protein that facilitates water movement across lipid layers, restored saliva flow in a human population using an adenovirus-based vector encoding AQP1 to a single previously irradiated parotid gland.2 The results from this study led AHN to research using UAGT technology targeting the salivary gland, which combines the use of nonviral DNA vector and lipid microbubbles with a low-frequency acoustic field to create a sonoporation effect allowing gene transfer to the cells of the salivary gland without the introduction of viral antigens.3

According to Mark Trombetta, MD, Director of Clinical Program Development for the AHN Cancer Institute, a radiation oncologist and co-investigator in the networks study of UAGT, thousands of cancer patients suffer from radiation-induced xerostomia, which can cause severe tooth decay, chronic oral pain, loss of taste, inability to eat properly and increased oral infections. Once xerostomia begins, it is a permanent condition.

We believe that UAGT may provide long-term relief of radiation-induced xerostomia, with adjustable dosing and potential for booster doses over time, said Warren Swegal, MD, an AHN head and neck surgeon and clinical lead of the networks UAGT program. No other existing treatment offers a long-lasting solution for this life-altering condition, and we are thrilled to be able to further develop and improve the therapy by leveraging Lantheus proven microbubble technology.

We are excited to support AHN in its efforts to progress this innovative development program. Xerostomia is a chronic and debilitating condition with limited treatment options for patients, said Mary Anne Heino, President and Chief Executive Officer of Lantheus. We believe our microbubble combined with AHNs UAGT technology have the potential to make a difference in the lives of these patients.

Lantheus will supply its microbubbles and activation devices to AHN. AHN will be solely responsible for all development work, future regulatory submissions and commercialization. The deal terms include a transfer price and royalties.

About Lantheus Holdings, Inc.

Lantheus Holdings, Inc. is the parent company of Lantheus Medical Imaging, Inc., Progenics Pharmaceuticals, Inc. and EXINI Diagnostics AB and an established leader and fully integrated provider of innovative imaging diagnostics, targeted therapeutics and artificial intelligence solutions to Find Fight and Follow serious medical conditions. Lantheus provides a broad portfolio of products, including the echocardiography agent DEFINITY Vial for (Perflutren Lipid Microsphere) Injectable Suspension; TechneLite (Technetium Tc99m Generator), a technetium-based generator that provides the essential medical isotope used in nuclear medicine procedures; AZEDRA for the treatment of certain rare neuroendocrine tumors; and RELISTOR for the treatment of opioid-induced constipation, which is partnered with Bausch Health Companies, Inc. The Company is headquartered in North Billerica, Massachusetts with offices in New York, New Jersey, Canada and Sweden. For more information, please visit http://www.lantheus.com.

About Allegheny Health Network

Allegheny Health Network (AHN.org) is an integrated healthcare delivery system serving the greater Western Pennsylvania region. The Network is composed of 13 hospitals, ambulatory surgery centers, Health + Wellness Pavilions, an employed physician organization, home and community-based health services, a research institute and a group purchasing organization. The Network provides patients with access to a complete spectrum of advanced medical services, including nationally recognized programs for primary and emergency care, cardiovascular disease, cancer care, orthopedic surgery, neurology and neurosurgery, womens health, autoimmune diseases, diabetes and more. AHN employs approximately 21,000 people, has more than 2,600 physicians on its medical staff and serves as a clinical campus for Drexel University College of Medicine and the Lake Erie College of Osteopathic Medicine.

Safe Harbor for Forward-Looking and Cautionary Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, that are subject to risks and uncertainties and are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by their use of terms such as anticipate, believe, confident, could, estimate, expect, intend, may, plan, predict, potential, project, target, will and other similar terms. Such forward-looking statements are based upon current plans, estimates and expectations that are subject to risks and uncertainties that could cause actual results to materially differ from those described in the forward-looking statements. The inclusion of forward-looking statements should not be regarded as a representation that such plans, estimates and expectations will be achieved. Readers are cautioned not to place undue reliance on the forward-looking statements contained herein, which speak only as of the date hereof. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law. Risks and uncertainties that could cause our actual results to materially differ from those described in the forward-looking statements include (i) expectations for future clinical trials, the timing and potential outcomes of clinical studies and filings and other interactions with regulatory authorities; (ii) the impact of legislative, regulatory, competitive and technological changes; (iii) AHNs ability to successfully launch its UAGT technology with our microbubble as a commercial product; and (iv) the risk and uncertainties discussed in our filings with the Securities and Exchange Commission (including those described in the Risk Factors section in our Annual Reports on Form 10-K and our Quarterly Reports on Form 10-Q).

1Pinna R, Campus G, Cumbo E, Mura I, Milia E. Xerostomia induced by radiotherapy: an overview of the physiopathology, clinical evidence, and management of the oral damage. Ther Clin Risk Manag. 2015; 11:171-188.2https://clinicaltrials.gov/ct2/show/NCT02446249 3Wang Z, Zourelias L, Wu C, Edwards PC, Trombetta M, Passineau MJ. Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion. Gene Therapy. 2015; 22:739-749.

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Lantheus Grants Allegheny Health Network Exclusive Rights for the Use of its Microbubbles in Combination with Ultrasound Assisted Gene Therapy for the...

CCRM and Amgen partner to advance emerging medical innovations – Canada NewsWire

New fund to support regenerative medicine discoveries with high potential impact for patients

TORONTO and MISSISSAUGA, ON, April 29, 2021 /CNW/ - New regenerative medicine discoveries, including cell and gene therapies, will never reach patients without access to the funding, expertise and other specialized support that are required to move along the commercialization pathway. To address this need, CCRM, a leader in developing and commercializing regenerative medicine-based technologies and cell and gene therapies, and Amgen, a global leader in the biotechnology industry, are announcing a multi-year fund, for early-stage regenerative medicine-based technologies and therapies to benefit patients and the health-care system. The collaboration is made up of equivalent investments from CCRM and Amgen.

To bridge the gap from bench to bedside, the program will identify, develop and commercialize promising technologies and therapies arising from research conducted in institutions that form CCRM's global network. CCRM and Amgen's contributions will range from financial support to in-kind technical services and expertise.

"CCRM's collaboration with Amgen to create this fund is a perfect example of how public-private partnerships can leverage resources and expertise to support development and commercialization, and change patients' lives," said Michael May, President and CEO, CCRM. "The regenerative medicineresearch ecosystemacross Canada offers a rich discovery pipeline and it is ready for such a program."

"There are few places in the world that have clustered all the necessary resources and talent to drive regenerative medicine from the bench to the bedside. Canada has consistently led the way for decades," said Alan Russell, Vice-President, Research, Amgen Inc."Amgen is delighted to have the opportunity to partner with CCRM and leverage an extraordinary platform to benefit patients."

To identify opportunities, a Joint Steering Committee, made up of representatives from both CCRM and Amgen, will assess proposals from CCRM's Canadian and international member institutions. Selected projects will possess high scientific merit, be developed by scientists and academics with established credentials and expertise, and demonstrate the greatest potential to have an impact in the industry.

Regenerative medicine, including cell and gene therapy, harnesses the power of (stem) cells, biomaterials, molecules and genetic modification to repair, regenerate or replace diseased cells, tissues and organs. It has the promise of creating revolutionary new treatments for devastating and costly conditions such as heart disease, diabetes and cancer.

About CCRM CCRM is a global, public-private partnership headquartered in Canada. It receives funding from the Government of Canada, the Province of Ontario, and leading academic and industry partners. CCRM supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of researchers, leading companies, strategic investors and entrepreneurs, CCRM accelerates the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding, and infrastructure. CCRM is the commercialization partner of the University of Toronto's Medicine by Design. CCRM is hosted by the University of Toronto. Visit us at ccrm.ca.

About Amgen in CanadaAs a leader in innovation, Amgen Canada understands the value of science. With main operations located in Mississauga, Ontario's vibrant biomedical cluster, and its research facility in Burnaby, B.C., Amgen Canada has been an important contributor to advancements in science and innovation in Canada since 1991. The company contributes to the development of new therapies and new uses for existing medicines in partnership with many of Canada's leading healthcare, academic, research, government and patient organizations. To learn more about Amgen Canada, visitwww.amgen.ca.

SOURCE Amgen Canada

For further information: CCRM: Stacey Johnson, Director, Communications and Marketing, 647-309-1830, [emailprotected]; Amgen Canada: Natasha Bond, Head of Corporate Affairs, 905-285-3007, [emailprotected]

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CCRM and Amgen partner to advance emerging medical innovations - Canada NewsWire

Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -…

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data presentations at the upcoming 24thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators will review new data from Rockets Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi Anemia (FA) gene therapy programs in oral and poster presentations.

Details for oral presentations are as follows:

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim ResultsSession: Genetic Blood and Immune DisordersPresenter: Donald Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesDate: Tuesday May 11, 2021Time: 6:15-6:30 p.m. EDTLocation: Room 7Abstract number: 39

Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric PatientsSession: Gene Therapies for HemoglobinopathiesPresenter: Jos Luis Lpez Lorenzo, M.D., Hospital Universitario Fundacin Jimnez Daz, Madrid, SpainDate: Wednesday May 12, 2021Time: 6:45-7:00 p.m. EDTLocation: Room 7Abstract number: 83

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene Therapy Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT) in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday, May 13, 2021Time: 10:00-10:45 a.m. EDTLocation: Room 7Abstract number: 36

Select results from Dr. Buerens presentation will also be highlighted by Paula Rio, Ph.D. Details for this Invited Presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCsSession: International Focus on Stem Cell Gene TherapyPresenter: Paula Ro, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundacin Jimnez DazDate: Thursday May 13, 2021Time: 10:00-11:45 a.m. EDT

Details for poster presentation are as follows:

Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical TrialsSession: Hematologic and Immunologic DiseasesPresenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of MedicineDate: Tuesday, May 11, 2021Time: 8:00-10:00 a.m. EDTLocation: Digital GalleryAbstract number: 697

Abstracts for the presentations can be found online at: https://annualmeeting.asgct.org/

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2021 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy -...

Freeline to Present Data at the American Society of Gene and Cell Therapy Annual Meeting 2021 – GlobeNewswire

LONDON, April 27, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline), a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that the Company will give six poster presentations at the American Society of Gene and Cell Therapy Annual Meeting 2021, taking place May 11 14.

We are excited to be presenting six posters at the upcoming ASGCT conference, which together highlight the scientific foundation that underlies our gene therapy programs and broader platform technology, said Theresa Heggie, CEO of Freeline. These posters are reflective of steady progress at Freeline, which we expect will include three gene therapies in the clinic by year end.

Poster Presentation Details

#329:FLT201, a Novel Investigational AAV-Mediated Gene Therapy Candidate for Gaucher Disease Type 1Presenter: RomualdCorbau, PhD, Chief Scientific Officer

#509:GLA Uptake and Metabolic Cross Correction in Fabry Disease Relevant Cell Lines: A Rationale for Liver-Directed AAV Gene TherapyPresenter:Jey Jeyakumar, PhD, Scientific Director

#821:Development of a 96-Well Plate-Based High-Throughput System forrAAVManufacturing Platform Optimization and Candidate SelectionPresenter: BettinaPrieler, PhD Student Technology Development

#843:Development and Scale Up of a Suspension Cell-Based AAV Manufacturing ProcessPresenter:Ahmed Youssef, Team Leader USP Development

#878:Defining a Reliable Quantification Assay Strategy for Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:FeliciaThoennissen, PhD, Scientist Analytical Development

#894:Development of an Assay to Measure Transduction Efficiency of Adeno-Associated Virus (AAV)-Based Gene TherapiesPresenter:Anita Heinlein, Scientist Analytical Development

Abstracts will be available on the ASGCT website starting today.

About Freeline Therapeutics

Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of the missing protein into the patients bloodstream. The Companys integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in Hemophilia B and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.

Forward-Looking Statements

This press release contains statements that constitute forward looking statements as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Companys opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. Examples include discussion of the Companys research, pipeline and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as anticipate, intend, believe, estimate, plan, seek, project or expect, may, will, would, could or should, the negative of these terms or similar expressions. Forward looking statements are based on managements current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to many risks and uncertainties, including the Companys recurring losses from operations; the development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review; the Companys ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt the Companys clinical trial pipeline; the Companys failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Companys control and could cause its actual results to differ materially from those it thought would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law. For further information, please reference the Companys reports and documents filed with the U.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on the SEC website at http://www.sec.gov.

Contact

David S. ArringtonVice President Investor Relations & Corporate CommunicationsFreeline Therapeuticsdavid.arrington@freeline.life+1 (646) 668 6947

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Freeline to Present Data at the American Society of Gene and Cell Therapy Annual Meeting 2021 - GlobeNewswire

CRISPR Technology Market: Rise in focus on gene therapeutics is projected to contribute to the growth of the market – BioSpace

CRISPR Technology Market: Introduction

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Key Drivers, Restraints, and Opportunities of Global CRISPR Technology Market

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CRISPR Technology Market: Rise in focus on gene therapeutics is projected to contribute to the growth of the market - BioSpace

New developments in the research field of immuno-oncology – Drug Target Review

The exciting potential of immunotherapy for cancer treatment continues its exploration and here, Drug Target Review investigates three of the latest pre-clinical developments in immuno-oncology research.

Scientists at McMaster University, Canada, have shown in new research that changing the metabolism of natural killer (NK) immune cells allows them to overcome the hostile conditions found inside tumours and destroy advanced ovarian and lung cancer.

In this study, we discovered that the metabolism, or energy hub, of NK cells is paralysed by tumours, causing the NK cells to undergo an energy crisis and lose their tumour-killing functions, said Sophie Poznanski, a PhD student and lead author of the study. With that understanding, we were able to reverse the dysfunction of NK cells by repurposing a pre-existing metabolism drug that restored their energy production.

Published in Cell Metabolism, the study shows that NK cells can also be modified to mimic the metabolism of tumours. These modified NK cells proved to be far better adapted for the hostile tumour environment, the team found.

We were just hoping that the modified NK cells would better resist suppression in tumours. We were astounded to see that not only did they show no suppression, but they paradoxically functioned better inside of the tumour than outside of it, said Poznanski.

This is the first report of an antitumour immune cell that exploits the hostility of tumours for their own advantage, said senior author Professor Ali Ashkar. Generating cytotoxic immune cells to have tumour-like metabolism is key for their antitumour functions in a very hostile environment of a solid tumour. This could be a paradigm shift for immune cell-based cancer immunotherapy.

So far, NK cells have only proven effective against blood cancers. However, re-programmed and trained NK cells could afford patients with otherwise terminal cancers with a safe and effective treatment option. Furthermore, immunotherapy with NK cells has already proven safe with few, if any, side effects.

This research revealed a novel targeted immunotherapy approach that employs new antibodies against genetically altered proteins to target cancers. The technique was developed at the Johns Hopkins Kimmel Cancer Center, US.

The researchers targeted their immunotherapy approach to alterations in the common cancer-related p53 tumour suppressor gene, the RAS tumour-promoting oncogene or T-cell receptor genes. Their findings are reported in three related studies published inScience Immunology, Science and Science Translational Medicine.

Although common across cancer types, p53 mutations have thus far not been successfully targeted with drugs. Genetic alterations in tumour suppressor genes often result in their functional inactivation.

Traditional drugs are aimed at inhibiting proteins. Inhibiting an already inactivated tumour suppressor gene protein in cancer cells, therefore, is not a feasible approach, said Dr Emily Han-Chung Hsiue, lead author on theSciencepaper.

Instead of drugs, the researchers set out to target these gene alterations with antibodies. Conventional antibodies require an antigen target on the cell surface most commonly a protein that looks like a foreign invader to the immune system. However, the proteins produced by mutant oncogenes and tumour suppressor genes are inside the cells, out of reach from conventional antibodies. However, proteins are routinely degraded within cells, generating protein fragments called peptides.

These peptides can be presented on the cell surface when complexed with the human leukocyte antigen (HLA) proteins, saidKatharine Wright, postdoctoral fellow and a lead author on theScience Immunologypaper. Mutated proteins in cancer cells can also be degraded and generate mutant peptides presented by the HLA molecules. These mutant peptide HLA complexes serve as antigens and mark cancer cells as foreign to the immune system.

They developed an approach in the form of bispecific antibodies, comprising one component that specifically recognises cancer cells and another component that recognises immune cells and brings both cells together. In laboratory and animal tumour cell models, it resulted in the destruction of tumour cells.

This therapeutic strategy is dependent on a cancer containing at least one p53 or RAS alteration and the patient having an HLA type that will bind to the mutant peptide to present it on the cell surface, said senior authorAssociate Professor Shibin Zhou, a study leader.

In theScience Translational Medicinepaper, the researchers report that the powerful bispecific antibody approach they developed could also be used for the treatment of T-cell cancers. In animal models, the researchers showed that their approach selectively killed the cancerous T cells while sparing the majority of healthy T cells.

The scientists say the next research steps are to assess whether the strategy can be applied to other gene alterations in p53, KRAS and other cancer driver genes.

We intend to develop a large number of bispecific antibodies that would target such genes, said Alex Pearlman, PhD student and co-author of the three studies. Although any individual bispecific antibody would target a small fraction of cancer patients, a suite of antibodies would allow for the treatment of many patients.

Researchers at Moffitt Cancer Center, US, have investigated how to combine and sequence new therapies to improve survival of patients with advanced melanoma. In a new article published inCancer Immunology Research, the team demonstrated that sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses in pre-clinical models and may be a potential treatment option for patients.

One of the most common genetic alterations in melanoma are mutations of the BRAF gene, which affect approximately 50 percent of patients. These alterations result in downstream signalling through the protein MEK and stimulation of cell growth, invasion and survival. Several therapies that target both BRAF or MEK have been approved and standard treatment for patients who have BRAF mutations is now combination therapy with BRAF and MEK inhibitors.

Given the success of both BRAF/MEK inhibitors and immunotherapies, researchers have attempted to combine these two classes of anticancer therapies. However, some of these combinations have not always been successful.

Initial attempts to develop targeted therapy/immunotherapy combinations clinically were not successful due to severe toxicity, said Dr Keiran Smalley, lead researcher.

These toxicities led the research team to determine whether using a sequential treatment approach rather than a simultaneous combination approach would lead to durable antitumour activity in BRAF and NRAS-mutated melanoma.

The researchers analysed sequential combinations of immunotherapy followed by either BRAF/MEK-targeted therapy or another targeted therapy combination (ceritinib/trametinib) and vice versa in pre-clinical mouse models. They discovered that immunotherapy followed by targeted therapy was more effective than either treatment approach alone. While using targeted therapy followed by immunotherapy was better than targeted therapy alone, it was not as effective as the immunotherapy-targeted therapy sequence.

The team discovered the immunotherapy-targeted therapy sequence modulated the environment to promote immune cell functions resulting in antitumour activity while simultaneously blocking activity that permitted tumour cell escape from immune detection. They also showed that the immunotherapy-targeted therapy sequence enriched the melanoma cells for proteins that promoted immune cell detection and suppressed signalling pathways associated with drug resistance.

sequential administration of immunotherapy followed by targeted therapy prolongs antitumour responses

Results from this research suggest that sequential treatment approaches with anti-PD-1 agents followed by targeted therapy may be clinically beneficial and several ongoing trials are currently investigating this hypothesis.

Together, our data demonstrates that sustained antitumour responses to targeted therapy are dependent upon a vigorous, sustained immune response and that upfront use of immunotherapy can augment this, said Smalley.

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New developments in the research field of immuno-oncology - Drug Target Review

Precision BioSciences Announces Poster Presentation at the Upcoming American Society of Gene & Cell Therapy Annual Meeting – Business Wire

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies, today announced that the following poster, highlighting a preclinical research collaboration using its ARCUS genome editing platform for treatment of transthyretin amyloidosis (ATTR), will be presented at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, scheduled for May 11-14, 2021.

Title: Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal modelsPoster Session: Metabolic, Storage, Endocrine, Liver and Gastrointestinal Diseases, Abstract 497Date/Time: Tuesday May 11, 2021 8:00 AM - 10:00 AMPresenting Author: Jenny A. Greig, Ph.D., Senior Director, Gene Therapy Program, Perelman School of Medicine, University of PennsylvaniaCo-Authors: Cassandra Gorsuch2, Joanna K. Chorazeczewski1, Melanie K. Smith1, Thomas Furmanak1, Alexa N. Avitto1, Scott N. Ashley1, Wendy Sharer2, Hui Li2, Jeff Smith2, Peter Clark1, Camilo Breton1, Derek Jantz2, and James M. Wilson1

Transthyretin amyloidosis is a rare disease caused by the progressive accumulation of misfolded transthyretin (TTR) protein into amyloid fibrils, which leads to peripheral neuropathy and/or cardiomyopathy. Research to be presented at the annual ASGCT meeting, led by Dr. Jenny A. Greig at the Perelman School of Medicine, University of Pennsylvania, used an AAV vector for in vivo delivery of ARCUS gene editing nucleases to knock out the TTR gene, which is responsible for ATTR.

With this program, we are excited to continue building a dataset demonstrating in vivo gene editing in large animal models using ARCUS nucleases, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. In this study, use of an optimized ARCUS nuclease to knock out the TTR gene was found to be effective in both mice and nonhuman primates, where we observed a good correlation between TTR gene editing in the liver and reductions of TTR in the serum. This approach addresses the root cause of the disease and results in genomic edits that are expected to be permanent. These results continue to demonstrate the power and versatility of ARCUS nucleases, particularly for in vivo editing.

Abstracts for the ASGCT 2021 Meeting are available on the meeting website.

About ARCUSARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including more than 75 patents to date.

About Precision BioSciences, Inc.Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its wholly proprietary ARCUS genome editing platform. ARCUS is a highly specific and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene correction therapy candidates to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward Looking StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding statements regarding the potential results, uses and advancement of our in vivo gene editing programs and ARCUS-based gene editing technology, including, without limitation, its attributes and effects upon the transthyretin gene, transthyretin serum levels and transthyretin amyloidosis. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, believe, could, eligible, expect, expected, should, plan, intend, estimate, target, mission, goal, may, will, would, should, could, target, potential, potentially, promising, project, predict, contemplate, potential, or the negative thereof and similar words and expressions.

Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events effects of the outbreak of COVID-19, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the year ended December 31, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors & Media page of our website at investor.precisionbiosciences.com.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

1 Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA2 Precision BioSciences, Inc., Durham, NC, USA

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Precision BioSciences Announces Poster Presentation at the Upcoming American Society of Gene & Cell Therapy Annual Meeting - Business Wire

Gene Therapy Market Worth US$ 20.9 billion UnivDatos Industry Analysis- by Size, Share, Growth, Trends, and Forecast 2021-2027 The Courier – The…

As per the research report, Global Gene Therapy Market is expected to reach the market valuation of US$ 20.9 billion by 2027 expanding at a reasonable CAGR of 29.7% during the forecast period (2021-2027) from US$ 3.5 billion in 2019.

Gene therapy is a technique that involves inserting genetic material into cells in order to correct for abnormal genes or produce a desirable protein. The growing number of ongoing clinical trials, as well as significant mergers, acquisitions, and venture capital investments in the gene-therapy field, suggest that the future of such therapies is promising. About 2,600 gene therapy clinical trials have been completed, are in progress, or have been accepted around the world to date. Gene therapy researchers are working more than ever to find a path to the clinic and the market. About two thousand clinical trials in human gene therapy have been published worldwide, and about 20 gene therapies have been approved. Such progress raises the prospect of treating devastating rare and hereditary illnesses, as well as incurable diseases.

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As of the end of the first quarter of 2019, 372 gene therapy clinical trials were in development, according to the Alliance for Regenerative Medicines (ARM) Quarterly Regenerative Medicine Global Data Study. Surprisingly, trials in Phase II had the largest margin (217 or 58 percent), followed by Phase I (123 or 33 percent), and Phase III (123 or 33 percent) (32 or 9 percent). The number of gene therapy clinical trials increased by ten from the previous years total of 362 trials. In fact, from 319 trials in progress in Q1 2018, the number of clinical tests has increased by 17% year over year. Many gene therapy clinical trials have focused on treating cancers such as breast, gynaecological, skin, urological, neurological, and gastrointestinal tumours, as well as haematological malignancies and paediatric tumours. According to the International Agency for Research on Cancer, 1 in 5 people will grow cancer over their lifetime, with 1 in 8 men and 1 in 11 women dying from it. According to these recent estimates, nearly 50 million people are living within five years of a previous cancer diagnosis. Globally, ageing demographics and socioeconomic risk factors continue to be major factors driving this increase.

For a detailed analysis of the applications in the Gene Therapy Market browse through https://univdatos.com/report/global-gene-therapy-market-current-analysis-and-forecast-2020-2027

Insights Presented in the Report

Amongst vectors, Viral vectors segment holds the major share

The market is divided into viral and non-viral vectors based on the form of vector. Due to major advances in vector engineering, distribution, and protection, viral vectors dominated the industry in 2019 with an 88.6 percent share and are projected to continue their dominance over the forecast era.

Amongst viral vectors, adeno-associated virus vectors are anticipated to dominate the market during the analyzed period

Lentivirus, adeno-associated virus, retrovirus & gammaretrovirus, transformed herpes simplex virus, and adenovirus are the major segments of the viral vector market. Because of the fast penetration into the host genome, no viral genes, ability to transduce cells that are not actively dividing, a wide variety of host cells, and being non-inflammatory and non-pathogenic, the adeno-associated virus sub-segment accounted for a maximum market sales share of 34% in 2019 and is projected to remain dominant throughout the studied timeframe. The non-viral vector, on the other hand, is expected to rise at the fastest rate over the projected period.

Amongst gene type, antigen segment dominated the market during the forecast period

The market is divided into antigen, cytokine, tumour suppressor, suicide, deficiency, antibody, and others based on gene form. The antigen category has the highest market sales share of 19.2 percent in 2019 and is projected to remain dominant over the forecast period.

Amongst indication, oncology segment holds the major share

The industry is divided into oncology, rare disorders, respiratory, neurology, infectious diseases, among others based on indication. Thanks to the prevalence of cancer cases, the oncology segment accounted for the largest market share of 48.6% in 2019 and is projected to be the leading segment of the gene therapy market over the forecast period.

Amongst the delivery method, in-vivo segment dominated the market during the forecast period

The market is primarily divided into in-vivo and ex-vivo delivery methods, depending on the method of delivery. Due to the direct transfer of genes into patients, the in-vivo segment accounted for a maximum market revenue share of 87.5 percent in 2019 and is projected to remain dominant throughout the studied timeframe.

North America represents one of the largest markets of Gene Therapy market

A systematic study of the consumer dynamics of the gene therapy market was performed for various regions around the world, including North America (the United States, Canada, and the Rest of North America), Europe (Germany, France, Italy, Spain, United Kingdom and Rest of Europe), Asia-Pacific (China, Japan, Australia, South Korea, and the Rest of APAC), and the Rest of the World. Because of the high incidence of cancer, the presence of high disposable income, and a rise in support for R&D initiatives associated with gene therapy, North America led the industry and reported sales of US$ 1.7 billion in 2019.

Customization Options:

The Gene TherapyMarket can further be customized as per the requirement or any other market segment. Besides this, UMI understands that you may have your own business needs, hence feel free to connect with us to get a report that completely suits your requirements.

Table of Contents

1 Market Introduction

2 Research Methodology or Assumption

3 Industry Performance

4 Executive Summary

5 Top Start-Ups Under Gene Therapy Sector

6 COVID-19 Impact

7 Market Insights by Vector

8 Market Insights by Gene Type

9 Market Insights by Indication

10 Market Insights by Delivery Method

11 Market Insights by Region

12 Gene Therapy Market Dynamics

13 Legal & Regulatory Framework

14 Demand and Supply Side Analysis

15 Value Chain Analysis

16 Gene Therapy Market Opportunities

17 Gene Therapy Market Trends & Insights

18 Competitive Scenario

19 Company Profiled

20 Disclaimer

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Gene Therapy Market Worth US$ 20.9 billion UnivDatos Industry Analysis- by Size, Share, Growth, Trends, and Forecast 2021-2027 The Courier - The...

FDA removes clinical hold on uniQure’s haemophilia gene therapy – PMLiVE

The US Food and Drug Administration (FDA) has removed a clinical hold on Dutch biotech uniQures haemophilia B gene therapy, following concerns over a case of cancer in a patient in a pivotal trial.

In December 2020, the FDA placed a hold on uniQures haemophilia B clinical programme following the diagnosis of hepatocellular carcinoma (HCC) a type of liver cancer in a patient in the HOPE-B trial evaluating AMT-061 (etranacogene dezaparvovec).

In a statement, uniQure said that the patient diagnosed with HCC had multiple risk factors associated with this type of cancer. This included a 25-year history of hepatitis C (HCV) as well as a history of hepatitis B (HBV). Chronic infections with both HCV and HBV are associated with around 80% of HCC cases.

Multiple analyses, following a surgical resection of both the patients tumour and adjacent liver tissue, showed that AAV vector integration in the tissue sample was extremely rare, accounting for 0.027% of the cells in the sample.

The integration events that were present were randomly distributed, the company added, with no signs of clonal expansion or any dominant integration event.

Whole genome sequencing of the tumour also confirmed that it had genetic mutations characteristic of HCC, independent of vector integration.

UniQure noted that a gene expression analysis of the tumour and adjacent tissue suggested a precancerous state in the liver that could have predisposed the patient to developing HCC.

Patient safety is our top priority, and we are grateful to our advisors and the FDA for their help in resolving this clinical hold, said Ricardo Dolmetsch, president of research and development at uniQure.

Our comprehensive investigation showed that AMT-061 is very unlikely to have contributed to the HCC in our patient. We look forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter, he added.

UniQure added that all patients in its haemophilia clinical programme have abdominal ultrasounds performed one year after dosing, with patients continuing to receive this test every six months.

AMT-061 has been granted breakthrough therapy designation by the FDA and a priority medicine (PRIME) designation by the European Medicines Agency (EMA).

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FDA removes clinical hold on uniQure's haemophilia gene therapy - PMLiVE

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