Archive for the ‘Gene Therapy Research’ Category

ALISO VIEJO, Calif.--(BUSINESS WIRE)--The international rare disease patient advocacy organization, Global Genes, is pleased to announce the 6th Annual RARE Drug Development Symposium (RDDS) in partnership with the Orphan Disease Center of the University of Pennsylvania. The June 9-11 virtual event will connect and educate hundreds of advocates, clinicians, and academic and industry researchers to explore the latest science, opportunities, and challenges to the advancement of therapies for more than 7,000 rare diseases.

New to this year's event is an optional preconference workshop targeted to attendees who are in the earlier stages of their research efforts or careers and looking to build knowledge in core competencies that will be expanded upon in the main RDDS program. The main program will address the current landscape of rare drug development and allow attendees to interact with subject matter experts and other rare disease stakeholders to help them better understand and develop their impact and role in advancing potential treatments.

This year, the RDDS keynote speaker will be David Fajgenbaum, M.D., MBA, MSc, co-founder and executive director of the Castleman Disease Collaborative Network (CDCN), assistant professor of medicine in Translational Medicine & Human Genetics at the University of Pennsylvania, associate director, Patient Impact for the Orphan Disease Center of the University of Pennsylvania, and author of the national bestselling book, Chasing My Cure: A Doctors Race to Turn Hope Into Action.

Dr. Fajgenbaum has been a leader in helping researchers to prioritize treatments for COVID-19 clinical trials and inform patient care through the CORONA (COvid19 Registry of Off-label & New Agents) Project. Were thrilled to have him share his insights during this critical time in health care and understand how we can apply these approaches to accelerate progress in rare disease research and treatments, said Craig Martin, CEO at Global Genes. The rare disease leaders who will be featured during the RDDS have tremendous depth of knowledge to share, and we look forward to sharing it with members of the rare community during this event.

RDDS will continue to host the CureAccelerator Live! For Rare Diseases 2021 event on June 10 in partnership with Cures Within Reach, a not-for-profit organization exclusively dedicated to using the speed, safety, and cost-effectiveness of already approved drugs, devices, diagnostics, nutraceuticals, and combination products to impact patients with unmet medical needs driving more treatments to more patients more quickly.

The emergence of therapeutic platforms creates unprecedented opportunities for treatments to improve the lives of those living with rare diseases, said Jim Wilson, M.D., Ph.D., director, Gene Therapy Program, and Rose H. Weiss, professor and director at the Orphan Disease Center of the University of Pennsylvania. We are delighted to collaborate with Global Genes to educate the rare disease community on research directed to these treatments.

Thank you to our gold sponsors, Horizon Therapeutics and Greenwich Biosciences, and silver sponsor, Pfizer, Inc., for their generous support of this important event.

For more information, visit http://www.globalgenes.org/rdds.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes and Orphan Disease Center of the University of Pennsylvania to Convene Patient Group Leaders, Researchers, Clinicians and Industry for...

Professor Naoto Hirano of the Temerty Faculty of Medicine and collaborators have developed a new technology that rigorously and robustly identifies the immune cells that are capable of recognizing and eliminating cancer cells.

The findings, published in Nature Biotechnology, pave the way for novel immunotherapies to help more patients, regardless of their genetic ancestry, live longer and healthier lives.

Adoptive cell therapy, a promising cancer treatment that uses our immune system to eliminate cancer cells, is effective only for a small subset of individuals with specific types of cancer and specific inherited genes. The new technology developed by Hirano, a professor in the department of immunology, allows researchers to develop new immunotherapies for cancer patients that are not limited by the differences or heterogeneity of tumour cells, expanding the potential impact of immunotherapy for patients around the world.

The technology applies to an immunotherapy approach called T cell receptor (TCR) gene therapy that is based on genetically-engineered immune cells (T cells) recognizing and binding to specific molecules, called peptide-loaded human leukocyte antigens (HLA), on the surface of cancer cells. Although there has been progress in TCR therapy, there are more than 28,000 different variations of HLA found in humans and current TCR therapies only work for a few of these variations.

Historically, TCR treatments have been developed for those who had the most common and well-studied HLA alleles, which often meant that these immunotherapies only worked for people from Caucasian ancestry, says Hirano, who is also a senior scientist at the Princess Margaret Cancer Centre and Ontario Institute for Cancer Research clinician scientist.

It was an important goal for us to develop a technology that could work for a broad range of HLA alleles. Were proud of what we developed because it could help many more cancer patients in the future.

The technology presented in this study involves a methodology that can in a single step, at a low expense form a functional protein structure, called a dimer, that is composed of any peptide and HLA molecule, regardless of type, and can bind to and identify a variety of T cells. The method improves the binding affinity between T cells and HLA molecules nearly 200-fold relative to prior methods, which could allow researchers to better identify and engineer the T cells for novel immunotherapies.

The technology has been licensed to TCRyption Inc., a company co-founded by Hirano,for further development, translationand large-scale implementation. In the future, it may be applied to fields other than cancer research and care, including autoimmune diseases such as rheumatoid arthritis and type 1 diabetes.

Im grateful for the cancer research communitys support over the years, which has enabled me to focus on important and challenging issues, says Hirano, who was named the University Health Networks Inventor of the Year last year for developing these analysis techniques. Only with the support for rigorous experimentation, deep expertise, and innovative thinking, were we able to make this breakthrough.

This article was originally published by the Ontario Institute for Cancer Research.

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New technology could allow more cancer patients to benefit from immunotherapy: U of T researchers - News@UofT

BioCentriq, the New Jersey Innovation Institutes cell and gene therapy development and manufacturing center, on March 23 announced it partnered with Kytopen, a Cambridge-based startup spun out of the Massachusetts Institute of Technology (MIT).

Our mission at BioCentriq is to work with innovative industry partners like Kytopen to advance the production and manufacturing of cell and gene therapies, making them accessible and affordable for the patients who so desperately need them, said Haro Hartounian, and SVP, general manager, BioCentriq. This partnership aligns perfectly with that mission.

Kytopen leadership team KYTOPEN

Kytopens proprietary Flowfect technology is a flexible, complete technology solution for non-viral cell engineering that integrates the discovery, development, and manufacturing of cell and gene therapeutics. The platform speeds therapies from the clinic to commercial use by enabling cell engineering without compromising functionality or viability. Kytopens technology reduces risk and provides maximum control and flexibility to drive higher yields, faster approvals, and better outcomes for curative cellular disease treatment.

The Flowfect platform is a transformative solution that eliminates the complexity of gene delivery for cell engineering and links discovery, development and manufacturing in one flexible scalable solution, stated Paulo Garcia, CEO and co-founder of Kytopen. Our goal is to enable simple and efficient non-viral manufacturing of cell therapies in days versus weeks to help patients; our partnership with BioCentriq accelerates that goal.

Our Flowfect technology utilizes a novel combination of electrical energy and continuous fluid flow to engineer cells, said Bethany Grant, head of research and development at Kytopen. Our ability to engineer billions of cells in minutes with minimal disruption unlocks new opportunities to enable curative therapies in autologous or allogeneic therapeutic applications.

In the initial phase of the collaboration, the Kytopen and BioCentriq teams will demonstrate the impact to both autologous and allogeneic cell therapies by integrating this novel transfection technology with other steps in the manufacturing process.

BioCentriq has a manufacturing facility in Newark and a pilot plant in South Brunswick.

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BioCentriq partners with Kytopen to advance production and manufacturing of cell and gene therapies - NJBIZ

The global cell and gene therapy drug delivery devices market was valued at $55.75 thousand in 2019, and is expected to reach $375.13 thousand by 2030, registering a CAGR of 16.61% during the forecast.

The global cell and gene therapy drug delivery devices marketis projected to reach $375.13 thousand by 2030, reveals the premium market intelligence study by BIS Research. The study also highlights that the market is set to witness a CAGR of 16.61% during the period between 2020 and 2030.

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BIS Research study indicates that the increasing global geriatric population, prevalence of genetic disorders, the increased demand for gene therapeutics that not only cure the chronic conditions completely but also improve the quality of life of the patients are the major factors anticipated to contribute to the growth of the global cell and gene therapy drug delivery devices market.

The study highlights the various emerging opportunities, such as strong pipeline and drug approvals of cell and gene therapies, introduction of cell and gene therapy drug delivery devices, potential technologies in cell and gene therapy drug delivery devices market, original equipment manufacturers, clinical trial scenario, and approved cell and gene therapy drug delivery devices. Scope of cell and gene therapy drug delivery devices, the clinical trial landscape of cell and gene therapies in China, the U.S, and across the world, challenges in cell and gene therapy drug delivery devices, and massive scope for adoption of cell and gene therapy drug delivery devices in emerging nations that can be leveraged by players operating in the market.

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To emphasize the dominance of the intravenous catheter segment of cell and gene therapy drug delivery devices market by product segment over other segments under the product category of cell and gene therapy drug delivery devices market in 2020 and 2030, Raviteja Palakurthy, Senior Research Analyst BIS Research, states, "The reason for market growth and the dominance of intravenous catheter segment can be attributed to the increasing global usage of intravenous catheters to deliver drugs that are dosed frequently for fairly long periods of time and for specific disease conditions. For most of currently approved cell and gene therapies such as Kymriah, Yescarta, Zolgensma, Provenge, Strimvelis, Zynteglo, and Tecartus intravenous catheter are used as its drug delivery device

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BIS Research Study Highlights the Global Cell and Gene Therapy Drug Delivery Devices Market to Reach $10.82 Billion by 2030 investigated in the latest...

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Maze Therapeutics, a company translating genetic insights into new precision medicines, today revealed its first three lead therapeutic candidates in the companys wholly owned pipeline. The candidates include:

Each of the three lead candidates was enabled by Mazes COMPASS platform, which uncovered important new findings for the genetic target, discerning which specific signals may be critical for the treatment of patients, and which are likely non-actionable. The Maze pipeline will have the potential to serve as precision medicines for rare diseases and mechanistically defined subsets of common diseases based on certain genetic drivers.

In addition, Maze is concurrently leveraging COMPASS to advance additional discovery-stage research programs across three main therapeutic areas of focus: metabolic, cardio/renal and neurological diseases. These programs will constitute a broad, diverse pipeline for Maze and will be a combination of wholly owned and partnership-led collaborations.

Maze was built by co-founders, including Charles Homcy and other preeminent thinkers in the field of genetics, on a bold vision to leverage growing knowledge of genetic drivers of disease in order to create precision medicines for the treatment of both rare and more common diseases, said Jason Coloma, Ph.D., president and chief executive officer of Maze. Since our founding, we have been leveraging insights from leading geneticists, combined with the growing availability of paired human genetic and clinical data, the evolution of functional genomic technologies and advances in computational power, to build our COMPASS platform in order to bring unique insights into efficient, genetics-based drug development. We are excited by the significant progress we have made with our platform and pipeline, bringing us an important step closer to our goal of delivering the right drug to the right patient at the right time.

Mazes therapeutic candidates are designed to: 1) target genes whose activity affects the phenotype associated with another, often distant, gene, referred to as genetic modifiers; 2) mimic the activity of protective genetic variants; 3) correct the effects of toxic genetic variants; or 4) leverage new genetic insights to address otherwise challenging drug targets.

COMPASS is a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. In addition, Maze is exploring applications of COMPASS in diseases of haploinsufficiency by identifying genetic mechanisms that increase levels of a deficient protein and translating them into therapeutics.

New findings using COMPASS helped fill in fundamental data gaps, turning known but challenging targets into exciting, differentiated approaches to the genetic drivers of disease for our first three programs, said Sarah Noonberg, M.D., Ph.D., chief medical officer of Maze. While it has been shown that targets with human genetic evidence are more likely to yield efficacious treatments, very few groups have had the capabilities to then turn genetic insights into viable drug programs. We believe our COMPASS platform, integrated with our extensive drug discovery capabilities, will allow us to accelerate the pace of therapeutic development, as well as increase the likelihood of producing therapies that provide meaningful clinical benefit for patients. We are excited to advance these initial programs and look forward to continued progress toward the clinic as efficiently as possible.

About Mazes Wholly Owned Programs

GYS1 Program for Pompe DiseasePompe disease is a rare, inherited autosomal recessive disorder with an incidence of approximately 1 in 40,000 live births in the U.S., and is estimated to affect 5,000 to 10,000 patients worldwide. It is caused by mutations in the GAA gene, which codes for an enzyme responsible for breaking down lysosomal glycogen into glucose. As a result of this mutation, glycogen accumulates in various tissues, particularly skeletal and cardiac muscle tissues, causing progressive weakness and respiratory insufficiency.

Maze is developing a novel, oral approach to treating Pompe disease by inhibiting the protein muscle glycogen synthase, which is encoded by the gene GYS1. Targeting this protein leads to reduction in the synthesis of glycogen, which is expected to restore glycogen balance through a mechanism called substrate reduction. While GYS1 has been a therapeutic target of interest, its attractiveness as a therapeutic target has been limited due to its structural complexity and uncertainties related to the tolerability of a long-term reduction in muscle glycogen levels. Critical insights derived from COMPASS have enabled Maze to overcome these challenges. Maze has interrogated the structurally complex protein to develop an oral inhibitor of muscle glycogen synthase, a target not previously addressable by small molecule therapies. Maze is rapidly progressing its GYS1 program toward an Investigational New Drug application and expects to initiate clinical trials in the first half of 2022.

APOL1 Program for Chronic Kidney DiseaseCKD affects approximately 37 million people in the U.S., including more than 700,000 patients who suffer from end-stage renal disease (ESRD), many of whom require chronic dialysis. Individuals of African ancestry are at an approximately 3.5-fold greater risk of developing ESRD than individuals of European ancestry. Previous studies have shown that two coding variants of the apolipoprotein L1 (APOL1) encoded by the gene APOL1 cause toxic gain-of-function variants and are important genetic drivers of kidney disease that are responsible for much of the increased risk for CKD and ESRD in individuals of African ancestry. There are currently no approved therapies that address the underlying causes of APOL1-associated CKD, and efficacious treatment options for individuals with APOL1 risk variants and CKD represent a significant unmet medical need.

Maze employed COMPASS to functionalize human genetic variants to uncover the underlying biology of the target and has designed a small molecule that corrects the effects of toxic gain-of-function variants to potentially enable a therapeutic solution. Maze plans to name the development candidate in early 2022.

ATXN2 Program for Amyotrophic Lateral SclerosisALS is a progressive and fatal neurodegenerative disease with a prevalence of approximately 16,000 patients in the U.S. Current available treatments for ALS primarily focus on providing symptomatic relief and have limited impact on disease progression. A high variability in disease phenotype and life expectancy is observed and believed to be related to the presence of genetic modifiers.

One of Mazes founders, Aaron Gitler, identified a potent genetic modifier, ATXN2, whose inhibition has been shown to limit the toxicity of a certain protein, TDP-43, which is involved in pathologic aggregates seen in up to 97% of all ALS cases. Maze is translating these important insights by developing a novel microRNA gene therapy that targets ATXN2 and has used the proprietary application of its functional genomics tools to optimize its properties. Maze plans to name the development candidate in early 2022.

About Maze TherapeuticsMaze Therapeutics is focused on translating genetic insights into new precision medicines for rare diseases and mechanistically defined subsets of common diseases. Maze has developed the COMPASS platform, a proprietary, purpose-built platform that combines human genetic data, functional genomic tools and data science technology to map novel connections between known genes and their influence on susceptibility, timing of onset and rate of disease progression. Using COMPASS, Maze is building a broad portfolio, including wholly owned programs targeting Pompe disease, chronic kidney disease and amyotrophic lateral sclerosis, as well as partnered programs in cardiovascular and ophthalmic diseases. Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn.

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Maze Therapeutics Reveals Its Initial Three Lead Programs Targeting Underlying Genetic Drivers of Life-Threatening Diseases - Business Wire

Company to use its novel gene editing technology to explore a potential in vivo gene therapy treatment for cystic fibrosis

RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- Life Edit Therapeutics Inc, a next generation gene-editing company, today announced that it has received an award from the Cystic Fibrosis Foundationto identify potential gene editing approaches to treat certain patients with cystic fibrosis (CF). The award will enable Life Edit to screen its library of proprietary base editors for a potential treatment targeting people with CF that are not able to be treated by existing small molecule treatments due to what are known as nonsense genetic mutations (also known as stop mutations). This award is part of the Cystic Fibrosis Foundations Path to a Cure initiative that was launched in October 2019 to address and treat the underlying cause of CF.

Due in large part to the efforts of the CF Foundations support for the development of new medicines, there are now effective therapies available to most people living with the disease, but there remain as many as 7% of patients with CF for whom recent medical advances are not effective, said Mitchell Finer, Ph.D., Chief Executive Officer, Life Edit Therapeutics. Were looking forward to working with the CF Foundation to leverage the unique benefits that our platform offers to develop a highly targeted gene editing approach for these individuals. We believe our science and this approach can be applied across a range of diseases, which will be our focus as we work to build a pipeline of life-changing therapies for severe genetic diseases like CF.

Dr. Allie Crawley, Principal Investigator for the project and member of the Life Edit team, continued by saying, We are thankful to be a part of the Path to a Cure initiative from the CF Foundation which is focused on curing cystic fibrosis by addressing the underlying cause of the disease. We believe our base editor technology has potential to make a great impact in the lives of cystic fibrosis patients with nonsense mutations and are excited about the opportunity to begin early research in this development.

Despite tremendous progress in advancing therapeutics to help people with CF live longer and healthier lives, there remain unmet needs to help all those living with this disease. Approximately 13% of people living with CF have nonsense mutations. These mutations cause the cells to stop the production of the cystic fibrosis transmembrane conductance regulator (CFTR) protein midway through the process, resulting in shortened, non-functional protein.

As part of the $400,000 award from the Foundation, Life Edit will explore its large collection of adenine base editors, or A-base editors, that can potentially be used to correct the six most common, Class I, cystic fibrosis nonsense mutations to restore CFTR function in vivo. A unique feature of the base editors under development by Life Edit is their small size which will allow in vivo delivery with Adeno-associated viruses (AAV) vectors to specific tissue types in the lungs. As part of the agreement, Life Edit will benefit from materials, resources, and expertise from the Cystic Fibrosis Foundation.

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare genetic disease found in more than 30,000 people in the U.S. CF is a hereditary disease that affects the lungs and digestive system that is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that affect the production of the CFTR protein. When the CFTR protein is not made correctly, it affects the balance of salt and fluids inside and outside of the cell. This imbalance leads to thick, sticky mucus in the lungs, pancreas, and other organs. In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. CF is a progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.

About Genome Editing and Life Edit Therapeutics Platform

Genome editing technologies have revolutionized the way cell and gene therapies and regenerative medicines are discovered and developed by allowing genetic material to be removed, added, or altered at specific locations in the genome. While these technologies are in widespread use experimentally, enzymes that offer broader coverage and greater specificity are needed for creating novel cell and gene therapies.

To meet the need for better genome editing approaches, Life Edit Therapeutics has built one of the worlds largest and most diverse arrays of novel RNA-guided nucleases (RGNs) and base editors that are active in mammalian cells. These RGNs were developed using AgBiomes proprietary collection of more than 90,000 microbes and their complete genomes. Life Edit Therapeutics is investigating these proprietary RGNs, which are sourced exclusively from non-pathogenic organisms, to develop new gene editing tools with higher fidelity, novel functionality, reduced immune response risk, and easier delivery. Life Edit Therapeutics nuclease collection also has a broad range of Protospacer Adjacent Motifs (PAMs) short sequences that must follow the targeted DNA sequence in order for the enzyme to make cuts that offer unprecedented access to genomic loci of interest. The Life Edit Therapeutics RGNs offer flexible editing options which encompass knock-out and knock-in capabilities, transcriptional regulation, and base editing when coupled with its proprietary deaminases.

Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. In addition to developing its own pipeline of gene therapies, Life Edit Therapeutics will continue to build its platform of novel nucleases, provide gene editing expertise to strategic partners and ElevateBios portfolio companies, and form other third-party partnerships to discover and develop new therapies.

About Life Edit Therapeutics Inc.

Life Edit Therapeutics is a next-generation gene editing company that has built a highly innovative genome editing platform with one of the worlds largest and most diverse collections of novel RNA-guided nucleases (RGNs) and base editors. Life Edit Therapeutics next generation editing systems will propel the development of novel human therapeutics by enabling ex vivo engineering for cell therapies and regenerative medicines and in vivo delivery of gene therapies. The company is continuing to strengthen the platform, developing a pipeline of in vivo gene therapies to address severe genetic disease, and sharing its expertise through strategic partnership. Life Edit is an ElevateBio portfolio company. For more information visit lifeeditinc.com.

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Life Edit Therapeutics Announces Award from Cystic Fibrosis Foundation - BioSpace

- Initial data from Cohort 1 of the PEARL-1 study shows safety and tolerability of repeat KB301 injections

- Dr. Bhushan Hardas M.D., MBA appointed President, Jeune, Inc.

PITTSBURGH, March 24, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (Krystal) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced the launch of Jeune, Inc., a wholly owned subsidiary of Krystal Biotech, and initial safety data from the ongoing Phase 1 trial of Jeunes lead product candidate, KB301 for treatment of aesthetic skin conditions.

Jeune was formed to advance innovative aesthetic medicines and has an exclusive license to a portfolio of candidates derived from Krystals proprietary technology platform. Jeunes products are designed to directly address biological changes in the skin associated with intrinsic and extrinsic aging. The lead product candidate, KB301, delivers the human COL3A1 gene to increase production of normal type III collagen at the site of administration.

My initial clinical experience with KB301 injections has been highly encouraging, said Dr. Mark Nestor, director of the Center for Clinical and Cosmetic Research and the Center for Cosmetic Enhancement. Not only were the injections well-tolerated, but we see clear signs of new collagen generation which underscores the potential of this treatment to directly address the declining levels of collagen that lead to wrinkles and other skin changes.

Initial data from Cohort 1 in the PEARL-1 studyThe Phase 1, open-label, dose-ranging study is being conducted in adult subjects aged 18-75 (NCT04540900). The primary outcome measure in this first-in-human study was to assess the safety profile of KB301. Secondary outcome measures include COL3A1 transgene expression. In Cohort 1, three different dose levels of KB301 were evaluated in seven (7) healthy subjects who received two intradermal injections into healthy buttock tissue spaced 30 days apart (day 0, day 30). KB301 injected areas were compared to uninjected or saline injected control tissue within the same subject. Treatment and control sites were biopsied at day 2 or day 32. Initial results are as follows:

More detailed data from Cohort 1 will be presented as an e-Poster talk at the Society for Investigative Dermatology (SID) Annual Meeting, held virtually May 3-8.

The presentation will be available on-demand for those registered for the SID conference from May 3, 2021 May 31, 2021. The poster will also be available on the companys website at http://www.jeuneinc.com

The company plans to begin enrollment in the efficacy cohorts of the Phase 1 study in the second half of 2021.

Jeune, Inc. LeadershipJeune has assembled a veteran team of leaders and advisors, comprised of pharmaceutical and biotechnology executives who together have decades of experience developing products in the aesthetic medicine space. Dr. Bhushan Hardas M.D., MBA will join the company on March 29th, 2021 as President of Jeune. Before joining Jeune, Dr. Hardas served as Chief Scientific Officer, Executive Vice President, Global Head of Licensing at Almirall and previously served as Chief Medical Officer of Allergan's Dermatology and Medical Aesthetics business.

I am thrilled to be joining Jeune at such an exciting time. With the ability to deliver genes directly to skin cells, this platform has tremendous potential to address underlying biological changes in aging or photo-damaged skin, noted Dr. Hardas. We are starting with KB301 and type III collagen which I look forward to advancing through the clinic, and the team is already working on pipeline programs that will address additional proteins of interest.

Prior to joining Allergan, Dr. Hardas served as Senior Vice President, Global Head of Dermatology and Aesthetics R&D and Chief Scientific Officer of the North American Business at Merz Pharmaceuticals. Dr. Hardas received advanced training in clinical immunology and molecular biology at King's College at the University of London, in London, England. He also completed a research fellowship in the Department of Dermatology at the University of Michigan, and received his Master of Business Administration degree in healthcare management from the University of California - Irvine.

We are thrilled to welcome Bhushan to Jeune, said Krish S. Krishnan, chairman and chief executive officer of Krystal Biotech. His expertise and development experience in aesthetics is an important asset presently and will help guide next steps for both the pipeline and Jeune overall.

Jeune, Inc. Board Krish Krishnan, Chairman and CEO at Krystal Biotech will serve as the Chairman of the Jeune Board. Joining Mr. Krishnan on the Board are Marc Forth, President and CEO of Aeon BioPharma and Suma Krishnan, Founder and COO of Krystal Biotech.

AboutJeune Inc. Jeune Inc., a subsidiary of Krystal Biotech, is a biotechnology company leveraging a clinically validated gene-delivery platform to fundamentally address and reverse the biology of aging and/or damaged skin. For more information, please visithttp://www.jeuneinc.com

AboutKrystal BiotechKrystal Biotech, Inc.(NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its novel, redosable gene therapy platform and in-house manufacturing capabilities to develop therapies to treat serious rare diseases. For more information, please visit http://www.krystalbio.com.

Forward-Looking StatementsAny statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc., or its subsidiary Jeune, Inc., including but not limited to statements about the development of Krystals and Jeunes product candidates, such as plans for the design, conduct and timelines of ongoing clinical trials of KB301 the clinical utility of KB301, the ability of these candidates to fundamentally address and potentially reverse the biology of aging or damaged skin, plans to pursue research and development of other product candidates; and other statements containing the words anticipate, believe, estimate, expect, intend, may, plan, predict, project, target, potential, likely, will, would, could, should, continue, and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation and conduct of clinical trials, availability and timing of data from clinical trials, whether results of early clinical trials or trials will be indicative of the results of ongoing or future trials, uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including KB301 and such other important factors as are set forth under the caption Risk Factors in Krystals annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystals views as of the date of this release. Krystal anticipates that subsequent events and developments will cause its views to change. However, while Krystal may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing Krystals views as of any date subsequent to the date of this release.

CONTACTS:

Investors:Whitney Ijemwijem@krystalbio.com

Media:Mary CoyleTellMed Strategiesmary.coyle@tmstrat.com

Source: Krystal Biotech, Inc.; Jeune, Inc.

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Krystal Biotech Announces Launch of Jeune, a Gene-Based Aesthetics Company, and Initial Phase 1 Safety Data for KB301 in Aesthetic Indications -...

-- Protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at Day 60, as measured by western blot ---- Mean NSAD score improvement of 5.7 points from baseline was sustained through 24 months in low-dose cohort, and mean NSAD score improvement of 4.0 points from baseline at one year in high-dose cohort ---- Results in both cohorts continue to reinforce the safety and tolerability profile of SRP-9003 --

CAMBRIDGE, Mass., March 18, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc.(NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Companys investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). In the first look at expression data from biopsies taken two years after a single administration of SRP-9003, results found sustained protein expression in muscle tissue. In functional outcomes assessments taken two years following treatment in Cohort 1 (low-dose cohort) and one year after treatment in Cohort 2 (high-dose cohort), patients continued to demonstrate stability in their NSAD (North Star Assessment for Dysferlinopathies) total score and improvements on timed function tests. Results are being presented today at the 2021 Muscular Dystrophy Association (MDA) Annual Clinical and Scientific Conference.

SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan (beta-SG) proteins. SRP-9003 is a gene therapy construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-SG protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

This data is the first look at longer-term expression data with any gene therapy for muscular dystrophy. The meaningful and sustained levels of beta-sarcoglycan protein expression at two years and continued strength of the functional outcomes measured are tremendously positive and support continued advancement of this investigational treatment for patients, said Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics. In Cohort 2, we also saw strong expression of delta-sarcoglycan and gamma-sarcoglycan proteins in addition to beta-sarcoglycan, which suggests that SRP-9003 is working to restore the dystrophin associated protein complex, or DAPC, which provides biological support for the sustained functional benefits observed in both cohorts. LGMD2E is one of the most severe forms of LGMD and causes significant disability in children while frequently leading to early mortality and the data continue to suggest this treatment could bring much needed hope to these patients.

Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed.

Cohort 1 (Dosed at 1.851013 vg/kg), 24 months following treatment:

Cohort 2 (Dosed at 7.411013 vg/kg), 12 months following treatment:

In an exploratory evaluation of all SRP-9003 treated patients compared to a natural history cohort; patients treated with SRP-9003 demonstrated significant improvements in functional outcomes after 24 months. The mean decline in total NSAD score for patients in the natural history cohort was 4.6 points while SRP-9003 treated patients demonstrated a mean improvement of 4.6 points for a clinically meaningful difference of 9.2 points.

Since the last update from this study in October 2020, there have been no new drug-related safety signals observed, and no decreases in platelet counts outside of the normal range and no evidence of clinical complement activation observed in either dose cohort.

About SRP-9003 and the StudySRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.

This open label, first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 1.851013 vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 7.411013 vg/kg based on linear standard qPCR titer method. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Childrens Hospital.

About Limb-girdle Muscular DystrophyLimb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.comor follow us onTwitter,LinkedIn,InstagramandFacebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases; the potential benefits of SRP-9003, including its potential to restore the dystrophin associated protein complex (DAPC); the potential benefits of MHCK7 and the AAVrh74 vector, including its potential to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle; and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

InternetPosting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com.Weencourageinvestorsandpotentialinvestorsto consult our website regularly for important information about us.

Source:Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics' Investigational Gene Therapy SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E Shows Sustained Expression and...

MONTPELLIER, France--(BUSINESS WIRE)--Regulatory News:

Sensorion (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, announces today its full-year 2020 financial results and provides an update on its business activities and outlook for 2021.

We are pleased with the progress made in 2020. We raised 36 million in equity financing to help advance our otoprotective small molecule SENS-401 and our pipeline of promising preclinical gene therapies from our strategic partnership with Institut Pasteur. We expanded our unique technology platform in gene therapy by further building our internal preclinical, process development and analytical capabilities. In Q4 2021, we expect the topline data readout from the Phase 2 trial of SENS-401 to treat sudden sensorineural hearing loss (SSNHL). In H2 2021, we also expect to initiate a clinical trial of SENS-401 to treat cisplatin-induced ototoxicity, a large indication with a significant unmet medical need said Nawal Ouzren, CEO of Sensorion.

Key developments in 2020: science and operational

Gene therapy collaboration with Institut Pasteur on hearing loss

On June 9, 2020, Sensorion announced positive preliminary preclinical data in non-human primates for its gene therapy program targeting Otoferlin deficiency (OTOF-GT), showing that it could deliver an intracellular marker to the inner hair cells at levels that reflect the future clinical requirements. The company plans to discuss its OTOF-GT program with regulatory authorities in H1 2021.

Additionally, Sensorion announced an agreement with Novasep on October 27, 2020, for the process development and manufacturing of adeno-associated viruses (AAV) gene therapy products for the Companys OTOF-GT program.

Technology platform expanded

Sensorion has built a unique R&D technology platform over the years to deepen the understanding of the pathophysiology and etiology of inner ear related diseases. The platform is being actively deployed to select targets, identify biomarkers and to optimize small molecules and gene therapy candidates.

The strengthened platform includes in-vitro assays encompassing the key cochlear cell types, systems to investigate explant tissue and advanced electrophysiological methods for assessing neuronal activity.

On the in-vivo side, Sensorion is developing a suite of validated preclinical models reflecting specific pathologies and inner ear lesions, as well as advancing the use of techniques such as auditory brainstem response (ABR) and distortion product oto-acoustic emission (DPOAE) audiometry for measuring and analysing hearing parameters in those models.

The last pillar of the Companys technology platform focuses on developing biomarkers to improve diagnosis and guide treatment in the key areas of unmet medical need in hearing loss.

Drug candidate SENS-401

Our clinical program with small molecule SENS-401 continued to progress in 2020.

Sensorion is conducting a Phase 2 clinical trial of SENS-401 in the treatment of SSNHL in adults. This Phase 2, randomized, double-blinded, placebo-controlled study is being conducted in multiple countries in Europe and Canada.

On February 17, 2020, Sensorion received Ethics Committee approval to include new military sites in this study, allowing clinical investigators to recruit volunteer military personnel who have suffered from acute hearing loss.

On March 13, 2020, Sensorion provided an update on the recruitment schedule for the ongoing SENS-401 Phase 2 study for the treatment of SSNHL. Due in part to the impact of COVID-19 on patient enrolment, the availability of topline data were expected to be delayed to Q4 2021 (see below in 2021 announcements).

On June 5, 2020, the independent Data Safety Monitoring Board (DSMB) confirmed the absence of safety concerns and recommended continuation of the Phase 2 trial as scheduled.

Having demonstrated otoprotective activity in several preclinical models, SENS-401 is being studied as a potential option to preserve residual hearing in people with cochlear implants under a collaboration with Cochlear, the world leader in implantable hearing solutions. Positive preclinical data were announced on January 5, 2021 (see below in 2021 announcements). Next steps are being discussed with Cochlear.

On December 15, 2020, Sensorion and Sonova Holding AG, a leading provider of hearing solutions, announced that Sonova had acquired a 3.7% equity stake in Sensorion via an investment of 5 million. At the same time, the two companies signed a letter of intent to exclusively explore potential plans for a strategic collaboration in the field of innovative diagnostic and therapeutic solutions for certain types of hearing loss. Discussions between Sensorion and Sonova are ongoing.

Strengthened scientific and medical leadership

As part of Sensorions strategic move into gene therapy for hearing restoration, on February 19, 2020 the Company announced the appointment of Dr. Graldine Honnet as Chief Medical Officer. Dr. Honnet has extensive expertise both in gene therapy and small molecule clinical development across numerous disease areas.

During 2020, Sensorion also increased its R&D headcount by 30% strengthening its team with experienced scientists and researchers in gene therapy, the inner ear and neurosciences. In addition to Dr Honnet, Sensorion also appointed a preclinical head in gene therapy and a new CMC gene therapy lead with more than 20 years of experience in the field.

On July 29, 2020, Sensorion announced the appointment of five distinguished experts to its Scientific Advisory Board (SAB); Prof. Alain Fischer, Dr. Robert Dow, Prof. Paul Avan, Dr. Diane Lazard and Dr. Hernn Lpez-Schier. The SAB is chaired by Prof. Christine Petit, Founding Director of the French Hearing Institute and a world-renowned geneticist and neurobiologist in hearing and hearing disorders.

Scientific communications

Sensorion presented at various scientific congresses and hosted two Key Opinion Leaders (KOLs) calls in 2020, including:

Governance: appointment of a new Chairman of the Board of Directors

On July 6, 2020, Sensorions Board of Directors was strengthened by the appointment of Edwin Moses as Chairman of the Board of Directors. Dr. Moses has more than 25 years of executive experience as both CEO and Chairman of numerous life science companies, including Ablynx, where he led its rapid growth from a small research-focused organization to one of Europes leading biotechnology companies, prior to its $4.8 billion acquisition by Sanofi in 2018.

2021 announcements

Since the end of the fiscal year, the key business updates are as follows:

On January 5, 2021, Sensorion provided an update on plans and progress made in the development of SENS-401 for the prevention of hearing loss. After encouraging efficacy data in preclinical models, the Company expects to begin a proof-of-concept clinical trial with SENS-401 to treat patients suffering from cisplatin-induced ototoxicity (CIO) in the second half of 2021. A natural history study of CIO in adult cancer patients is expected to start in the first half of 2021. Successful clinical findings would significantly expand Sensorions target market for SENS-401: approximately 500,000 cancer patients are treated annually with cisplatin in the USA and EU5, a significant proportion of whom will experience severe hearing loss.

At that time, Sensorion also announced a delay in the availability of the topline results from the Phase 2 study of SENS-401 in SSNHL to Q4 2021, due to the impact of COVID-19. Additionally, Sensorion indicated that it planned to review the study design and consider opportunities to aid its successful on-time completion. Following this review, an amendment to the statistical analysis plan (SAP), which would significantly reduce the sample size without compromising on the quality and potential outcome of the trial, has been submitted to the regulatory authorities. The responses from regulators so far have been encouraging and this increases the Companys confidence in being able to generate topline data this year.

On January 19, 2021, Sensorion announced positive preclinical data demonstrating the potential of SENS-401 to preserve residual hearing after cochlear implantation, in a collaboration with Cochlear, the global leader in implantable hearing solutions. Cochlear implants are very effective in treating severe to profound hearing loss, but preserving acoustic hearing in patients with residual hearing who receive cochlear implants could provide substantial benefit. Sensorion and Cochlear are making progress in the discussion around potential clinical study designs. Next steps are being discussed with Cochlear.

On February 15, 2021, Sensorion announced a third gene therapy collaboration with Institut Pasteur around the GJB2 gene targeting important pediatric and adult deafness markets. GJB2 mutations had already been widely recognized as the most prevalent cause of congenital deafness and now, new findings from Institut Pasteur have now demonstrated that GBJ2 mutations also underlie a wide range of other instances of severe hearing loss in the adult population. Sensorion will pursue three initial GBJ2-related indications: congenital deafness, progressive childhood hearing loss and early onset of severe presbycusis in adults.

2021 strategy and prospects

As of 31st December 2020, the Company had 62 million in cash, boosted by a successful 31 million Reserved Offering conducted in September and the December investment of 5 million by Sonova. Sensorion intends to use the new funds to develop its current gene therapy programs (OTOF-GT, GJB2-GT and USHER-GT), to support its pharmacology and clinical studies of SENS-401 and for general corporate purposes.

Nawal Ouzren, CEO of Sensorion commented: We are working hard to secure further approvals of the protocol amendment to reduce the sample size for the SENS-401 Phase 2 SSNHL trial which will help ensure that we could complete this study on time. Sensorion will also engage with regulatory authorities in Europe and the US to discuss our potential Otorferlin gene therapy clinical trial design. We recently added a promising third program to the Sensorion gene therapy pipeline centered around the GJB2 gene, the most prevalent cause of congenital deafness. This program will focus on major new markets with an estimated patient population of 300,000 children and adults in Europe and the United States. We remain very focused on delivering on our ambitious goals this year and we will stay true to our vision to serve patients with hearing loss.

Expected future milestones and estimated timelines:

2020 financial results

The annual accounts at 31 December 2020, drawn up according to IFRS standards and approved by the Board of Directors on 17 March 2021, have been duly reviewed by statutory auditors.

The simplified income statement as of 31 December 2020 is as follows:

In Euros IFRS standards

31.12.2020

31.12.2019

Operating income

2,421,267

2,522,717

Research & Development expenses

-7,679,365

-10,208,520

General & Administrative expenses

-3,631,123

-3,128,236

Total operating expenses

-11,310,488

-13,336,756

Operating profit/loss

-8,889,220

-10,814,039

Financial charges

-88,869

-1,282,141

Net profit/loss

-8,978,089

-12,096,181

For the year ended 31st December 2020, Sensorion reported operating income of 2.4 million, which included 1.8 million in research tax credit and 0.6 million in grants from Audinnove (RHU) and Patriot (PSPC) collaborations. The decrease of -0.1 million compared to 2019 is explained by a decrease of -0.7 million in research tax credit partly offset by an increase of +0.6 million in grants.

Operating expenses declined by 15% from 13.3 million in 2019 to 11.3 million for fiscal year 2020.

R&D expenses decreased by 2.5 million mainly due to the completion of the SENS-111 clinical trial in 2019 and a slowdown of SENS-401 activities linked to the global Covid-19 pandemic situation, and partly offset by an increase in headcounts to strengthen the R&D team with Gene Therapy expertise.

G&A expenses increased by 0.5 million, mainly driven by an increase in headcount.

Operating loss at 31 December 2020 was -8.9 million compared with -10.8 million at 31 December 2019.

The net financial charges decreased by 1.3 million compared to 2019 which was mainly due to the costs related to the convertible bond operations.

Net loss was -9.0 million at 31 December 2020 compared with -12.1 million at 31 December 2019.

As of 31 December 2020, the company employed 28 persons.

Financial structure

The simplified balance sheet at 31 December 2020 is as follows:

In Euros IFRS standards

31.12.2020

31.12.2019

Non-current Assets

1,474,117

1,724,348

Other Current Assets

4,254,909

5,946,864

Cash & cash equivalent

62,174,948

30,428,319

Total Assets

67,903,976

38,099,532

Equity

58,379,653

13,218,525

Non-current Liabilities

5,246,408

2,036,933

Current Liabilities

4,277,915

22,844,074

Total Liabilities

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Sensorion Reports Full-Year 2020 Financial Results and Provides Business Highlights - Business Wire

NEW YORK, March 24, 2021 /PRNewswire/ --Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) ("Actinium" or the "Company") today announced that its collaborator, Memorial Sloan Kettering Cancer Center ("MSK"), has commenced patient enrollment in the Phase 1 study evaluating Iomab-ACT for targeted conditioning prior to treatment with MSK's CD19 targeted CAR T-cell 19-28z. Iomab-ACT is a low dose version of Actinium's Phase 3 drug candidate Iomab-B, a CD45 targeting antibody radiation conjugate ("ARC"). Actinium and MSK were jointly awarded National Institutes of Health Small Business Technology Transfer grant funding for this first ever trial to evaluate ARC-based targeted conditioning prior to CAR-T therapy. The scientific rationale for this trial builds on preclinical data published in 2020 and is further supported by clinical observations from the SIERRA trial to justify combining MSK's 19-28z CAR T-cell therapy with Iomab-ACT. Manufacturing of patient CAR T-cells has commenced and patient conditioning with Iomab-ACT followed by 19-28z CAR T-cell infusion is expected early in the second quarter of 2021, with proof-of-concept data expected in the second half of 2021.

Results of a Phase 2 trial in 53 patients with relapsed and refractory B-cell acute lymphoblastic leukemia with MSK's 19-28z CAR-T published in the New England Journal of Medicine reported complete remissions in 83% (44/53) of patients, median event-free survival (EFS) of 6.1 months and median overall survival (OS) was 12.9 months at a median follow up period of 29 months (range 1 65 months). There was a 26% (14/53) rate of Grade 3 of greater cytokine release syndrome (CRS), with 1 patient death as a result, and 42% of patients experienced Grade 3-4 immune effector cell-associated neurotoxicity syndrome (ICANS).

Dr. Dale Ludwig, Actinium's Chief Scientific and Technology Officer, said "MSK's 19-28z CAR T-cell therapy has produced high response rates in patients with relapsed or refractory B-ALL who have previously undergone several lines of standard therapy. However, toxicities such as cytokine release syndrome and neurologic toxicity, as well as durability of response, remain a significant challenge. The ARC technology Iomab-ACT employs enables the delivery of targeted radiation that selective and specifically targets immune cells including those implicated in the CAR-T-associated toxicities of neurotoxicity and cytokine release syndrome. We are eager to begin treating patients in this first of its kind pilot study to explore the potential of Iomab-ACT targeted lymphodepletion to modulate the immune system and improve the safety profile of CAR T-cell therapy. We are hopeful this technology may ultimately enhance our ability to deliver CAR T-cell therapies more safely. Positive results from pilot study could have a meaningful impact on the way we condition patients for CAR-T and other adoptive cell therapies, which have transformed the treatment of patients with blood cancers."

Sandesh Seth, Actinium's Chairman and CEO, said "This is a major milestone for Actinium and one we are very excited by. Adoptive cell therapies like CAR-T and gene therapies have emerged as some of the most promising areas in medicine and hold tremendous promise for patients, many of whom have limited or no treatment options remaining. This promise has led to a large and growing field of therapies where we intend to establish Iomab-ACT as the universal solution for targeted, non-chemotherapy conditioning, that harnesses the power of radiation. With Iomab-B nearing complete enrollment in the Phase 3 SIERRA trial for bone marrow transplant conditioning, starting to treat patients in this Iomab-ACT trial for CAR-T conditioning could not come at a better time. We look forward to continuing to build out our strategic business unit in targeted conditioning to best serve patients seeking potentially curative bone marrow transplant, adoptive cell therapy and gene therapy to improve patient access and outcomes."

About Iomab-ACT

Iomab-ACT targets cells that express CD45, an antigen found on immune cells such as lymphocytes and macrophages as well as leukemia and lymphoma cancer cells and delivers the radioisotope warhead iodine-131 to achieve cell depletion. Iomab-ACT is intended to deplete CD45+ immune cells such as macrophages that are implicated in CAR-T related toxicities and may also have an anti-tumor effect on chemo-refractory cancers. Iomab-ACT is a low dose extension of Actinium's lead program, Iomab-B, which is being studied in a pivotal Phase 3 trial for targeted conditioning prior to a bone marrow transplant. Preclinical data supporting Iomab-ACT's application in targeted lymphodepletion prior to ACT such as CAR-T was recently published in the journalOncotarget(https://www.oncotarget.com/archive/v11/i39/).

In addition, clinical data with trace doses of Iomab-B has shown transient, reversible lymphodepletion in patients and drug clearance pharmacokinetics that fit within the vein-to-vein time of CAR-T manufacturing and administration.

About Actinium Pharmaceuticals, Inc. (NYSE: ATNM)

Actinium Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing ARCs or Antibody Radiation-Conjugates, which combine the targeting ability of antibodies with the cell killing ability of radiation. Actinium's lead application for our ARCs is targeted conditioning, which is intended to selectively deplete a patient's disease or cancer cells and certain immune cells prior to a BMT or Bone Marrow Transplant, Gene Therapy or Adoptive Cell Therapy (ACT) such as CAR-T to enable engraftment of these transplanted cells with minimal toxicities. With our ARC approach, we seek to improve patient outcomes and access to these potentially curative treatments by eliminating or reducing the non-targeted chemotherapy that is used for conditioning in standard practice currently. Our lead product candidate, I-131 apamistamab (Iomab-B) is being studied in the ongoing pivotal Phase 3 Study of Iomab-B in Elderly Relapsed or Refractory Acute Myeloid Leukemia (SIERRA) trial for BMT conditioning. The SIERRA trial is over seventy-five percent enrolled and positive single-agent, feasibility and safety data has been highlighted at ASH, TCT, ASCO and SOHO annual meetings. Iomab-ACT (low dose I-131 apamistamab) is also be studied as a targeted conditioning agent in a Phase 1 study with a CD19 CAR T-cell Therapy with Memorial Sloan Kettering Cancer Center and is intended to be studied for conditioning prior to gene therapy. In addition, we are developing a multi-disease, multi-target pipeline of clinical-stage ARCs targeting the antigens CD45 and CD33 for targeted conditioning and as a therapeutic either in combination with other therapeutic modalities or as a single agent for patients with a broad range of hematologic malignancies including acute myeloid leukemia, myelodysplastic syndrome and multiple myeloma. Ongoing combination trials include our CD33 alpha ARC, Actimab-A, in combination with the salvage chemotherapy CLAG-M and the Bcl-2 targeted therapy venetoclax. Underpinning our clinical programs is our proprietary AWE (Antibody Warhead Enabling) technology platform. This is where our intellectual property portfolio of over 140 patents, know-how, collective research and expertise in the field are being leveraged to construct and study novel ARCs and ARC combinations to bolster our pipeline for strategic purposes. Our AWE technology platform is currently being utilized in a collaborative research partnership with Astellas Pharma, Inc. Website:https://www.actiniumpharma.com/

Forward-Looking Statements for Actinium Pharmaceuticals, Inc.

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Contacts:

Hans Vitzthum LifeSci Advisors, LLC[emailprotected](617) 430-7578

SOURCE Actinium Pharmaceuticals, Inc.

http://www.actiniumpharma.com/

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Actinium Announces Initiation of Patient Enrollment in Iomab-ACT Trial for Targeted Conditioning Prior to CD19 CAR T-Cell Therapy - PRNewswire

BOONE, Iowa, March 19, 2021 /PRNewswire/ -- Gene therapy company ENDSULIN has expanded to a second study site at Boone Veterinary Hospital, where it is looking for diabetic dogs to participate in a study to evaluate the effectiveness of a treatment that could reduce or even eliminate insulin injections.

The study could be a critical step in advancing a one-time gene therapy treatment for diabetes in both dogs and humans. The treatment, which has proven efficacy in hundreds of small animals, is based on more than 25 years of research at the University of WisconsinHospitals and Clinics in Madison, where the company is based.

"We hope to free families who are caring for their diabetic pets around the clock," said Hans Sollinger, ENDSULIN founder. "Giving dogs and their families their independence back is a step in our mission to do the same for millions of people suffering with diabetes."

ENDSULIN covers the cost of the procedure, which takes about 30 minutes total and is administered by a certified veterinarian. After treatment, the ENDSULIN team will periodically monitor dogs' health to observe the long-term effects.

While this particular gene therapy is novel, dogs treated with gene therapies in Barcelona, Spain have been followed for up to 8 years, with no evidence of adverse events.

Ideal dogs are small, and have been recently diagnosed with diabetes. Families must be able to bring their pet to either the Boone or Waunakee, WI, clinics for the one-time treatment and five follow-up visits, and also provide follow-up information to the ENDSULIN research team.

People with diabetic dogs can learn more about the study or ask about enrollment at endsulin.com/pilot-study, or contact ENDSULIN directly at [emailprotected].

ABOUT ENDSULINENDSULIN is reshaping the way we approach a cure for diabetes. They are working to free patients from daily injections and 24/7 management using the most cutting-edge gene therapy technology, developed from decades of research by noted diabetes leader Hans Sollinger, MD, PhD, Dr hc, at the University of Wisconsin Hospitals and Clinics. Their sole focus is to get a durable, one-time treatment to the millions of people who need it.

SOURCE ENDSULIN

https://www.endsulin.com

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Diabetic Dogs Needed for Study that Could Reduce or Eliminate Insulin Injections - PRNewswire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--ElevateBio, a cell and gene therapy technology company focused on powering transformative cell and gene therapies, today announced that the companys Chief Scientific Officer of Regenerative Medicine, Dr. Melissa Carpenter, has been appointed to the International Society for Stem Cell Research (ISSCR) Board of Directors. In this role, Dr. Carpenter will work with the ISSCR officers and board to advance the organizations mission of bringing together researchers, clinicians, academics, and industry to promote excellence in stem cell science and applications to human health.

I am honored to have been elected to, and serve on the Board of, the ISSCR and foster the continued progress in advancing stem cell science alongside this impressive leadership and fellow board members, said Melissa Carpenter, PhD, Chief Scientific Officer of Regenerative Medicine at ElevateBio. Collaboration across the stem cell professional community is critical to our ability to translate promising stem cell research and regenerative medicine science into treatments that can have dramatic benefit for global human health globally.

Dr. Carpenter served on the ISSCR Task Force to revise the Guidelines for Stem Cell Research and Clinical Translation that will be released in May and advocated in support of the value of stem cell research as part of the Societys 2019 Advocacy Day, meeting with members of the U.S. Congress. She has also served on the Clinical Translation Committee.

We are delighted to welcome Melissa Carpenter to the ISSCR Board of Directors, said Christine Mummery, ISSCR President. Melissas dedication to supporting the translation of stem cell discoveries into therapeutics and her leadership has been crucial for advancing the clinical development of multiple therapies. Her experience will be an asset to the Board as the field of stem cell science continues to rapidly evolve.

The International Society for Stem Cell Research is the preeminent global, cross-disciplinary, science-based organization dedicated to stem cell research and its translation to the clinic. With nearly 4,000 members from more than 60 countries, the ISSCR mission is to promote excellence in stem cell science and applications to human health.

About ElevateBio:

ElevateBio is a cell and gene therapy technology company built to power the development of transformative cell and gene therapies today and for many decades to come. The company has assembled industry-leading talent, built world-class facilities, and integrated diverse technology platforms necessary for rapid innovation and commercialization of cell, gene, and regenerative therapies. The company has built an initial technology stack, including gene editing, induced pluripotent stem cells, and protein, viral, and cellular engineering. At the center of the business model is ElevateBio BaseCamp, a centralized R&D and manufacturing company that offers research and development (R&D), process development (PD), and Current Good Manufacturing Practice (CGMP) manufacturing capabilities. The company is focused on increasing long-term collaborations with industry partners while also continuing to develop its own highly innovative cell and gene therapies. ElevateBio's team of scientists, drug developers, and company builders are redefining what it means to be a technology company in the world of drug development, blurring the line between technology and healthcare.

ElevateBio is headquartered in Cambridge, Mass, with ElevateBio BaseCamp located in Waltham, Mass. For more information, visit us at http://www.elevate.bio, or follow Elevate on LinkedIn, Twitter, or Instagram.

*As of the date of this press release, SoftBank Group Corp. has made capital contributions to allow investments by SoftBank Vision Fund 2 ("SVF 2") in certain portfolio companies. The information included herein is made for informational purposes only and does not constitute an offer to sell or a solicitation of an offer to buy limited partnership interests in any fund, including SVF 2. SVF 2 has yet to have an external close, and any potential third-party investors shall receive additional information related to any SVF 2 investments prior to closing.

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ElevateBio Announces Chief Scientific Officer of Regenerative Medicine, Melissa Carpenter, PhD, Elected to the International Society for Stem Cell...

Dive Brief:

Sarepta has three marketed products treating Duchenne, plus a beefy pipeline of 39 experimental RNA-based programs and genetic medicines for rare diseases. One towers above them all, however: SRP-9001. Its clinical trial setback in January cut the company's valuation in half as a rival treatment from Pfizer appeared to be taking a lead.

With the path to market looking longer for SRP-9001, other pipeline projects may figure more prominently in Sarepta's outlook, and the data from the follow-up project, known as SRP-9003, may be reassuring to investors.

SRP-9003 treats a type of muscular dystrophy called Limb-girdle that particularly affects the arms and legs. To do so, it helps replenish the deficiency of a protein called beta-sarcoglycan, the lack of which is thought to trigger the disease.

The first three patients in Sarepta's early-stage trial were infused with a low dose of the gene therapy, and after 18 months of treatment had improved their score by 5.7 points on a 20-item test that measures their ability to do such tasks as stand up from a chair or stand briefly on one foot. That improvement was sustained at two years, Sarepta reported Thursday.

In a research note, SVB Leerink's Schwartz wrote that untreated patients would have been expected to decline by 4.6 points over the same time period. Biological measures, such as expression of the beta-sarcoglycan protein, also showed positive signs, although they were short of expression seen in the healthy population.

Three higher-dose patients also showed improvements at one year. Their four-point increase on the functional test was short of the six points seen in the lower-dose group at 12 months, although Schwartz noted that biomarkers indicated the high-dose group may have been less disabled when they received the gene therapy.

The company hasn't seen any new safety signals. One adverse event was reported in an earlier data release, a patient who was dehydrated from vomiting.

Schwartz wrote that the data could set the stage for a study that could be submitted to regulators to support approval. There are no treatments for limb-girdle muscular dystrophy, so a regulatory pathway will need to be discussed with the Food and Drug Administration, as well as a quality assessment of the batch of gene therapy to be used in that trial. That could make the path forward for limb-girdle gene therapy different than the one Sarepta's traversing in Duchenne, where there are at least some marketed drugs for the condition.

The next study should begin this year, Schwartz wrote.

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With new results, Sarepta's 2nd gene therapy holds steady - BioPharma Dive

GERMANTOWN, Md., March 18, 2021 (GLOBE NEWSWIRE) -- Orgenesis Inc. (NASDAQ: ORGS) (Orgenesis or the Company), a global biotech company working to unlock the full potential of celland gene therapies, announces that it has entered a collaboration with MIDA Biotech B.V. (MIDA). Together, the companies will work to establish point-of-care centers at hospitals and other medical institutions across western Europe.

Orgenesis and MIDA plan to deploy Orgenesis Mobile Processing Units and Labs (OMPULs) at leading hospitals for the onsite development of promising cell and gene therapies and immunotherapies from MIDA. The OMPULs are multi-purpose, mobile, autonomous good manufacturing practice (GMP) facilities used to develop, optimize, and manufacture cell and gene therapies at the point of care. Via the collaboration, the teams will align with various hospitals with projects focused on scaling the therapies through to commercialization with regulatory compliance and governmental approval standards as guiding principles.

In connection with the Agreement, Orgenesis and MIDA entered into agreements to establish point-of-care centers and deploy OMPULs within leading hospitals in Italy, Germany, Spain and Benelux, as well as other activities including joint research, development and validation activities related to the development of cell and gene therapies.

These agreements mark a major milestone towards the commercial launch of our OMPULs across Europe, and we believe provide further validation of our platform, as each of the respective hospitals conducted extensive due diligence and verification around our technology and capabilities prior to signing the agreements, stated Vered Caplan, CEO of Orgenesis. We see MIDA as an ideal partner based on their established relationships with the leading medical centers across western Europe and their promising science. Our teams are aligned in the desire to help lower costs and eliminate the logistical nightmares of building a centralized production facility or cleanrooms in the hospitals, which can stand in the way of getting a therapy to market effectively.

The additional sites will significantly expand the Orgenesis POCare Network capacity, which already includes active development centers in the United States, Belgium, Israel, and South Korea, as well as a growing number of joint venture agreements with regional partners and ongoing therapeutic development programs.

About OrgenesisOrgenesis is a global biotech company working to unlock the full potential of celland gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis POCarePlatform is comprised of three enabling components: a pipeline of licensedPOCare Therapeuticsthat are processed and produced in closed, automatedPOCare Technologysystems across a collaborativePOCare Network. Orgenesisidentifies promising new therapies and leverages its POCare Platform to provide a rapid, globally harmonized pathway for these therapies to reach and treat large numbers of patients at lowered costs through efficient, scalable, and decentralized production. The POCare Network brings together patients, doctors, industry partners, research institutes and hospitals worldwide to achieve harmonized, regulated clinical development and production of the therapies. Learn more about the work Orgenesis is doing atwww.orgenesis.com.

Notice Regarding Forward-Looking StatementsThis press release contains forward-looking statements which 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 and Exchange Act of 1934, as amended. These forward-looking statements involve substantial uncertainties and risks and are based upon our current expectations, estimates and projections and reflect our beliefs and assumptions based upon information available to us at the date of this release. We caution readers that forward-looking statements are predictions based on our current expectations about future events. These forward-looking statements are not guarantees of future performance and are subject to risks, uncertainties and assumptions that are difficult to predict. Our actual results, performance or achievements could differ materially from those expressed or implied by the forward-looking statements as a result of a number of factors, including, but not limited to, our reliance on, and our ability to grow, our point-of-care cell therapy platform, our ability to achieve and maintain overall profitability, our ability to manage our research and development programs that are based on novel technologies, our ability to control key elements relating to the development and commercialization of therapeutic product candidates with third parties, the timing of completion of clinical trials and studies, the availability of additional data, outcomes of clinical trials of our product candidates, the potential uses and benefits of our product candidates, our ability to manage potential disruptions as a result of the coronavirus outbreak, the sufficiency of working capital to realize our business plans, the development of our POCare strategy, our trans differentiation technology as therapeutic treatment for diabetes, the technology behind our in-licensed ATMPs not functioning as expected, our ability to further our CGT development projects, either directly or through our JV partner agreements, and to fulfill our obligations under such agreements, our license agreements with other institutions, our ability to retain key employees, our competitors developing better or cheaper alternatives to our products and the risks and uncertainties discussed under the heading "RISK FACTORS" in Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31, 2020, and in our other filings with the Securities and Exchange Commission. We undertake no obligation to revise or update any forward-looking statement for any reason.

Contact for Orgenesis:Crescendo Communications, LLCTel: 212-671-1021Orgs@crescendo-ir.com

Excerpt from:
Orgenesis Enters into Collaboration Agreement with MIDA to Deploy OMPULs for Point-of-Care Research and Development of Promising Cell and Gene...

SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting

Paris, March 19, 2021 SparingVision (the Company), a genomic medicine company developing vision saving treatments for ocular diseases, announces today that three abstracts highlighting the companys recent research into ocular diseases and its lead gene therapy treatment SPVN06 have been accepted for the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting, to be held virtually from 1-7 May. The three abstracts will be given as poster presentations for which the details can be found below.

Title: SPVN06, a Novel Mutation-Independent AAV-based Gene Therapy, Protects Cone Degeneration in a Pig Model of Retinitis PigmentosaDate and Time: May 3, 2021 from 11:15 AM to 1:00 PM EDT

Presenter: Dr. Jennifer Noel, University of LouisvilleSession Title: Drug delivery and Gene Therapy

Title: Correlations between progression markers in rod-cone dystrophy due to mutations in RHO, PDE6A, or PDE6BDate and Time: May 3, 2021 from 4:30 PM to 6:15 PM EDT

Presenter: Dr. Daniel Chung, Chief Medical Officer, SparingVisionSession Title: Visual Impairment - Assessment and Measurement

Title: A 1-Month Toxicology and Biodistribution NHP Pilot Study Evaluating a Single Subretinal Bilateral Administration of SPVN06 - A Novel AAV-Based Gene Therapy for the Treatment of Rod-Cone Dystrophies Agnostic of the Causative Mutation Date and Time: May 5, 2021 from 2:45 PM to 4:30 PM EDT

Presenter: Dr. Melanie Marie, SparingVisionSession Title: AMD and retinal physiology

**ENDS**

Contacts:

NOTES TO EDITORS:

About SparingVision:SparingVision is a genomic medicines company, translating pioneering science into vision saving treatments. Founded to advance over 20 years of world-leading ophthalmic research from its scientific founders, SparingVision is leading a step shift in how ocular diseases are treated, moving beyond single gene correction therapies. At the heart of this is SPVN06, a gene independent treatment for retinitis pigmentosa (RP), the most common inherited retinal disease affecting two million people worldwide. SPVN06 could form the basis of a suite of new sight saving treatments as it could be applicable to many other retinal diseases, regardless of genetic cause.

The Company is supported by a strong, internationally renowned team who aim to harness the potential of genomic medicine to deliver new treatments to all ocular disease patients as quickly as possible. SparingVision has raised 60 million to date and its investors include 4BIO Capital, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, UPMC Enterprises, Jeito Capital and Ysios Capital. For more information, please visit http://www.sparingvision.com.

About SPVN06:SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors, which inevitably leads to blindness in patients with rod-cone dystrophies (RCD). SparingVisions primary disease target is Retinitis Pigmentosa (RP), one of the most common inherited retinal diseases that affects two million patients worldwide. There is currently no treatment approved to treat RP patients independently of their genetic background. This approach is potentially applicable to many more diseases where the loss of rods is known to be an early signal of the disease. First-in-man trials, with SPVN06 in patients with RP, will be commencing in H2 2021.

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SparingVision Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2021 Annual Meeting - GlobeNewswire

24 Mar 2021

FPIs raison detre is to get effective, international clinical trials on the road (see Part 6of this series). At ICFTD, FPIs co-leader, Adam Boxer of the University of California, San Francisco, summarized what trials exist already, and offered a glimpse of the future.

Currently, the field lacks biomarkers for the underlying neuropathology of FTD, so there is no concrete way to know whether tau fibrils or TDP-43 inclusions lurk within the brain of a patient who has no known autosomal-dominant mutation. Hence, present-day FTD trials are limited to carriers of pathogenic C9ORF72, GRN, or MAPT mutations, and to people whose clinical syndromes are known to be tauopathies, such as progressive supranuclear palsy.

Trials for carriers of GRN mutations are the farthest along. At centers that are part of FPI, Alector is conducting Phase 2 and Phase 3 trials evaluating AL001, an anti-sortilin antibody meant to slow progranulins degradation. Early trials of AL001 indicated that it restores CSF progranulin levels in mutation carriers. The Phase 3 trial is the first in the field to include presymptomatic mutation carriers. They can enroll if their plasma NfL is elevated, suggesting they are nearing symptom onset. The trial aims for 180 participants total, and also includes symptomatic carriers. It uses the CDR-NACC-FTLD as a clinical primary endpoint, and is slated to run through 2023.

Two companiesPrevail Therapeutics (recently acquired by Eli Lilly & Company) and Passage Bioare pursuing a gene-therapy approach. Both are hoping to replenish progranulin levels by injecting an adeno-associated virus bearing the progranulin gene directly into the cisterna magna of GRN mutation carriers with FTD. Prevails trial of PR006started in July 2020, while Passage Bios trial of PBFT02 is slated to start this month.

Julio Rojas of the University of California, San Francisco, said the combination of sensitive disease biomarkers (CSF progranulin, plasma NfL, and others) and progranulin-targeted treatment in trials bodes well for this form of familial FTD. Its likely well see a treatment for GRN mutation carriers with FTD before we see one for AD, Rojas said.

Carriers of hexanucleotide expansions in the C9ORF72 gene are also being included within the progranulin umbrella. Owing to the lysosomal dysfunction wrought in both familial forms of the disease, C9ORF72 carriers are included in Alectors Phase 2 study of AL001.

Other trials are targeting the C9ORF72 mutation specifically. They enroll people with ALS and/or FTD. The furthest along is Ionis/Biogens antisense oligonucleotide BIIB078, in Phase 1. The multiple-ascending-dose trial includes 114 people with ALS; results are expected later this year. Participants in the randomized portion of the trial are being enrolled in a two-year, open-label extension. WaveLife Sciences, Cambridge, Massachusetts, is planning to start a Phase 1 trial of its C9-ASO, called WVE-004, in people with ALS and FTD due to C9ORF72 expansion this year (see press release).

Yet another C9-ASO, called afinersen, was designed in Robert Browns group at the University of Massachusetts in Worcester. At ICFTD, Boxer showed data from a man with ALS who was treated with afinersen for over a year. During this time, his CSF levels of poly-GP, a dipeptide translated from transcripts of the hexanucleotide expansion, plummeted, and his disease stayed stable during treatment.

The diabetes drug metformin is being tested at the University of Florida, Gainesville, in a Phase 1 clinical trial for C9ORF72 hexanucleotide expansion carriers with ALS or FTD. Besides its better-known effects on glucose levels and insulin sensitivity, metformin has been reported to squelch repeat-associated non-AUG (RAN) translation, the mechanism responsible for translating toxic dipeptides from the C9 repeats (Zu et al., 2020). This open-label study includes 18 participants, and is expected to finish in August 2022.

What about trials for FTLD-tau? So far, most trials have focused on people with clinical syndromes, such as progressive supranuclear palsy, which is known to have underlying tau pathology, but not necessarily a pathogenic tau mutation. Two different anti-tau monoclonal antibodies failed in trials for PSP last year (Jul 2019 news; Dec 2019 news). This prompted cancellation of a basket trial that had also included people with non-fluent primary progressive aphasia, corticobasal syndrome, and MAPT mutation carriers with FTD.

Why did these trials fail? A definitive answer is not in, but Boxer noted that CSF studies from the trials indicated that both antibodies, which target taus N-terminus, did not rid the brain of all forms of tau pathology. Boxer said the field eagerly awaits results of Biogens trial of BIIB080, a tau-targeting ASO in people with mild AD, which could provide proof-of-concept data for primary tauopathies. That Phase 2 trial will finish in May of 2022.

One hurdle to treating primary tauopathies is the lack of fluid biomarkers or PET imaging tracers for many non-AD forms of tau. While some tracers have been found to bind to the 4R-tauopathies PSP and CBD, so far none reliably tag tau in people with bvFTD or PPA, who could have underlying tau or TDP-43 pathology (Jul 2020 news). Once the field develops a marker for either one, people with sporadic FTD will be able to join clinical trials targeting their specific pathology.

MAPT mutations are the rarest cause of familial FTD, i.e., patients with these mutations are scarce. To ensure that trials in this far-flung group of patients are run with the most power possible, the FPI is working to coordinate international platform trials for them, which will test multiple therapies simultaneously against a shared placebo group, Boxer said.Jessica Shugart

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FTD Trials: The Now and the Future | ALZFORUM - Alzforum

Guest editorial: Technology to meet the challenge of efficiency in the regulated AAV vector space

In this guest editorial, based on a blog post by Gyros Protein Technologies, learn how scientists at AstraZeneca developed a reliable method for adeno-associated virus (AAV) capsid titer quantification.

Vectors based on AAV are used widely in gene therapy for in vivo gene delivery. The success of AAV-based therapeutic production depends on the availability of laboratory tools that can efficiently deliver reliable data, including the determination of virus particle titer (physical titer). To achieve this, a team at AstraZeneca searched for an immunoassay platform that could improve on ELISA in terms of dynamic range, sample volume, and productivity. Its evaluation showed that Gyrolab systemmet its needs for AAV process development, with a more reliable measurement of AAV capsid titer for in-process and purified samples.

Gene therapy has seen a massive explosion in activity since the first clinical trial in 1989, and, by the latter half of 2020, had engaged 536 advanced therapy developers, with 373 clinical trials ongoing1. There are currently three gene therapy products approved by the US Food and Drug Administration (FDA), and the field continues to expand with more than 900 investigational new drug (IND) applications for ongoing gene therapy clinical studies2.

Recombinant vectors based on the non-pathogenic AAV are currently one of the most widely used vehicles for delivery in gene therapy. Different serotypes of AAV can be used to specifically target certain tissues and organs and are the subject of intensive research to improve their properties. AAV has also been used in a vaccine against COVID-19.

Regulatory authorities are increasing their support for initiatives in cell and gene therapy, resulting in new guiding documents on gene therapy manufacturing and clinical development of products3, but there are issues with inconsistent directives that are confounding manufacturing efforts4.

Some of the biggest challenges in manufacturing AAV therapeutics concern chemistry, manufacturing, and control (CMC). Gene therapy drugs often target rare diseases with trials involving only 1020 participants, and yet organizations must conduct all the CMC operations normally required for product commercialization in much larger manufacturing batches.

Advances in AAV-based therapeutics range from vector engineering to increasing transduction efficiency, to fine-tuning tissue tropism, and the avoidance of host immune response activation, as well as optimization of the small- and large-scale vector production. Support of bioprocess development relies on the availability of fit-for-purpose analytical tools to assess potential critical quality attributes (pCQAs) that are relevant to characterization and lot release testing. In the case of AAV-based therapeutics, this includes the ability to determine the concentrations of viral genomes (genome titer) and viral particles (capsid titer). Genome titer can be determined by PCR, but determining the capsid titer is more of a challenge.

In a recent webinar on capsid titer quantification for AAV-based therapeutics, AstraZeneca scientist Dr. Tomasz Witkos talks about how miniaturized immunoassays can be used to measure purified AAV vector as well as in-process samples and how the immunoassay experimental setup can be modified to measure several AAV serotypes.

He describes the need for a method to measure titer that could meet several criteria:

The drive to engineer recombinant AAVs to improve their properties can make capsid titer determination a challenge. The few commercial kits available for measuring capsid titer did not meet AstraZenecas needs in terms of dynamic range and accuracy. The team also needed a kit that could handle small sample volumes, preferably on an automated platform to save time.

In the webinar, Witkos presents an immunoassay developed on Gyrolab platform with AAV serotype-specific antibodies that can reliably measure AAV capsid titer in purified and in-process samples, delivering accurate and precise data suitable for AAV process development. The assay is versatile, with an antibody pair comprising a capture antibody that recognizes a broad range of AAV serotypes and another that is serotype-specific.

This assay setup can be readily adapted to titer measurements of various AAV serotypes and has a broader quantification range (3 3.5 logs) compared to a commercially available capsid ELISA kit (1 1.5 logs). The Gyrolab assay could be used to test neat samples and at low dilutions, which increases reproducibility compared to ELISA, which requires greater than 1,000x dilutions. Throughput was estimated at 50 samples in four dilution series in a 5-CD run, with reproducibility in the range of 1015 % CV. The team also appreciates Gyrolab Viewer as a very valuable tool to spot any issues with AAV particle aggregation.

Find out more about how Gyrolab system is improving the productivity of gene therapy efforts at AstraZeneca, by watching the Gyros Protein Technologies webinar, Capsid titer quantification for AAV-based therapeutics.

Discover the new Gyrolab AAVX Titer Kit when used in combination with Gyrolab system, this kit is designed to quickly deliver high-quality titer data for AAV serotypes 18 and AAVrh10 using smaller sample volumes compared to ELISA.

References

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Capsid titer quantification for AAV-based therapeutics - SelectScience

PUNE, India, March 22, 2021 /PRNewswire/ --Genetic Testing Market:Global Size, Trends, Competitive, Historical & Forecast Analysis, 2021-2027", Rising incidence of blood cancer, genetic disorders, cardiovascular diseases and surge in availability of new tests are some major factors driving the growth of the global genetic testing market.

Genetic Testing Market is valued at USD 10801.98 Million in 2020 and expected to reach USD 23143.42 Million by 2027 with the CAGR of 11.5% over the forecast period.

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Genetic testing is a type of medical test that detects changes in chromosomes, genes, or proteins. The results of a genetic test can confirm a suspected genetic condition and helps to control a person's chance of developing or passing on a genetic disorder. Genetic testing, also called as DNA testing. Genetic testing can also contain measuring the results of genetic changes such as RNA analysis as an output of gene expression, or through biochemical analysis to measure specific protein output. There are various type of genetic test are available to determine disorders such as predictive testing, carrier testing, prenatal and newborn testing, diagnostic testing, pharmacogenomic testing, nutrigenomics and others. Among all, newborn testing can identify genetic disorders early in life so treatment can be started as early as possible.

Global genetic testing market has turn out to be an important topic during COVID-19 pandemic outbreak. Genetic testing market gaining huge attention due to pandemic outbreak.

Genetic testing market report is segmented on the basis of test type, application and by regional & country level. Based upon test type, genetic testing market is classified into predictive testing, carrier testing, prenatal and newborn testing, diagnostic testing, pharmacogenomic testing, nutrigenomics and others. Based upon application, genetic testing market is classified into cancer, genetic diseases, cardiovascular diseases and others.

Genetic Testing MarketSegmentation

By Test Type:

By Application:

Primary Key Players who are working in Genetic Testing Market:

Genetic testing market report covers prominent players are Ambry Genetics, Illumina, Inc., IntegraGen, BGI, Roche Diagnostics, Biocartis Group NV, Bio-Helix Co. Ltd., 23andMe, Inc., bioMerieux SA, Abbott, Blueprint Genetics, Quest Diagnostics, Sequenom, Inc., Cepheid, Counsyl, Inc., deCODE Genetics, GeneDx, Genomic Health, Inc., Genomictree, Inc., LabCorp (Laboratory Corporation of America Holdings), Invitae Corporation, Luminex Corporation, Molecular MD, Myriad Genetics, Inc., Natera, Inc., Pacific Biosciences of California, Inc., Pathway Genomics Corporation, Qiagen, Siemens Healthineers AG, HTG Molecular Diagnostics, Inc. and Others.

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BGI has Launched NGS-Based HPV Test to the Global Market

News: April 20, 2018, BGI launched NGS-based HPV genotyping product, SentisTM-HPVseq, to the global market. This product provides detection of both high-risk and low-risk HPV types, which is used to screen for cervical cancer. It isrecognized as an effective method for primary cervical cancer screening. This HPV testing initiative has helped BGI expand its genetic testing portfolio and market share.

Genetic Testing Market Dynamics:

Rising incidence of blood cancer, genetic disorders, cardiovascular diseases and technological advancements and availability of new tests are some major factors driving the growth of the global genetic testing market. According to the American Heart Association, about 2,200 Americans die of disorder every day, a mean of 1 death every 40 seconds. According to Leukemia Research Foundation, every three minutes, someone is diagnosed with blood cancer more than 175,000 new cases are expected in the United States in 2019. Additionally, Leukemia is diagnosed 10 times more often in adults than children. New cases of leukemia, lymphoma and myeloma are expected to account for 10 percent of the estimated 1,762,450 new cancer cases diagnosed in the US in 2019.

According to American Cancer Society's, estimates for leukemia in the United States for 2020 are about 60,530 new cases of leukemia and 23,100 deaths from leukemia in all ages as well as about 19,940 new cases of acute myeloid leukemia in adults only. Moreover, due to the advancement in cancer research, more patients with cancer are being successfully treated will propel growth of markets. However, high costs of genetic testing and lack of skilled professionals will hamper the development of genetic testing market. According to the National Institutes of Health, the cost of genetic testing can range from under USD 100 to more than USD 2,000, depending on the nature and complexity of the test. However, innovations and advance development in genetic test and the increasing investment in cancer research is expected to boost the opportunity for the growth of genetic testing market.

Genetic Testing Market Regional Analysis:

North America is dominating the genetic testing market with the highest share due to rising awareness among the people about advanced treatment for healthcare. Presence of key players, established healthcare infrastructure, and availability of branded drugs are a number of the factors liable for its large share. Besides, favorable government initiatives and increase in number of research collaborations are some of the drivers expected to accelerate the regional market growth. The growth of genetic testing market in this region is primarily driven by the increasing blood cancer patient. In 2016, National Cancer Institutes stated that, there were an estimated 414,773 people living with leukemia in the United States. Cancer is one among the leading causes of death and disease within the U.S. The American Cancer Society (ACS) estimates that roughly 1.7 million new cases of cancer are going to be diagnosed within the U.S. in 2017.

According to The Leukemia & Lymphoma Society, an estimated combined total of 176,200 people in the US are expected to be diagnosed with leukemia, lymphoma or myeloma in 2019. Cancer usually develops in older people; 87% of all cancers in the United States are diagnosed in people 50 years of age or older. U.S. Department of Health and Human Services, the number of new cases of leukemia was 14.1 per 100,000 men and women per year and the number of deaths was 6.5 per 100,000 men and women per year.

The Asia Pacific is expected to emerge as the fastest-growing regional market over the forecast period with due to the increase in healthcare expenditure, large population rate, growing prevalence of cardiac diseases, rising awareness about early diagnosis, developing healthcare infrastructure, and availability of effective treatment in emerging countries, such as China and India. According to American Heart Association, in 2016, among NH Asians, CVD caused the deaths of 11,023 males and 10,672 females. In the Asia-Pacific region, rising disposable income, adoption of western lifestyle, living more sedentary lives and consuming junk foods with higher energy and fat. Asia Pacific has leading innovation in the treatment of blood cancers by developing and providing transformational treatments that extend and enhance lives. These are the major factors increase the growth of genetic testing markets. According to leukemia study report, in 2017, there were 11,923 combined new cases of leukemia, lymphoma and myeloma in Australia. India has the third highest number of blood cancer patients in the world after the US and China. Blood cancer contributes to 7% of all cancer cases in India.

Continued Genetic TestingMarket Report

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Contract Research Organizations (CRO) Marketis valued at USD 35.09 Billion in 2018 and expected to reach USD 50.65 Billion by 2025 with the CAGE of 6.31% over the forecast period.

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At 11.5% CAGR, Genetic Testing Market Size Expected to Reach 23143.42 Mn by 2027 Says Brandessence Market Research - PRNewswire

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of a protein called a clotting factor. There are about 13 types of clotting factors that act on platelets, which are needed to initiate the clotting process. There are three forms of hemophilia: A, B, and C. Hemophilia A is the most common form and is caused by a deficiency of clotting factor VIII. Hemophilia B is caused by a deficiency of clotting factor IX, and hemophilia C is caused by a deficiency of clotting factor XI. Hemophilia cannot be cured with current treatment options, which not only reduces symptoms such as spontaneous bleeding in muscles and joints, but increases the risk of intracranial bleeding.

The Research Insights has added a new market aptitude report to its extensive collection of research. The report is titled as Hemophilia Gene Therapy Market which emphases in describing the primary prospects and outlines in the market. Moreover, it gives a broad overview of the global market including the cataloguing, descriptions and executions. Additionally, it also converses the growth strategies along with the cost structures and production processes.

Hemophilia Gene Therapy market is expected to exhibit robust growth rate during the forecast period. Growing inclination towards digital payments has significantly driven the overall online payment gateways market. Various national governments as well banking organizations are now encouraging digital payment in order to reduce their operating costs and better visibility of transactions.

For Sample Report: https://www.theresearchinsights.com/request_sample.php?id=319357

Top Key Vendors in Market: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics, Bioverativ, Shire PLC, Freeline Therapeutics, BioMarin, uniQure

The report has been put together in a chapter-wise arrangement, by separating required illustrations transversely. This report is an expedient tool to get responses to some of the queries that hold significance for the growth of the Hemophilia Gene Therapy market during the forecast period. The evidence in the report was congregated from qualified organizations & dependable sources and was further authenticated by industry specialists for increased integrity.

The report also uses feedbacks given by industry experts to support the present and new players in enclosing effective business policies in the upcoming years. The report has been accumulated by taking the aid of info graphics, charts and tables to present the historical data and appraised figures of the Hemophilia Gene Therapy Market.

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This report is a thorough piece of work and assembled by primary as well as secondary research. The top segments in the Hemophilia Gene Therapy market have been emphasized clearly in the report for the readers to comprehend in a condensed manner. These sectors have been presented by giving information on their existing and anticipated state by the end of the forecast period.

The major stratagems approved by the well-known players for a better diffusion in the Hemophilia Gene Therapy market also forms a key section. The global market has also been analyzed in terms of revenue and also determines the regional outlook. The market crescendos such as market drivers, challenges, opportunities, and trends have been also presented.

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Table of Content:

Global Hemophilia Gene Therapy Market Research Report 2020-2026

Chapter 1: Industry Overview

Chapter 2: Hemophilia Gene Therapy Market International and China Market Analysis

Chapter 3: Environment Analysis of Hemophilia Gene Therapy.

Chapter 4: Analysis of Revenue by Classifications

Chapter 5: Analysis of Revenue by Regions and Applications

Chapter 6: Analysis of Hemophilia Gene Therapy Market Revenue Market Status.

Chapter 7: Analysis of Hemophilia Gene Therapy Industry Key Manufacturers

Chapter 8: Sales Price and Gross Margin Analysis

Chapter 9: Marketing Trader or Distributor Analysis of Hemophilia Gene Therapy.

Chapter 10: Development Trend of Hemophilia Gene Therapy Market 2020-2026.

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Hemophilia Gene Therapy Market is Thriving by World during Upcoming Year | Top Companies: Spark Therapeutics, Ultragenyx, Sangamo Therapeutics,...

NEW YORK, March 24, 2021 /PRNewswire/ --The push for psychedelic medicine is surging across North America and around the world. Scientific evidence supports the life-changing impact this safe and natural alternative has in treating mental illness of all types. New research from a number of organizations, including Johns Hopkinsin Baltimore and Imperial Collegein London, which revealed that patients with depressive disorders had a clinically significant positive response to psilocybin-assisted therapy may represent a therapeutic breakthrough in treating multiple neurological disorders. As research expands, so does support for the revolutionary treatment, which has actually been around for centuries. As seen before, anecdotal opinions often trigger scientific research which then, with validation, drives growth in the industry as well investor interest. That's exactly what's occurring in the psychedelic therapeutics space, major upticks in both new companies entering the space as well as investors excited about the possibilities. Potential stakeholders in this game-changing new market are looking for companies that can weather the complex and expensive process of running clinical trials and bringing a drug to market. A leader in psychedelic therapeutic research and drug development, Cybin Inc. (NEO: CYBN) (OTCQB: CLXPF)(Profile)has a strong clinical pipeline with phase 2 trials underway, holds ten provisional patents,and boasts a proven leadership and a time-tested tradition. Other companies vying for position in the psychedelics medicine space include COMPASS Pathways Plc (NASDAQ: CMPS), Mind Medicine Inc (OTCQB: MMEDF) (NEO: MMED), Numinus Wellness Inc. (OTCPK: LKYSF) (NEO: NUMI) (TSX.V: NUMI) and Field Trip Health Ltd.(OTCQX: FTRPF) (CSE: FTRP). Each of these companies is focused on providing better treatments for the millions suffering from mental health issues.

Click hereto view the custom infographic of theCybin editorial.

The Psychedelic Answers

More than 700 million people worldwide struggle with some form of mental illness, be it depression, addiction or post-traumatic stress disorder and the people counted are just those who seek help. The actual number is likely much higher, given the high frequency of nondiagnosis as well as stigmas and lack of effective treatments. One in four people will face a mental or neurological disorder at some point in their lives, yet "current treatments and the dominant model of mental health care do not adequately address the complex challenges of mental illness, which accounts for roughly one-third of adult disability globally."

More and more people are looking outside conventional protocols for solutions, and they're finding those solutions in the world of psychedelic medicine, specifically psilocybins. Psilocybins are a hallucinogenic substance found in certain types of mushroom, dubbed magic mushrooms, and used for centuries by indigenous cultures for religious, spiritual and health-related purposes. As is often the case, the modern world is learning from the ancient as mounting evidence points to these prolific fungi as a source for long-sought-for help in mental health and neurological disorders.

A Legacy of Success

Cybin Inc. (NEO: CYBN) (OTCQB: CLXPF)is intent on becoming the leader in this exciting breakthrough for mental well-being. The company is at the forefront of the revolution in mental health therapeutics and is developing a new class of psychedelic medicines and treatment protocols. Driving its commitment to excellence is the company's impressive leadership team of experienced professionals with a combined 80-plus years in the pharmaceutical industry.

CEO Doug Drysdale has three decades in the health-care sector, successfully building and turning around three pharmaceutical companies, and co-founder Eric So has raised hundreds of millions for various companies, directing value creation and strategic exits. Cybin's executive team has the experience and critical insights to navigate through the complexities the promising psychedelics industry offers.

Staffed with luminaries, the company's scientific team,helped develop widely used drugs such as Allegra, Sabril, Anzemet and Vaniqa, and is the only scientific team to have successfully commercialized a psychedelic drug to date.In addition, the team has facilitated more than $2 billion in pharmaceutical sales alongside being collectively involved in 37 exits across the biotech sector and various other verticals. Cybin's leadership team has a rich legacy of success and has the proven ability to both guide the company and maximize opportunity.

Tested, Proven Fundraising Ability

The importance of being well funded can't be overlooked and is especially important for research and drug development. Cybin and its leadership team have demonstrated ability to raise funds for key, strategic steps necessary for success. Most recently, Cybin raised C$45 million in the largest go-public capital raise in the Canadian psychedelic sector and a total of C$88 million across Seed, Series-A and Series-B financing rounds.

Part of that capital was used in the company's strategic acquisition of Adelia Therapeutics Inc., whose novel psychedelic molecules allowed Cybin to diversity its portfolio and provided access to multiple future indications. The acquisition also resulted in Cybin obtaining a range of technologies related to novel therapeutic delivery methods and therapeutic regimens, along with six patent applications. The acquisition also brought with it an expanding library of psychedelic derivative drug development candidates, with the first lead compounds expected to enter clinical studies this year.

A Three-Pillar Pipeline

Cybin is committed to a strong, well-established IP portfolioand clinical pipeline, as indicated by the Adelia acquisition. The company leads the industry in therapeutic development programs and innovative drug delivery systems using a three-pillar strategy with its novel drug-discovery platform, optimal novel and proprietary drug-delivery systems, and an innovative treatment regimen.

The company's IP model is diverse, covering chemically synthesized molecules, delivery mechanisms, screeners, protocols and new drug formulations alongside a merger and acquisition strategy focused on acquiring proprietary technologies and novel compounds and molecules. In addition, Cybin holds worldwide exclusive rights to sublingual film delivery technology from Intelgenx for the delivery of psilocybin and other psychedelic molecules.

In addition, the company has key partnerships in place. Cybin recently partnered with neurotech pioneer Kernelto leverage its Kernel Flow, an innovative technology designed to detect hemodynamic changes in the brain that pulses light through the skull and into the bloodstream to measure how much oxygen the blood is carrying at any given time. Cybin anticipates that the quantitative measurements enabled by Flow may improve the development, delivery and scaling of its psychedelic therapeutics.

"Access to Kernel's innovative Flow technology adds another exciting dimension to the investigative work that Cybin is doing to develop breakthrough treatments for mental health disorders such as depression and addiction," said CEO Doug Drysdale. "Currently, clinical investigators rely on limited subjective information from patients. The ability to collect quantitative data from our sponsored drug development programs is potentially game-changing in terms of our ability to measure where psychedelics work in the brain in real time, and how we ultimately design our future therapeutics. . . . This new cornerstone component of our sponsored clinical programs follows a record-setting capital raise, listing on the NEO Exchange and the acquisition of Adelia Therapeutics Inc., which added significant scientific capabilities, novel molecules, delivery mechanisms and intellectual property."

Just this week Cybin announced that it had signed a drug-development agreement with Catalent Inc., the leading global provider of advanced delivery technologies, development, and manufacturing solutions for drugs, biologics, cell and gene therapies, and consumer health products. Cybin will be applying Catalent's proprietary Zydis(R)orally disintegrating tablet technology for the delivery of its novel deuterated tryptamine, or CYB003, a potential therapy for treatment-resistant psychiatric disorders.

"We are excited to partner with the team at Catalent with the aim of developing fast-acting, shorter-duration formulations of CYB003, recently acquired as part of our acquisition of Adelia Therapeutics," Drysdale said. "Our focus on reducing the need for health system resources, such as in-clinic therapist time, is an important part of our goal to create scalable, more accessible treatments for mental health disorders."

There's little doubt about the lack of effective treatments for mental health and neurological disorders. Scientific research is revealing a new, more effective approaches to address the massive unmet medical need. Leaders in this new frontier of medicine will likely be both appreciated by patients and rewarded by the markets.

Making a Profit and a Difference

Cybin isn't alone in the quest to make a difference in the mental health space. Savvy companies see an opportunity in psychedelic therapeutics and are jostling for position in an industry destined to make a mark.

COMPASS Pathways Plc (NASDAQ: CMPS) intends to accelerate patient access to evidence-based innovation in mental health. "We focus our efforts on those who are not helped by current treatments," states the company, which is developing its COMP360 psilocybin therapy designed to offer relief for the millions of people who suffer with treatment-resistant depression (TRD). The company is pioneering the development of this therapy, in which its proprietary formulation of synthetic psilocybin, COMP360, is administered in conjunction with psychological support.

Mind Medicine Inc (OTC: MMEDF) (NEO: MMED)is a psychedelic medicine biotech company that discovers, develops and deploys psychedelic-inspired medicines and therapies to address addiction and mental illness. The company is assembling a compelling drug development pipeline of innovative treatments based on psychedelic substances including psilocybin, LSD, MDMA, DMT and an Ibogaine derivative, 18-MC.

MMED is also actively pursuing the development of LSD-assisted therapies through its Project Lucy, including a Phase2btrial for anxiety disorders planned to be conducted fully through the FDA pathway.

Numinus Wellness Inc. (OTC: LKYSF) (NEO: NUMI) (TSX.V: NUMI)supports access to psychedelic-assisted psychotherapy through ketamine-assisted psychotherapy and special access and compassionate trials. Numinus partners with practitioners in providing clients with access to psychedelic-assisted psychotherapy, and through its recent acquisition of Mindspace, a Quebec-based psychedelic programming leader, Numinus now supports practitioners across three clinic locations.

Field Trip Health Ltd.(OTCQX: FTRPF) (CNX: FTRP), a global leader in the development and delivery of psychedelic therapies, recently partneredwith WHOOP, a human performance company, to measure the biometric effects of Field Trip's psychedelic therapies. Field Trip is opening Field Trip Health centers across North America and Europe for the delivery of psychedelic therapies, which have demonstrated significant efficacy in treating mental health conditions such as depression, anxiety and PTSD.

Surging interest in psychedelic therapeutics has sparked rising involvement from savvy companies interested in making both a profit and a difference as well as investors looking for the next space to strategically make a move. Companies such as Cybin that offer the science, the leadership and the expertise needed to succeed have a good chance of making a real difference in the nascent industry.

For more information about Cybin, please visitCybin Inc.

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Science Drives Surging Interest in Psychedelic Therapeutics - PRNewswire

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

For more information on this Phase I clinical trial, contact Michelle Mendoza at 858-249-3015 or email alphastemcellclinic@ucsd.edu

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First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimer's Disease - UC San Diego Health

WATERTOWN, Mass., and Research Triangle Park, N.C., Feb. 17, 2021 /PRNewswire/ --Selecta Biosciences, Inc. (NASDAQ: SELB) and Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced the initiation of a Phase 1 dose-escalation trial of SEL-399, an adeno-associated viral serotype 8 (AAV8) empty vector capsid (EMC-101) containing no DNA combined with ImmTOR. The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

"We are pleased to further evaluate ImmTOR's ability to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing by having initiated this dose-escalation study of SEL-399," said Carsten Brunn, Ph.D., president and chief executive officer of Selecta. "This trial builds upon our strong preclinical data in non-human primates and marks the first time that ImmTOR in conjunction with an AAV capsid has been dosed in humans, which is a significant milestone. Data from this study will inform the design of future clinical trials in patients as we seek to unlock the full potential of gene therapy."

The dose-escalation trial of SEL-399 is designed to evaluate the safety and preliminary efficacyof ImmTOR in gene therapy. The study, being conducted in healthy volunteers at the SGS Life Sciences Clinical Pharmacology Unit in Antwerp, Belgium, plans to enroll up to 45 subjects to investigate increasing doses of ImmTOR and EMC-101. Subjects will be randomized in a 3:1 ratio of ImmTOR plus empty AAV8 capsid to empty capsid alone. Preliminary efficacy will be measured by assessing levels of AAV8-specific neutralizing antibodies.

Jude Samulski, Ph.D., chief scientific officer and co-founder of AskBio said, "By determining the dose at which ImmTOR is able to inhibit the formation of AAV-specific antibodies,this study could be a significant first step toward overcoming some of the unwanted immune responses associated with gene therapies. We look forward to using these findings to inform future studies as we work to develop strategies for repetitive dosing of AAV, thus extending durability of expression."

Selecta and AskBio expect to report initial results from this clinical trial in the fourth quarter of 2021.

AboutSelecta Biosciences, Inc.Selecta Biosciences Inc. (NASDAQ: SELB) is leveraging its clinically validated ImmTOR platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses. With a proven ability to induce tolerance to highly immunogenic proteins, ImmTOR has the potential to amplify the efficacy of biologic therapies, including redosing of life-saving gene therapies, as well as restore the body's natural self-tolerance in autoimmune diseases. The company's first program aimed at addressing immunogenicity to AAV gene therapies is expected to enter clinical trials in early 2021 in partnership with AskBio for the treatment of methylmalonic acidemia (MMA), a rare metabolic disorder. A wholly-owned program focused on addressing IgA nephropathy driven by ImmTOR and a therapeutic enzyme is also in development among additional product candidates. Selecta recently licensed its Phase 3 clinical product candidate, SEL-212, in chronic refractory gout to Sobi. For more information, please visitwww.selectabio.com.

About AskBioAsklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020,is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson's disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio's gene therapy platform includes Pro10, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK, the company has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. Founded in 2001 and an early innovator in the gene therapy field, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids. Learn more atwww.askbio.comor follow us onLinkedIn.

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2019, the Group employed around 104,000 people and had sales of43.5 billion euros. Capital expenditures amounted to2.9 billion euros, R&D expenses to5.3 billion euros. For more information, visit http://www.bayer.com.

AskBio Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's pipeline of development candidates; AskBio's collaboration with Selecta; AskBio's clinical trials, including its ability to enroll subjects, the timing of any such trials and any potential side effects; whether ImmTOR will be able to reduce the formation of antibodies to AAV capsids and potentially enable gene therapy redosing; the timing of and results from the SEL-399/101 trial; whether the SEL-399/101 study could be a significant first step in overcoming the immunogenicity concerns associated with gene therapies; AskBio's strategies for repetitive dosing of AAV, extending durability of expression; AskBio's goal of developing life-saving medicines aimed at curing genetic diseases; and the potential benefits of AskBio's development candidates to patients. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned regulatory milestones and timelines, clinical development plans and bringing its product candidates to market, due to a variety of reasons, including the ongoing COVID-19 pandemic, possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Selecta Forward-Looking StatementsAny statements in this press release about the future expectations, plans and prospects ofSelecta Biosciences, Inc.("the company"), including without limitation, statements regarding the unique proprietary technology platform of the company, and the unique proprietary platform of its partners, the potential of ImmTOR to enable re-dosing of AAV gene therapy, the potential treatment applications of product candidates utilizing the ImmTOR platform in areas such as gene therapy, the ability of the Company and AskBio to develop gene therapy products using ImmTOR and AskBio's technology, the novelty of treatment paradigms that the Company is able to develop, whether the observations made in non-human primate study subjects will translate to studies performed with human beings, the potential of any therapies developed by the company and AskBio to fulfill unmet medical needs, the company's plan to apply its ImmTOR technology platform to a range of biologics for rare and orphan genetic diseases, the potential of the company's intellectual property to enable repeat administration in gene therapy product candidates and products, the ability to re-dose patients and the potential of ImmTOR to allow for re-dosing, the potential to safely re-dose AAV, the ability to restore transgene expression, the potential of the ImmTOR technology platform generally and the company's ability to grow its strategic partnerships, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "hypothesize," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including, but not limited to, the following: the uncertainties inherent in the initiation, completion and cost of clinical trials including proof of concept trials, including the uncertain outcomes, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a particular clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, the ability to predict results of studies performed on human beings based on results of studies performed on non-human primates, the unproven approach of the company's ImmTOR technology, potential delays in enrollment of patients, undesirable side effects of the company's product candidates, its reliance on third parties to manufacture its product candidates and to conduct its clinical trials, the company's inability to maintain its existing or future collaborations, licenses or contractual relationships, its inability to protect its proprietary technology and intellectual property, potential delays in regulatory approvals, the availability of funding sufficient for its foreseeable and unforeseeable operating expenses and capital expenditure requirements, the company's recurring losses from operations and negative cash flows from operations raise substantial doubt regarding its ability to continue as a going concern, substantial fluctuation in the price of its common stock, and other important factors discussed in the "Risk Factors" section of the company's most recent Quarterly Report on Form 10-Q, and in other filings that the company makes with theSecurities and Exchange Commission. In addition, any forward-looking statements included in this press release represent the company's views only as of the date of its publication and should not be relied upon as representing its views as of any subsequent date. The company specifically disclaims any intention to update any forward-looking statements included in this press release.

SOURCE Asklepios BioPharmaceutical, Inc.

http://www.askbio.com

Original post:
Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR in Gene Therapy - PRNewswire

Researchers at University of California San Diego School of Medicine have launched a first-in-human Phase I clinical trial to assess the safety and efficacy of a gene therapy to deliver a key protein into the brains of persons with Alzheimers disease (AD) or Mild Cognitive Impairment (MCI), a condition that often precedes full-blown dementia.

The protein, called brain-derived neurotrophic factor or BDNF, is part of a family of growth factors found in the brain and central nervous system that support the survival of existing neurons and promote growth and differentiation of new neurons and synapses. BDNF is particularly important in brain regions susceptible to degeneration in AD.

In previous published research, principal investigator Mark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine, and colleagues described the prevention and reversal of brain cell degeneration and death in animal models.

Mark TuszynskiMark Tuszynski, MD, PhD, professor of neuroscience and director of the Translational Neuroscience Institute at UC San Diego School of Medicine.

We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimers disease the entorhinal cortex and hippocampus was able to reverse the loss of connections and to protect from ongoing cell degeneration, said Tuszynski. These benefits were observed in aged rats, aged monkeys and amyloid mice.

Amyloid mice are genetically engineered to inherit a mutation in the gene encoding the amyloid precursor protein, and as a result develop amyloid plaques aggregates of misfolded proteins in the brain that are considered a hallmark characteristic of AD.

BDNF is normally produced throughout life in the entorhinal cortex, an important memory center in the brain and one of the first places where the effects of AD typically appear in the form of short-term memory loss. Persons with AD have diminished levels of BDNF.

But BDNF is not easy to work with. It is a large molecule and cannot pass through the blood-brain barrier. As a result, researchers will use gene therapy in which a harmless adeno-associated virus (AAV2) is modified to carry the BDNF gene and injected directly into targeted regions of the brain, where researchers hope it will prompt production of therapeutic BDNF in nearby cells.

The injections are precisely controlled to contain exposure to surrounding degenerating neurons since freely circulating BDNF can cause adverse effects, such as seizures.

The three-year-long trial will recruit 12 participants with either diagnosed AD or MCI to receive AAV2-BDNF treatment, with another 12 persons serving as comparative controls over that period.

This is the first safety and efficacy assessment of AAV2-BDNF in humans. A previous gene therapy trial from 2001 to 2012 using AAV2 and a different protein called nerve growth factor (NGF) found heightened growth, axonal sprouting and activation of functional markers in the brains of participants.

The BDNF gene therapy trial in AD represents an advance over the earlier NGF trial, said Tuszynski. BDNF is a more potent growth factor than NGF for neural circuits that degenerate in AD. In addition, new methods for delivering BDNF will more effectively deliver and distribute it into the entorhinal cortex and hippocampus.

Despite billions of dollars of research investment and decades of effort, there are only two symptomatic treatments for AD. There is no cure or approved way to slow or stop progression of the neurological disorder that afflicts more than 5 million Americans and is the sixth leading cause of death in the United States.

Numerous clinical trials are ongoing to assess pharmaceutical remedies. Tuszynski said gene therapy, which debuted in 1980 and has been tested on multiple diseases and conditions, represents a different approach to a disease that requires new ways of thinking about the disease and new attempts at treatments.

We hope to build on recent successes of gene therapy in other diseases, including a breakthrough success in the treatment of congenital weakness in infants (spinal muscular atrophy) and blindness (Leber Hereditary Optic Neuropathy, a form of retinitis pigmentosa), Tuszynski said.

BDNF gene therapy has the potential, unlike other AD therapies currently under development, to rebuild brain circuits, slow cell loss and stimulate cell function. We are looking forward to observing the effects of this new effort in patients with AD and MCI.

Read more here:
UC San Diego: First-in-Human Clinical Trial to Assess Gene Therapy for Alzheimers Disease - India Education Diary

The deal, which is coming fast on the heels of the CROs January 2021 acquisition of antibody discovery company, Distributed Bio, is expected to close in the first quarter of 2021.

Charles River said the acquisition will establish it as a scientific partner for cell and gene therapy (CGT) development, testing, and manufacturing providing clients with an integrated solution from basic research through CGMP production.

It noted the Memphis biotechs services, primarily cell therapy and plasmid production, serve a market worth about US$1.5bn globally.That market is expected to grow annually by at least 25% over the next five years, said the CRO.

Jim Foster, Charles Rivers CEO, commented."This acquisition will be an exceptional strategic fit, adding to our comprehensive suite of early-stage research and manufacturing support solutions."

In a Q4 2020 earnings call yesterday, Foster said the COVID-19 pandemic has also enhanced the global focus on scientific innovation, which is generating biomedical breakthroughs across multiple therapeutic areas, including for COVID-19 vaccines.

This innovation has fueled continued investment in and the proliferation of more complex research techniques involving advanced drug modalities, such as cell and gene therapies.

The complexity of these new modalities is increasing our clients' reliance on a high science outsourcing partner like Charles River. To enhance our ability to meet our clients' needs in these emerging areas of scientific innovation and to take advantage of the significant growth opportunities that these advanced drug modalities present, we are expanding our portfolio and scientific expertise through a combination of acquisitions, strategic partnerships and internal investments.

In November 2019, Charles River singled out CGT as the fastest growing component of its biologics business. The following month, it revealed a US$380m (340m) deal to buy CGT specialist, HemaCare, a company that had worked on three cell therapies approved in the US - Kymriah (tisagenlecleucel), Yescarta (axicabtagene ciloleucel) and Provenge (sipuleucel-T).

Then in August last year the CRO announced the acquisition of Memphis based Cellero, (formerly Key Biologics and Astarte Biologics), a provider of cellular products for cell therapy developers and manufacturers worldwide, for US$38m.

The Cellero deal, said Charles River, would complement the HemaCare business by boosting the supply of critical biomaterials, including a wide range of human-derived primary cell types to support the discovery, development, and manufacture of cell therapies.

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Charles River acquires Cognate BioServices to expand cell and gene therapy programs - BioPharma-Reporter.com

HACKENSACK, N.J., Feb. 18, 2021 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization leading the fight to endDuchenne muscular dystrophy (Duchenne), andDuchenne UK, a UK-based patient organization,are pleased to announce ProfessorKanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of their Joint Research Grant Call of 2020. The full title of the research project is "Targeting the innate immune system to block acute inflammatory and chronic immune response to transgene and AAV vector in DMD".Professor Nagaraju's research will receive funding from the organizations in the amount of $350,000.

These are promising times for research into Duchenne muscular dystrophy (Duchenne). Several companies are now testing an approach that uses a shortened dystrophin gene to replace the faulty dystrophin gene in Duchenne. This is known as gene transfer using micro-dystrophin, or more commonly, gene therapy. The companies are using viruses known as AAVs (adeno-associated viruses) to deliver the therapy.

However, challenges exist in getting this treatment to the entire Duchenne population. This is mainly because of immune responses: some patients have pre-existing antibodies to the AAVs. This means they will not, currently, be able to have the treatment because their bodies will recognize the virus and stop it from delivering the micro-dystrophin to the cells. In addition, as gene therapy is a new treatment, it is not yet clear if another dose will be required at a later stage, and it is not currently possible to re-dose with the same AAV.

This is why Duchenne UK & PPMD launched a call for projects last year that would specifically address this challenge.

The organizations received a large number of proposals, and three were taken forward for final review from a panel of highly qualified, specialized scientists. They looked at a wide variety of factors, including significance to the Duchenne community, and the ability to translate the research into treatments for patients.

Professor Nagaraju's research is looking at blocking the mechanism by which the body is able to recognise an AAV virus and mount an immune response to it. Importantly, he is using medicines that are already in use in humans, in an approach known as repurposing.

If this approach were successful, it would allow more micro-dystrophin to get to the cells, potentially requiring a lower dose of the AAV than is currently being administered in the trials. It may also allow patients who have already been dosed with gene therapy to receive further doses. Further to this, by using repurposed drugs, this treatment should be more easily transferable to patients. Professor Nagaraju believes that "targeting initial immune recognition pathways is one way to improve efficacy and safety profiles of AAV mediated gene therapy".

PPMD's Founding President & CEO, Pat Furlong, and Duchenne UK's CEO, Emily Crossley explained in a joint statement:"Supporting patients and accelerating innovative research is at the heart of what we do at Duchenne UK and PPMD. We are pleased to partner with each other and award this grant. Gene therapy is offering great promise, but there are challenges associated with the immune response which are limiting the rate of progress and a barrier to ensuring all patients can have access to these potentially transformative therapies.We would like to thank all those who participated and supported our Joint Grant Call and are very much looking forward to working with Professor Nagaraju on this vitally important project for the Duchenne community."

To learn more about PPMD's innovative research agenda and our investment portfolio, visit PPMD's website.

About Parent Project Muscular Dystrophy

Duchenneis a fatal genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won four FDA approvals.

Everything we doand everything we have done since our founding in 1994helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.

About Duchenne UK

Duchenne Muscular Dystrophy (DMD) is a devastating muscle-wasting disease. It is the most common and severe form of Muscular Dystrophy. Diagnosed in childhood, it mainly affects boys. There is currently no cure. Started by families affected by the disease, Duchenne UK has one clear aim to end Duchenne.

Duchenne UK are funding research that's focused on getting treatments to those affected now as well as pushing for an effective treatment in the future.

Duchenne UK connects leading researchers with industry, the NHS and patients to challenge every stage of drug development, from research to clinical trials to drug approval. They connect families with each other to create a network of mutual support and to pool resources, knowledge and experience.

For more information about Duchenne UK: visit http://www.duchenneuk.org.

SOURCE Parent Project Muscular Dystrophy (PPMD)

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Duchenne UK and Parent Project Muscular Dystrophy Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne Muscular Dystrophy -...