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PARIS--(BUSINESS WIRE)-- Genethon, a non-profit research organization focused on developing gene therapies for rare diseases, today marks International Rare Disease Day by highlighting its efforts to bring gene therapies to patients suffering from rare diseases such as limb girdle muscular dystrophies, Crigler Najjar syndrome and Duchenne muscular dystrophy. Globally more than 300 million people, most of them children, are living with 7,000 rare diseases. Eighty percent are genetic diseases and nearly 95% have no effective therapies. This places a heavy burden on health care systems, costing billions of dollars. Because patient populations are small, these diseases don’t readily fit into the biopharma industry’s business models. “I am happy to say that thousands of patients around the world already are benefitting from our research through traditional licensing agreements such as the one that led to the development of the first gene therapy approved in 2019 for spinal muscular atrophy,” said Frederic Revah, Ph.D., Genethon CEO. “However, after 30 years of pioneering gene therapies, we realized that we had to create different development strategies beyond licensing out to biopharma the discoveries made by our more than 200 dedicated scientists.” One of those strategies involves creating its first spinout, Atamyo Therapeutics, in 2020. Atamyo is developing gene therapies for limb girdle muscular dystrophies (LGMD). The gene therapies were designed by researchers in Genethon’s Progressive Dystrophies Laboratory under the leadership of Isabelle Richard, Ph.D., who is Atamyo’s Co-Founder & Chief Scientific Officer and Head of Progressive Muscular Dystrophies Team at Genethon. LGMD are a group of rare diseases and the fourth most common genetic cause of muscle weakness. It has more than 30 subtypes related to genetic mutations with an estimated worldwide prevalence of all forms ranging from one person in 14,500 to one person in 123,000. Led by CEO Stephane Degove, Atamyo is already conducting clinical trials in Europe for LGMD2I/R9 and is ready to initiate clinical trials for LGMD2C/R5. It has IND-enabling studies under way for a third, LGMD2A/R1; and the U.S. Food and Drug Administration has cleared Atamyo’s IND for clinical trials of LGMD2I/R9. Another strategy involves Genethon pursuing clinical development itself. Genethon is conducting pivotal trials for a gene therapy for Crigler Najjar syndrome, a life threatening liver disease. It would be the first-ever gene therapy for this ultra-rare disease which affects about 1 person (male or female) per 750,000 to 1 million people worldwide. The pivotal trials of the Crigler-Najjar gene therapy will attempt to confirm positive data from Phase 1/2 trials. If successful, the results could support regulatory filing in Europe in two years. A third strategy involves Genethon’s partnership with Sarepta Therapeutics of Cambridge, MA, for co-development of a gene therapy for Duchenne muscular dystrophy, a genetic disease resulting in progressive muscular degeneration and weakness. The disease affects mostly males and occurs in about 6 children per 100,000 in Europe and North America. Genethon’s collaboration with Sarepta involves co-development of the clinical gene therapy program and, under the terms of agreement, Genethon is responsible for commercializing the product GNT0004 in Europe (excluding the UK) and Sarepta is responsible for the rest of the world. Founded in 1990 by patients for patients, Genethon from the start has embraced a patient-centric drug development approach based on applying the most advanced science with a pledge of ensuring affordability to gene therapy medicines. “Despite challenges in generating interest with biopharma companies and investors, Genethon will never abandon patients suffering from rare and ultra-rare diseases,” said Dr. Revah. “Gene therapies have proved their curative potential, and we will continue to advance new clinical development and commercialization strategies to try to ensure all patients benefit from them.” About Genethon As a pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a non-profit laboratory that was established by AFM-Telethon. A first gene therapy for spinal muscular atrophy to which Généthon contributed has obtained a product license. With more than 200 scientists and professional staff, Genethon is pursuing its aim to develop therapies which change the lives of patients suffering from rare genetic diseases. Thirteen products stemming from by Genethon’s R&D or from collaborations are in clinical trial for diseases of the liver, blood, immune system, muscles and eyes. Seven other products could enter clinical trials over the next five years. More information at . View source version on businesswire.com: Contacts U.S. Contact: Charles Craig Opus Biotech Communications 404-245-0591 charles.s.craig@gmail.com EU Contact: Stéphanie Bardon AFM-Téléthon Press Relation Manager +33 1 69 47 12 78 +33 6 79 34 15 68 SBARDON@afm-telethon.fr Source: Genethon View this news release online at:

WESTPORT, Conn., Sept. 21, 2023 /PRNewswire/ -- Today, Coalition to Cure Calpain 3 (C3) in partnership with the National Organization for Rare Disorders (NORD®) launched a study with global reach to research Calpainopathy (including limb-girdle muscular dystrophy type 2A/LGMDR1 and limb-girdle muscular dystrophy type 1I/LGMDD4), a rare form of muscular dystrophy that causes progressive muscle weakness and wasting. Calpainopathy currently has no cure. Continue Reading Coalition to Cure Calpain 3 in Partnership with the National Organization for Rare Disorders (NORD®) Launches LGMD2A/Calpainopathy Registry The new study, the LGMD2A/Calpainopathy Registry, creates a platform to bring the Calpainopathy community together and collect patient data that is an essential requirement for policy makers, academic researchers, and therapeutics companies to advance treatments for this disease. Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials. The LGMD2A/Calpainopathy Registry replaces C3's original patient registry. While the original registry served primarily to contact Calpainopathy patients, the new registry will have the capability to collect longitudinal data by having participants periodically update their information. This information is vital to help us understand the progression of symptoms over time, which will inform patient care, drug development, and clinical trial design. "Our goal is to enroll as many patients, or their parents or legal guardians, as possible," notes Jordan Boslego, C3 President. "The success of the registry is dependent upon community participation, and it's essential that the 1000+ patients in our current Global LGMD2A Registry take the time to register on this new platform." "The LGMD2A/Calpainopathy Registry will provide a complete picture of each patient's experience with this disease," shares Dr. Jennifer Levy, C3 Scientific Director. "We are launching this initiative to help fill in the missing information researchers and medical experts need to advance research and one day, find a cure." "This new study has tremendous promise as a strong partnership that engages the patient community and addresses current knowledge gaps for Calpainopathy. NORD is thrilled to be a part of driving research and innovation-based outcomes for all the families in the Calpainopathy community," says Aliza Fink, Director of Research Programs, NORD. For more information, visit . About Coalition to Cure Calpain 3 Coalition to Cure Calpain 3 (C3), a U.S.-based, public, 501(c)(3) non-profit organization, was founded in 2010 to fulfill a mission to drive high-quality research while educating the global community about the muscle-wasting disease limb-girdle muscular dystrophy type 2A (LGMD2A), a form of calpainopathy. C3 has committed over $2 million in grants to international leaders in the muscular dystrophy field to support the development of essential research tools, the identification of outcome assessments that will be used in future clinical trials, and the testing of potential therapies in animal models to determine if they can be safe and effective for LGMD2A. For more information on C3's efforts to treat and cure this disease, visit . About National Organization for Rare Disorders, Inc. (NORD®) The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit . rarediseases.org. Media Contacts Jennifer Levy, C3 [email protected] (203)429-4383 Marketing, NORD [email protected] SOURCE Coalition to Cure Calpain 3

A multicenter phase 1b study will evaluate safety, pharmacodynamic and efficacy of ATA-200 ATA-200 is the second Atamyo’s next generation gene replacement therapy entering the clinic ATA-200 is eligible to receive up to €8m ($8.6m) of non-dilutive financing from France 2030 program EVRY, France--(BUSINESS WIRE)-- Atamyo Therapeutics, a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular diseases, today announced the filing of a Clinical Trial Application (CTA) in Europe for ATA-200, its gene therapy targeting γ-sarcoglycan (SGCG) related limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5). The company also announced that ATA-200 has been granted a non-dilutive financing up to €8m ($8.6m) from France 2030 public program managed by Bpifrance, to support its clinical trials and manufacturing development programs. This clinical trial (EUCT 2023-506440-16-00) is a multicenter, Phase 1b, open-label, dose escalation study evaluating safety, pharmacodynamic, efficacy, and immunogenicity of intravenous ATA-200, a single-dose Adeno-Associated Virus (AAV) vector carrying the human γ-sarcoglycan transgene. “LGMD-R5 is the most rapidly progressive and debilitating form of LGMDs with onset of symptoms in early childhood, a loss of ambulation before adulthood, and frequent cardiac impairment. No curative treatment exists for this disease,” said Dr Sophie Olivier, Chief Medical Officer of Atamyo. “ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy,” said Isabelle Richard, Ph.D., Co-founder and Chief Scientific Officer of Atamyo. “This first-in-class experimental treatment represents a new hope for the patients.” “We are thrilled to European Clinical trial and to receive such a strong public financing for the devastating LGMD2C/R5 disease,” said Stephane Degove, Co-Founder and Chief Executive Officer of Atamyo. “With the ongoing clinical trial in LGMD-R9, the initiation of the clinical program for LGMD-R5/2C confirms our unique capabilities in bringing to patients suffering from limb-girdle muscular dystrophies a new generation of safe and effective gene therapies.” LGMD2C/R5 is a rare genetic disease caused by mutations in the gene that produces γ-sarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. It affects an estimated 2,000 people in Europe only. In the classical form, symptoms appear in early childhood and patients suffer from progressive muscular weakness leading to loss of ambulation before adulthood. Cardiac involvement, typically presenting as dilated cardiomyopathy is reported in approximately half of patients. There is no curative treatment. The management of LGMDR5 is solely supportive. ATA-200, the gene therapy for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan. In preclinical mice models, ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology. Atamyo plans to initiate dosing in patients for ATA-200 in the first half of 2024. In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein. About Atamyo Therapeutics Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments. For more information visit About France 2030 Launched by the President of France Republic in October 2021, France 2030 is a €54 ($58) billion program which aims to accelerate the transformation of key sectors of the French economy through innovation. From fundamental research, emergence of an idea, and up to the production of a new product or service, France 2030 supports the entire life cycle of innovation until its industrialization. More information on : france2030.gouv.fr View source version on businesswire.com: Contacts U.S. Contact: Charles Craig, Opus Biotech Communications charles.s.craig@gmail.com, 404-245-0591 European contact: contact@atamyo.com Source: Atamyo Therapeutics View this news release online at: