Genethon

EVRY, France--( BUSINESS WIRE )-- Atamyo Therapeutics , a biotechnology company focused on the development of new-generation gene therapies targeting neuromuscular disease, today announced its participation in six upcoming conferences in the US and Europe to highlight recent developments in its programs targeting limb-girdle muscular dystrophies (LGMDs). Atamyo CEO Stephan Degove will provide a corporate overview at Sach’s 20 th Annual Biotech in Europe Forum, Chardan's 8th Annual Genetic Medicines Conference and the Cell & Gene Meeting on the Mesa 2024. • Sach’s 20th Annual Biotech in Europe Forum, held in Basel, Switzerland, on September 25-26, 2024: Presentation on September 25, at 11:40 am (CEST), in the Ruby Room. • Chardan's 8th Annual Genetic Medicines Conference, held in New York City, on September 30 and October 1, 2024: Presentation on September 30 at 11:00 am (US EDT), in the Embassy Room. • Cell & Gene Meeting on the Mesa, held in Phoenix, AZ, on October 7-9, 2024: Presentation on October 8, at 5:45 pm (US MST), at the FLW Ballroom F. Two communications at the 29th Annual Congress of the World Muscle Society, October 8-12, 2024, in the Prague Congress Centre, Prague, Czech Republic, will describe Atamyo’s LGMD programs. • “Preliminary results from a Phase 1-2 gene therapy study of ATA-100, AAV9 vector encoding FKRP, in patients with Limb Girdle Muscular Dystrophy R9.” Oral presentation by S. Olivier (Atamyo), at Congress Hall on Saturday, October 12, 2024, 8 am - 8:15 am (CEST) • “MRI characterization of the cardiac involvement in LGMD2i/R9.” JY Hogrel (Institute of Myology, Paris) et al., Poster Board Number 87P at Forum Hall, on Wednesday, October 9, 2024, 5:15 pm - 6:15 pm (CEST). Two communications at the European Society of Gene & Cell Therapy 31 st Annual Congress, October 22-25, 2024 in La Nuvola, Rome, Italy, will describe Atamyo’s LGMD programs. Both posters will be presented during Poster Session II on Wednesday 23 October, 1:30 pm to 3 pm (CEST). • “Development of a quantitative alpha-dystroglycan glycosylation test in patients with Limb Girdle Muscular Dystrophy R9 treated in ATA-001-FKRP open-label multicenter AAV trial.” E Thevenot (Genethon) et al., Poster number P0088 • “Natural history of limb girdle muscular dystrophy R9: two-year follow-up of a European cohort.” S Olivier (Atamyo) et al., Poster number P0264 Two communications on Atamyo’s LGMD-R9 program will be presented at the ASGCT’s Breakthroughs in Muscular Dystrophy Conference, November 19-20, 2024, in Chicago, IL. • “Development of Gene Therapy for LGMD-R9.” Oral presentation by E. Gicquel (Genethon), on November 19, 2024, 5:15 p.m. - 5:30 p.m. (US CDT), Great Lakes East Ballroom at the Westin Michigan Avenue Hotel. • “Overcoming Phenotypic Variability in Dystrophic Models: A Machine Learning Method for Refined Gene Therapy Evaluation.” E. Brureau (Genethon) et al. Poster P27. Session on November 16, 2024, 8 a.m. - 7 p.m. (US CDT), Great Lakes East Ballroom at the Westin Michigan Avenue Hotel Publication in Nature Communications of a Next-Generation Gene Therapy Vector for Muscle Diseases, Using AI Predictive Methodology to Improve Efficacy and Safety. • “An engineered AAV targeting integrin alphaV beta 6 presents improved myotropism across species,” by Ai Vu Hong (Genethon), et al., was published in Nature Communications ( https://doi.org/10.1038/s41467-024-52002-4 ). The paper describes development of the next-generation LICA-1 capsid by Isabelle Richard, Ph.D., Atamyo’s Chief Scientific Officer, and her Progressive Muscular Dystrophies Team at Genethon. Atamyo has rights to the LICA-1 capsid for cardiomyopathies indications. 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 LGMD2I/R9 and LGMD2C/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 www.atamyo.com

As several well-funded companies race to make generative AI models that can design new protein therapies from scratch , one small startup has found a new use for existing AI systems: compressing large proteins to better package them in gene therapies or deliver them to hard-to-reach places like the back of the eye. The startup, called ZipBio, has raised $4 million in seed funding for the idea, it told Endpoints News exclusively. “The inspiration came from the compression of technology in the digital world,” CEO Roee Farber said in an interview. Of course, shrinking the structure of a protein and retaining its function isn’t as simple as compressing digital documents into a zip file. The startup is trying to isolate small parts of proteins that are most important for making a drug and shave off the rest. The problem is that the rest of the protein is there for a reason. Removing it can cause the entire thing to collapse. ZipBio’s solution is to use generative AI to create a new backbone that holds that key part of the protein in place. “In the past, nobody succeeded with that. Now it’s something we can do relatively trivially,” Farber said. The company hasn’t published details about how its approach works, but Farber said ZipBio is using and refining publicly available AI models for protein design, rather than creating its own. The startup was founded in 2023, began work on its lead drug just eight months ago, and is already testing it in animals, Farber said. That drug candidate builds on Regeneron’s antibody Eylea as a starting point — an eye therapy that made the company $5.89 billion last year. Eylea inhibits two forms of VEGF, a protein that can spur unwanted blood vessel growth in the eyes. But just blocking the A and B forms of the protein doesn’t fully treat everyone. ZipBio has created a compact inhibitor of VEGF-C and VEGF-D and meshed it with Eylea, using AI to create a hybrid molecule that Farber says is compact enough to travel to the back of the eye, something that a simple fusion of two full antibodies likely wouldn’t do. Clinical trials could begin as soon as 2026. The startup is also working with the French nonprofit Généthon to create a compact version of a protein that’s too big to fit into AAVs — the most common viral vectors used in gene therapy — to treat a rare disease called glycogen storage disorder III, a form of muscular dystrophy. ZipBio’s seed funding was led by NFX, a venture capital firm that invests in tech startups at a seed stage or earlier. MoreVC, which focuses on early-stage Israeli tech startups, also contributed. ZipBio’s team of 12 is currently split between Seattle and Tel Aviv, Israel.

PARIS--(BUSINESS WIRE)-- Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), provided updates in its latest Newsletter on results of its gene therapy clinical trials for Duchenne Muscular Dystrophy and advances in gene therapies for limb-girdle muscular dystrophies by its spinoff Atamyo Therapeutics. Also in an interview, Roger Hajjar, M.D., Director of the Gene and Cell Therapy Institute of Mass General Brigham in Boston, renowned gene therapy researcher and entrepreneur, offered insights on what attracted him to join Atamyo Therapeutics as the newest board member of the Genethon spinout company. “What resonated with me was the fact that Atamyo is a research endeavor by investigators within Genethon whose work is being translated into the clinic,” Dr. Hajjar said. “It is a very nice demonstration of a bench to bedside approach. It’s definitely a new model.” Genethon CEO Frederic Revah, Ph.D., commenting on gene therapy trials for Duchenne Muscular Dystrophy, observed, “The effective dose in this first phase of the trial is lower than that used in other gene therapy programs using microdystrophin. Based on the recommendation of the independent expert committee (Data Monitoring Committee), we are now preparing the pivotal part of our clinical trial in the European Union.” Dr. Revah’s commentary and Dr. Hajjar’s interview are part of Genethon’s current Newsletter highlighting the organization’s product development, research collaborations and scientific journal publications. Read the full Newsletter. 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 Genethon 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 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 Stéphanie Bardon 06.45.15.95.87 communication@genethon.fr Source: Genethon View this news release online at: