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 www.genethon.com.

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