The Phase 1/2 trial, which is under way in Denmark, France and the U.K, is sponsored by Atamyo Therapeutics, an Evry, France-based biotechnology company created by Genethon to accelerate development of gene therapies for LGMD, a group of devastating pathologies characterized by progressive degeneration of muscles of the upper and lower limbs.
“This major milestone is the result of 30 years of pioneering research conducted by Isabelle Richard, Ph.D., who is head of our Progressive Muscular Dystrophies team,” said Frederic Revah Ph.D., Genethon CEO. “The treatment of this first patient demonstrates the quality of the work carried out at Genethon and embodies our ‘raison d'être,’ which is creating new therapies for rare diseases without treatment. This trial is the 12th ongoing trial in the world resulting from the innovative R&D at Genethon.”
Dr. Richard, who is Atamyo’s Co-Founder and Chief Scientific Officer, added, “This treatment for patients suffering from LGMD2I/R9 is the culmination of years of work for my team and myself. This is a step that we researchers were all waiting for and I hope that this drug candidate will make it possible to stop the disease or reverse its course in patients. We expect ATA-100 to be the first of a series of new gene therapies for other limb girdle muscular dystrophies.”
LGMD2I/R9 is caused by mutations in the gene that produces fukutin-related protein (FKRP). It affects an estimated 5,000 people in the U.S. and Europe. Symptoms appear around late childhood or early adulthood. Patients suffer from loss of ambulation and are prone to respiratory impairment and myocardial dysfunction. There are no treatments for the disease.
There are more than 30 subtypes of LGMD associated with genetic mutations. Two other gene therapies for the limb girdle myopathies, gamma-sarcoglycanopathy and calpainopathy, also were developed by Dr. Richard's team at Genethon and licensed to Atamyo.
ATA-100 is a one-time replacement gene therapy combining an AAV vector and a normal copy of the FKRP gene. The Phase 1/2 clinical trial will assess the safety and efficacy of ATA-100. The first patient was treated by Prof. John Vissing, Director of the Copenhagen Neuromuscular Center at the Rigshospital National Hospital and principal investigator of this study. For more information see Atamyo’s press release.
About Genethon
A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. 12 products resulting from Genethon’s research are in clinical trials for eye, liver, blood, immune system and muscle diseases. A further 7 products are in the preparation phase for clinical trials over the next five years. Find out more: genethon.com
Contacts:
Dan Eramian
Opus Biotech Communications
http://opusbiotech.com/
425-306-8716
Charles Craig
Opus Biotech Communications
http://opusbiotech.com/
404-245-0591
charles.s.craig@gmail.com
Method of Research
Randomized controlled/clinical trial
Subject of Research
People