Today, it was announced that Sarepta Therapeutics, Inc. and Genethon have signed a gene therapy research collaboration to jointly develop treatments for Duchenne muscular dystrophy (DMD).
Unlike Sarepta’s current pipeline that is focused on specific dystrophin gene mutations, Genethon’s micro-dystrophin gene therapy approach can target most patients with DMD, and the company has demonstrated proof-of-concept via robust gene expression with their micro-dystrophin program in a large animal model of DMD.
DMD is primarily the result of a lack of functional dystrophin protein. Patients with the progressive muscle disorder have been known to lose the ability to walk as early as age 10, and suffer from life-threatening lung and heart complications in their late teens and 20s.
“Our agreement with Genethon strengthens our ongoing commitment to patients and is aligned with our strategy of building the industry’s most comprehensive franchise in DMD,” stated Edward Kaye, Sarepta’s chief executive officer.
“This partnership brings together our collective experience in Duchenne drug development and Genethon’s particular expertise in gene therapy for rare diseases. We look forward to working with Genethon given their knowledge, large infrastructure and state-of the-art manufacturing capabilities to advance next generation therapies for DMD.”
Moving forward, Genethon will be responsible for all early development work, and Sarepta will have the option to co-develop their micro-dystrophin program, if the preliminary data shows promise.
The initial phase 1/2a trial of micro-dystrophin gene therapy is slated to begin in the last quarter of 2017 at Nationwide Children’s Hospital in Columbus, OH. Funding for these studies has included support from the Parent Project Muscular Dystrophy (PPMD) who provided $2.2 million, in addition to money funded by Sarepta.
“Microdystrophin-based gene therapy is a very promising approach with potential application to a large majority of Duchenne patients. In order to accelerate the development of a treatment, we are very pleased to partner with Sarepta Therapeutics, which has demonstrated commitment and success for innovative therapies for Duchenne muscular dystrophy,” said Frederic Revah, CEO of Genethon.
“This partnership brings together the highly complementary and synergistic expertise of Sarepta and Genethon, to the benefit of the patients.”