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Fighting every day to free individuals — and the families who love them — from the harmful effects of muscle-debilitating diseases so they can live longer and grow stronger

Solid Biosciences Launches DMD Gene Therapy Trial

Nov. 30, 2017

Press Release: Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy

Background: Solid Biosciences today announced the launch of its first clinical trial for SGT-001, the company’s experimental gene transfer therapy for Duchenne muscular dystrophy (DMD).

The phase 1/2 study, called IGNITE DMD, is designed to assess safety and efficacy of a single dose of SGT-001 in children and adolescents with DMD. The underlying cause of DMD is an absence or deficiency of the muscle protein dystrophin, due to a mutation in the DMD gene. SGT-001 is a gene therapy designed to address the genetic cause of the disease via the delivery of highly miniaturized “microdystrophin” replacement genes that enable production of a functional protein to substitute for the missing dystrophin. Data from Solid Biosciences’ preclinical program suggest that the therapy potentially may be able to slow or stop the progression of DMD, regardless of genetic mutation or disease stage.

Enrollment at the first trial site United Sates will begin in the coming days, and additional sites are expected to open soon.

MDA has been supporting the development of gene therapy for neuromuscular diseases — including more than $13.5 million in investments in gene therapy for DMD — as a high priority, since the 1980s.

Statement from MDA Scientific Program Officer Laura Hagerty, Ph.D.: “This is an historic time for the DMD community. After decades of tireless work by top researchers, and after overcoming many obstacles and setbacks, we are seeing a burst of new gene therapy approaches maturing into clinical-stage testing. “MDA congratulates Solid Biosciences on the initiation of their first clinical trial for SGT-001 and, along with the DMD community, will be closely following the progress and awaiting the results of this important study.”

About MDA
MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at mda.org.

Roxan Triolo Olivas
MDA Vice President
Public Relations and Community Programs
rolivas@mdausa.org