The U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for the GALGT2 gene therapy program for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics and Nationwide Children’s Hospital recently announced.
Following the FDA clearance, Nationwide Children’s will continue to advance the program by initiating a Phase 1/2a clinical trial in individuals with DMD by the end of 2017 to assess gene expression after gene therapy with GALGT2.
The study will enroll at least six individuals to receive the gene therapy by direct injection into the femoral arteries of the legs at a dose that has been tested before and has shown robust gene expression. Assessing gene expression is the primary goal of the study. This will be measured with open muscle biopsies (at baseline and at three months) and needle muscle biopsies (at six and 12 months). The trial will also evaluate motor performance with tests including the six-minute walk test (6MWT) or time to arise from the floor.
The GALGT2 gene therapy program’s IND is a result of a license agreement signed by Sarepta and Nationwide Children’s in January 2017.
The program focuses on a potential surrogate gene therapy approach for the treatment of DMD that targets the dystroglycan complex to preserve muscle function. In past animal studies, over-expression of GALGT2 resulted in muscle that works normally, even in the absence of dystrophin.
“The field of gene therapy represents a potentially transformative approach to the treatment of Duchenne muscular dystrophy,” Douglas Ingram, president and chief executive officer of Sarepta, said in a press release. “The GALGT2 program offers the potential to treat the majority of dystrophin gene mutations responsible for this devastating disease, DMD, as well as having potential utility in other muscular dystrophies, and perfectly exemplifies our strategy of working with urgency to find and progress potentially life-changing new treatments for DMD through agnostically investing in precision genetic medicine.”
Paul Martin, PhD, an investigator at the Nationwide Children’s Center for Gene Therapy, developed the program. It is now led by Kevin Flanigan, MD, director of the center.
“This approach represents a potential new pathway to treat the root cause of DMD. We look forward to continuing to collaborate with Sarepta, whose dedication to DMD, to the individuals impacted by the disease, and to rigorous scientific exploration, matches our own here at Nationwide Children’s Hospital,” Flanigan said.
Nationwide Children’s produced a podcast in 2013 to discuss the GALGT2 gene therapy program. That podcast can be accessed here for more information.