UF researchers announce gene therapy project for Duchenne muscular dystrophy

The University of Florida’s Powell Gene Therapy Center has partnered with a biotechnology company to launch a gene therapy project aimed at developing potential treatments for Duchenne muscular dystrophy, the most common form of muscular dystrophy.

As part of a nearly $2 million agreement, scientists at the UF Powell Gene Therapy Center will manufacture an adeno-associated virus vector, which is a delivery mechanism that uses a harmless virus to deposit a missing protein into the cells that lack it. In this case, the vector will contain the genetic instructions for a shortened but functional version of dystrophin, a crucial protein in the membrane of muscle cells that is absent or deficient in boys with Duchenne muscular dystrophy. The lack of dystrophin leads to a cascade of progressive muscle loss, which typically causes boys to lose the ability to walk in their teen years and leads to premature death. Currently there are limited options for treatment of the disease, which affects about 1 in 3,500 male births worldwide.

Under the terms of the agreement with Solid GT LLC, UF scientists will also conduct preclinical studies in animal models and collaborate on the development of a clinical program intended for human studies. Collaborators include researchers at the University of Washington, Texas A&M University, the University of Missouri and others.

“Solid GT’s investment in our gene therapy platforms will have a big impact,” said Barry Byrne, M.D., Ph.D., director of the UF Powell Gene Therapy Center, a professor of pediatrics in the UF College of Medicine and member of the UF Genetics Institute. “We have already had promising results in mouse and dog models of Duchenne and we plan to begin human studies as quickly as possible, given the urgency of the clinical problem.”

The UF Powell Gene Therapy Center is a world leader in gene therapy, having successfully conducted trials in inherited retinal disorders, Pompe disease and related neuromuscular conditions. Researchers at UF pioneered the use of adeno-associated virus vectors, safe variants of viruses, which will be used in the project with Solid GT LLC.

“Gene therapy as a disease-modifying approach is a very comprehensive undertaking, and while Solid GT is building a team and making available the resources, much of the work is being done and will continue to be done with partners and experts, such as those at UF,” said Ilan Ganot, CEO of Solid GT, a subsidiary of Solid Biosciences LLC.

For media inquiries, call Mindy Cameron at 317-853-6294 or email: mindycameron@ufl.edu