Coalition to Cure Calpain 3 and Muscular Dystrophy Association partner to fund gene therapy grant

Muscular Dystrophy Association (MDA) and Coalition to Cure Calpain 3 (C3) partner to fund $300,000 research grant for gene therapy development in limb-girdle muscular dystrophy. This grant supports the work of Melissa Spencer, PhD, Director of the Neuromuscular Division in Neurology, Co-Director of the Center for Duchenne Muscular Dystrophy and member of the Broad Stem Cell Research Center at UCLA.

The grant will fund Dr. Spencer’s project titled “Development of Optimized Transgenes and Capsids for Treating LGMD2A/R1,” which focuses on advancing gene therapy for limb-girdle muscular dystrophy type 2A (LGMD2A/R1), a progressive neuromuscular disease.

This collaboration is part of MDA’s ongoing commitment to accelerate innovative research that could lead to life-changing treatments for individuals living with muscular dystrophies. Dr. Spencer’s research aims to optimize transgenes and viral capsids for gene therapy targeting calpain-3 (CAPN3) mutations, the underlying genetic cause of LGMD2A/R1. By enhancing the delivery and functionality of these therapeutic components, this project holds significant promise for creating more effective treatment options for patients affected by this form of muscular dystrophy.

“We are thrilled to support Dr. Spencer’s work, which represents a major step forward in gene therapy for LGMD 2A/R1,” said Sharon Hesterlee, PhD, Chief Research Officer, MDA. “Our partnership with C3 highlights the importance of collaboration in advancing research and finding potential cures for neuromuscular diseases. We are proud to invest in projects like these that can transform lives.”

Dr. Spencer’s work leverages state-of-the-art techniques in molecular biology and gene therapy, including the development of novel viral vectors that can efficiently target muscle cells. The ultimate goal of this research is to develop an optimized therapeutic strategy that can halt or reverse muscle degeneration in individuals with LGMD2A/R1.

“I am grateful to receive this support from MDA and C3,” said Dr. Spencer. “This grant will allow us to refine our gene therapy approach and move closer to a treatment that could significantly improve the quality of life for people living with LGMD 2A/R1.”

“We are excited to support Dr. Spencer’s groundbreaking work in advancing gene therapy for LGMD2A/R1,” said Jennifer R. Levy, PhD, Scientific Director, Coalition to Cure Calpain 3. “By funding this project, we are not only pushing the boundaries of what’s possible in genetic research but also reinforcing our commitment to finding life-altering treatments for those affected by this rare condition. This collaboration with MDA will enable us to accelerate progress and bring hope to the LGMD2A/R1 community.”