Coalition to Cure Calpain 3 (C3) is committed to treating and ultimately curing limb-girdle muscular dystrophy, type 2A (LGMD2A), a form of calpainopathy. C3’s mission is to fund therapeutically relevant research and clinical trials as we educate the global community about this disease. Gene replacement in this primary calpainopathy (CAPN3) is currently the most direct path for therapy development. C3 seeks to understand how best to facilitate the development and rigorous evaluation of candidate gene therapies for LGMD2A and organized a Gene Therapy Workshop on May 29, 2019, to achieve that goal. The Workshop findings will guide grant reviews and associated investments in development up through an Investigational New Drug (IND) filing, as well as inform opportunities to attract industry and venture capital engagement in development.
There were 16 participants, including three representatives from C3, four consultants (three with experience in biotech/pharma), six researchers and clinicians, and three FDA staff members. While many of the participants do not study LGMD2A, they were on hand to share their extensive experience in development of gene therapy and rare diseases in other contexts, leading to a robust dialogue.
The first five presenters gave short, focused talks to provide uniform background on Calpain 3 and LGMD2A clinical phenotype, regulatory perspectives, and an overview of a gene therapy preclinical program. This was followed by facilitated interactive discussions regarding the IND-enabling path and clinical program readiness. The day concluded with a discussion of actionable steps to be taken by C3 to maximize its impact by ensuring that programs are well-informed early on, and to prepare for evaluation and facilitation of future gene therapy programs. In addition to gene therapy, C3 will continue to explore and fund other approaches that could lead to a treatment or cure for LGMD2A.