Muscular Dystrophy Association Launches Initiative to Support the Development of Treatments for Ultra-Rare Neuromuscular Diseases

The Muscular Dystrophy Association (MDA) announced the launch of the MDA Kickstart Program, an initiative to advance the development of treatments for ultra-rare neuromuscular diseases. The first project under the MDA Kickstart Program will focus on the development of a gene therapy for a rare form of congenital myasthenic syndrome (CMS) and will involve collaboration from a team at the UC Davis Institute for Regenerative Cures.

The MDA Kickstart Program was designed to support the transition from academic research to the commercial drug development pipeline especially for the significant number of neuromuscular conditions that affect fewer than 1000 people in the United States. Treatments for these ultra-rare neuromuscular conditions have limited commercial value and therefore face barriers in the path towards regulatory approval and patient access. To support this initiative, the MDA has partnered with Forge Biologics (Columbus, OH) for assistance in adeno-associated virus (AAV) gene replacement therapy development.

Sharon Hesterlee, PhD, Chief Research Officer at the MDA, stated, “The MDA Kickstart program will help ensure that patients with ultra-rare forms of neuromuscular disease are not left behind in the era of genetic medicines and can also experience the potential benefits of gene therapies.”

The first MDA Kickstart Program project aims develop a gene therapy for CMS caused by mutations of the CHAT gene, a condition that affects approximately 200 people in the United States. This project was developed by Ricardo A. Maselli, MD, Professor of Neurology and Clinical Neuroscience at UC Davis, and was selected by an external group of leaders in gene therapy from industry and academia.

More information about the MDA Kickstart Program is available on the MDA website: https://www.mda.org/research/kickstart