NEW YORK – The Muscular Dystrophy Association on Monday said it was working with the University of California, Davis, to launch the first project under the MDA Kickstart for Ultra-Rare Neuromuscular Disease program, an in-house effort to develop gene therapies for seldom-seen genetic conditions.
The aim of the program is to de-risk development of ultra-rare gene therapies that drugmakers may not otherwise want to invest in due to their small market potential. The Muscular Dystrophy Association said it aims to work with academic, corporate, and community groups to improve the valuation of these therapies and "bridge the translational gap between early academic science, regulatory approval, access, and use."
Additionally, Forge Biologics will be the manufacturing partner in this program.
The MDA Kickstart program engaged an external group of gene therapy advisers to review proposals and pick projects focused on diseases with high unmet medical need that could benefit from gene therapies. In the first project, Ricardo Maselli, professor of neurology and clinical neuroscience at UC Davis, will lead efforts to develop a gene therapy for congenital myasthenic syndrome caused by mutations in the CHAT gene, which affects approximately 200 children and adults in the US.
This disease "can be easily diagnosed with blood genetic testing in newborns and infants," Maselli said in a statement. "Unfortunately, in many cases, the disease remains undiagnosed until adulthood."
The CHAT gene encodes choline acetyltransferase, an enzyme that is critical for synthesizing acetylcholine, a neurotransmitter that helps electrical signals reach nerves and muscles. When this process is disrupted due to CHAT gene mutations, it causes muscle weakness and respiratory arrest, which can be fatal.
Within this project, the Muscular Dystrophy Association will make its in-house regulatory and drug development expertise available to Maselli and his team so they can build the platform, personnel, and other resources necessary to conduct preclinical research and submit data to the US Food and Drug Administration for a pre-investigational new drug application meeting to discuss plans to move the therapy toward clinical trials.
Forge Biologics will provide the MDA Kickstart program with its adeno-associated virus process and analytical development manufacturing services, including its proprietary HEK293 suspension Ignition Cells and pEMBR adenovirus helper plasmid. The gene therapies developed in this program will be manufactured at Forge's facility in Columbus, Ohio.
The Muscular Dystrophy Association has also applied for orphan drug and rare pediatric disease designation for this investigational gene therapy for CHAT-mutated congenital myasthenic syndrome. With rare pediatric disease designation comes the chance to receive a priority review voucher, an important incentive for developing rare disease drugs that is up for reauthorization by Congress. The association is urging the public to contact legislators and ask them to pass the Creating Hope Reauthorization Act, so the rare pediatric disease priority review voucher program can continue.