MDA Kickstart Program Launches for Ultra-Rare Neuromuscular Disease Drug Development
MDA launches Kickstart program for ultra-rare neuromuscular disease drug development, collaborating with UC Davis and Forge Biologics. Focusing on gene therapy for Congenital Myasthenic Syndrome (CMS) caused by CHAT gene mutations, the program aims to bridge the gap between early science and FDA approval, leveraging Forge's AAV manufacturing services. MDA seeks Rare Pediatric Disease Priority Review Voucher reauthorization to support these efforts.
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MDA launches Kickstart program for ultra-rare neuromuscular disease drug development, collaborating with UC Davis and Forge Biologics. Focusing on gene therapy for Congenital Myasthenic Syndrome (CMS) caused by CHAT gene mutations, the program aims to bridge the gap between early science and FDA approval, leveraging Forge's AAV manufacturing services. MDA seeks Rare Pediatric Disease Priority Review Voucher reauthorization to support these efforts.
MDA launches MDA Kickstart for ultra-rare neuromuscular disease, focusing on gene therapy development for conditions with minimal commercial interest. The program aims to bridge the gap between academic research and FDA approval, starting with a gene therapy for Congenital Myasthenic Syndrome caused by CHAT gene mutations. MDA collaborates with UC Davis and Forge Biologics for project development and manufacturing, respectively, and seeks Rare Pediatric Disease Priority Review Voucher reauthorization.
UC Davis neurologist Ricardo Maselli will lead the MDA Kickstart for Ultra-Rare Neuromuscular Disease project, aiming to develop gene therapy for congenital myasthenic syndrome (CMS) caused by CHAT gene mutations. The MDA Kickstart program addresses the gap in commercial interest for ultra-rare neuromuscular diseases, ensuring patients with such conditions benefit from gene therapies.