Andrzej Rostek The U.S. FDA has selected Moderna's ( NASDAQ: MRNA ) mRNA therapeutic mRNA-3705 for methylmalonic acidemia ("MMA") due to methylmalonic-CoA mutase ("MUT") deficiency for a pilot program designed to accelerate development of rare disease drugs.
mRNA-3705, which is currently in a phase 1/2 study, is designed to treat MMA , an inherited metabolic disorder that is most commonly caused by a deficiency in the mitochondrial enzyme MUT that can lead to a toxic buildup of acids in the body and eventually into multi-organ disease. The Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program, which began in September 2023, gives drugmakers more frequent and rapid interactions with the FDA and will help to generate data intended to support candidate applications to the agency. In late May, the FDA also chose Larimar Therapeutics' ( LRMR ) nomlabofusp for Friedreich's ataxia for the program.
More on Moderna Moderna: Betting On Bird Flu Rally Is A Poor Strategy For A Value Investor Bird flu death in Mexico linked to strain not seen in humans before FDA advisors unanimously agree on COVID JN.1-lineage vaccine for fall Moderna, Inc. (MRNA) Moderna Oncology Investor Event (Transcript) Moderna, Inc.
(MRNA) Bernstein's 40th Annual Strategic Decisions Conference (Transcript).
