Mar 26, 2026
Avidity Biosciences announced promising Phase 1/2 trial results for its myotonic dystrophy type 1 drug delpacibart etedesiran, showing 40% reduction in DMPK mRNA and functional improvements. The therapy has received multiple regulatory designations and is now in Phase 3 testing with data expected in late 2026. The company's stock has surged 128% over the past year despite reporting a net loss of approximately $550 million.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 06, 2026
Dyne Therapeutics is preparing a U.S. Accelerated Approval submission for z-rostudirsen in Duchenne muscular dystrophy and gearing up for a Phase 3 trial of z-basivarsen in myotonic dystrophy, while scaling operations to support potential commercialization.
Mar 05, 2026
The FDA has placed a partial clinical hold on PepGen's Phase 2 FREEDOM2-DM1 trial for myotonic dystrophy type 1, citing questions about preclinical pharmacology and toxicology studies. The company is submitting additional analyses to address the FDA's concerns.
Feb 19, 2026
Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.
Feb 17, 2026
Japan's Ministry of Health, Labour and Welfare granted Orphan Drug designation to Dyne Therapeutics' investigational treatment zeleciment-basivarsen for myotonic muscular dystrophy type 1, adding to existing designations in the U.S. and Europe.
Feb 13, 2026
Vertex Pharmaceuticals reported fourth quarter 2025 revenue of $3.19 billion, up 10% year-over-year, and provided full year 2026 revenue guidance of $12.95 billion to $13.1 billion with over $500 million expected from non-CF products.
