Also known as: Sarepta, Sarepta Therapeutics
Sarepta Therapeutics is a precision genetic medicine company focused on RNA and gene therapy approaches for rare diseases. Its core programs include Duchenne muscular dystrophy and other neuromuscular indications.
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Sarepta Therapeutics said it will announce first quarter 2026 financial results. The event will be webcast on its investor relations website, with a replay archived for one year.
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.
Ultragenyx Pharmaceutical confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
PTC Therapeutics withdrew its resubmitted new drug application for Translarna in Duchenne muscular dystrophy after the FDA indicated the data was unlikely to meet the threshold for substantial evidence of effectiveness, marking the third unsuccessful US approval attempt.
PTC Therapeutics has withdrawn its New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy following FDA feedback that the data are unlikely to meet the threshold for substantial evidence of effectiveness.
