Sarepta Therapeutics' Dismal Quarter Marred by Drug Failures and a Long List of Excuses

Sarepta Therapeutics' dismal quarter marred by drug failures and a long list of excuses

A long-delayed confirmatory clinical trial fails to show a benefit for patients, and sales of the company's Elevidys gene therapy were lower.

Sarepta slumps as gene therapy setback adds to drug pipeline woes - Regional Media News

(Reuters) -Shares of Sarepta sank 36% before the bell on Tuesday after a trial for two gene-targeted therapies for a muscle-wasting disease missed a key goal, deepening concerns about the company’s treatment pipeline. The latest setback comes after Sarepta’s top-selling gene therapy, Elevidys, was briefly pulled from the market in July following the death of three patients from acute liver failure. The stock has lost about 80% of its value this …

Solid plans to seek FDA guidance in 2026 for Duchenne Gene Therapy Candidate SGT-003 S

Solid Biosciences today reported an update on the progress of the INSPIRE DUCHENNE open label, Phase I/II clinical trial with SGT-003 and their planned regulatory discussions in 2026. 23 Pediatric participants have been dosed (1E14vg/kg) so far in the INSPIRE DUCHENNE trial (data cutoff 31st October). SGT-003 was generally well tolerated, there were no cases of drug-induced liver injury observed (n = 23) and day-90 biopsy data from 10 treated pa…