Solid Restarts DMD Gene Therapy Trial, With Encouraging Interim Data To Back It Up
The Phase I/II trial testing SGT-001 in patients with Duchenne muscular dystrophy resumed after FDA lifted a clinical hold on the trial; interim data from the first six patients were encouraging.
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Public Company Edition: Instil Bio, Connect Biopharma, Finch Therapeutics, Gain Therapeutics and four heath care-focused SPACs launched initial public offerings; Endo raised $1.3bn via note sale; and Rubius Therapeutics, Solid Biosciences and others priced follow-on offerings.
Any time advantage Sarepta's Duchenne muscular dystrophy gene therapy may have had over rivals, notably Pfizer, has disappeared after SRP-9001 failed to significantly improve muscle function in a closely watched trial.
The US FDA has for the second time placed a clinical hold on Solid Biosciences’ potential gene therapy for Duchenne muscular dystrophy, a move linked to a 70% decline in the company’s share price. The company had reported a serious adverse event in a patient treated with higher doses of the viral vector-delivered gene therapy.