FDA advisors narrowly back Sarepta’s Duchenne gene therapy

An FDA advisory committee has given tentative backing to Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD), even though the agency’s reviewers said they were no

Solid Biosciences acquires AavantiBio, conducts $75 million ...

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a

Myocarditis case mars Sarepta DMD gene therapy readout

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety.

Sarepta says early filing for DMD gene therapy may be back o...

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file fo

Pfizer pauses Duchenne gene therapy trial after patient deat...

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient.

Solid Bio hit as FDA slaps clinical hold on DMD gene therapy