In the past two years, a spate of technological advances and promising results in mice have been laying the groundwork for ...

Sarepta sells $174M in Arrowhead shares to fund a $100M milestone payment as part of its RNAi drug collaboration.

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...

The stock has been in free fall since safety issues derailed a gene therapy analysts had expected to achieve blockbuster ...

Jefferies analysts said these detailed safety outcomes confirm the gene therapy's positive risk/benefit profile in ambulatory ...

With a strong launch underway for a bladder cancer gene therapy, Ferring Pharmaceuticals is finding the kind of commercial ...

Vinay Prasad is once again head of the FDA’s Center for Biologics Evaluation and Research, after Laura Loomer led a campaign ...

In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some ...

Sarepta Therapeutics said Friday that the deaths of two patients taking its marketed gene therapy Elevidys® (delandistrogene ...

Less than weeks after stepping down, Prasad will return to lead CBER “at the FDA’s request,” according to an HHS spokesperson ...

Sarepta reported a quarter-on-quarter dip in sales for its Duchenne gene therapy. Elsewhere, Jazz won FDA approval of a brain cancer drug and Novo decided to advance a Prothena medicine into Phase 3.

Sarepta Therapeutics Inc.’s shares soared after the US biotech reported revenue that beat estimates, which analysts ...