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 ...

Some oncologists and researchers have raised concerns about delays in the completion of confirmatory trials, but FDA now has new tools to push them ahead.

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 ...

One analyst speculated that, going forward, Prasad may be less “heavy-handed” in reviewing rare disease therapies given the ...

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