Sarepta's stock falls 48% after FDA critic Vinay Prasad's appointment and poor Elevidys sales. Company cuts revenue forecast ...

The FDA on Tuesday said it is planning on doing more surprise inspections of foreign factories, a day after Trump signed an ...

Vertex paused an early-stage trial of its inhaled treatment for cystic fibrosis that came from a partnership with Moderna.

Aldeyra Therapeutics reports Phase 3 success with reproxalap for dry eye disease, plans third FDA filing after previous ...

A Singapore-based startup raised a $45 million Series B to start testing a gene therapy in humans next year for a genetic ...

Vertex said it's gathering momentum with its new product launches, building out new franchises with its sickle cell therapy ...

Function Health is buying Ezra in a bid to create a haven for people who want copious amounts of data on their bodies. The ...

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J&J's gene therapy bota-vec failed Phase 3 trial for X-linked retinitis pigmentosa vision loss. Follows trend of big pharma ...

Deerfield Management is back with its third healthcare venture fund, this time securing more than $600 million to create new ...

FDA delays Stealth BioTherapeutics PDUFA date amid questions about agency disruption, while other companies like Sanofi and J ...

Initial clinical data from Genocury and EsoBiotec shows promise for in vivo CAR-T therapy, with complete remission in ...