Discover the current pipeline candidates advancing through the clinic for the treatment of sickle cell disease.

Sarepta Therapeutics Inc. is cutting more than one-third of its workforce and will add a black box warning label to its gene therapy for a fatal muscle disorder after two patient deaths raised doubts ...

The first patient has been dosed in a clinical trial testing Sardocor's one-time gene therapy for cardiomyopathy associated ...

An expert discusses how high-cost gene therapies require evidence standards including Phase 3 trial data showing complete response rates and durability, real-world evidence and health economic ...

An expert discusses how recent updates to AUA, EAU and NCCN guidelines have cautiously shifted toward more bladder-sparing approaches for BCG-unresponsive patients, incorporating new FDA-approved ...

The favorable market reaction indicates investor relief at management’s swift measures to rectify the company's worsening ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

Eight children in the UK have been spared from devastating genetic diseases thanks to a new three-person in vitro ...

US FDA grants fast track designation to Sanofi’s SAR446597 to treat geographic atrophy due to age-related macular degeneration: Paris Thursday, July 17, 2025, 10:00 Hrs [IST] Th ...

Sarepta stock catapulted Wednesday after the company announced a restructuring that includes 500 layoffs in the wake of two patient deaths.

Sarepta Therapeutics said it will eliminate 36% of its workforce—approximately 500 jobs—in a restructuring that follows the ...

Sarepta Therapeutics shares surged 34% in after-hours trading on Wednesday after the company said it would slash 500 jobs, or ...