A 51-year-old man died last month after receiving an experimental treatment from Sarepta, the third death this year tied to ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy ...

Tiny fat bubbles carrying gene therapy have successfully repaired DNA in the lungs and liver of animals with alpha-1 ...

Sarepta Therapeutics (NASDAQ:SRPT) said a third patient has died from acute liver failure after receiving one of its gene therapies, intensifying scrutiny over the safety of its treatments, Bloomberg ...

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

AAVB-039 advances gene therapy for Stargardt disease, aiming for innovative treatments for inherited retinal disorders.

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 ...

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 FDA granted fast track designation to SAR446597, an intravitreal gene therapy for the treatment of geographic atrophy due to age-related macular degeneration, according to a press release from ...

The US Food and Drug Administration (FDA) recently granted fast track designation to Sanofi’s SAR446597 for the treatment of geographic atrophy (GA). 1 ...

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