Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the ...

Data from the EMBARK trial suggest patients may have functional improvements taking the gene therapy between 8 and 9 years old.

Doctors will have to monitor the boy for years to ensure the experimental treatment is effective long-term. But for now, it's ...

A hot (if cautious) topic at the big gene therapy meeting: what Vinay Prasad's appointment might mean for approvals in rare ...

Though it may be a while before similar personalized treatments are available for others, doctors hope the technology can ...

Sarepta Therapeutics is meeting with the FDA in June to discuss expanding the use of its Duchenne muscular dystrophy gene ...

KJ Muldoon became the first patient to undergo personalized CRISPR treatment, a therapy that found the one uniquely mutated gene out of 20,000 in his little body, and fixed it.

Since Elevidys' accelerated approval in 2023, experts have been clamoring for more data, particularly in older and ...

Rocket Pharmaceuticals has shared early data from three patients that tie its gene therapy for an inherited heart disease to ...

The UNC technology, developed by Pediatrics Professor Chengwen Li, M.D., Ph.D., uses adeno-associated viruses (AAVs) to ferry ...

GlobalData analysts have tipped the global market for genomic therapies to balloon to almost $90 billion by 2030, driven by ...