Shortly after taking reins at BioMarin, CEO Alexander Hardy laid out three paths the company could take for its struggling ...

A top US regulator plans to unveil a faster approach to approving custom gene-editing treatments, a move designed to unleash a wave of industry investment that will yield cures for patients with rare ...

Pivotal results from uniQure’s gene therapy for Huntington’s disease have brought new light to patients who have known only ...

In 1962, a pediatric neurology resident at Columbia's Neurological Institute and his colleagues in the College of Physicians ...

Columnist Jennifer Lynne concludes a series on Domenic Catrine, whose life has improved after receiving gene therapy to treat hemophilia B.

DURHAM, N.C. (WTVD) -- Duke Eye Center has become the first academic medical center in the country to implant a new cell-based gene therapy for a rare eye disease called MacTel. This procedure offers ...

Just as Adverum Biotechnologies' cash reserves were running dry, Eli Lilly has swooped in | Just as Adverum’s cash reserves ...

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

Some of the most expensive drugs currently in use are gene therapies to treat specific diseases, and their high cost limits ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

Cleveland Clinic Children’s researchers have uncovered a genetic link to osteosarcoma, the most common bone cancer in ...

The U.S. Food and Drug Administration has placed a clinical hold on Intellia Therapeutics' two late-stage trials testing an ...