FiercePharma
FDA restricts Sarepta's gene therapy Elevidys after Duchenne patient deaths
The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...
The Boston Globe
Intellia tumbles after patient dies following Crispr treatment
Intellia Therapeutics Inc. shares dropped after the company said a patient died after suffering liver damage in a clinical trial for the company’s gene-editing treatment. The patient, who was in his ...
University of California
Gene therapy delivers lasting immune protection in children with rare disorder
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...