FDA Adds Strongest Warning to Sarepta Gene Therapy Linked to 2 Patient Deaths

The Food and Drug Administration is adding a new warning to a gene therapy linked to two patient deaths earlier this year ...

US FDA adds strongest warning to Sarepta gene therapy after fatal liver injuries

The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics' gene therapy Elevidys ...

Eli Lilly signs deal for MeiraGTx's gene therapy for severe eye disease

MeiraGTx Holdings has signed a deal with Eli Lilly , potentially worth over $475 million, granting the pharma giant rights to ...
MedPage Today on MSN

Gene Therapy Gets FDA's Strongest Warning After Fatal Liver Injuries

The FDA added a boxed warning to delandistrogene moxeparvovec (Elevidys) gene therapy for Duchenne muscular dystrophy and ...

Krystal Biotech: Vyjuvek Gene Therapy Well Positioned For Growth

Krystal Biotech drives Vyjuvek growth, premium margins, global expansion, a $1B goal, and pipeline data ahead with a $219.50 ...

Researchers to test gene therapy for rare diseases with a new FDA trial protocol

Earlier this year, researchers at Children's Hospital of Philadelphia and Penn created a first-of-its-kind drug customized to ...
BioSpaceOpinion

Opinion: How the FDA Is Accelerating Cell and Gene Therapy’s Next Chapter

Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, ...

Penn, CHOP will test gene therapy for rare diseases with new FDA trial protocol

Penn and CHOP researchers want to develop urea cycle disorder treatments using CRISPR gene-editing therapy. Get unlimited access to Inquirer.com and The Inquirer App, plus 5 articles each month to ...
STAT

Makary, Prasad outline pathway for bespoke gene therapy

FDA Commissioner Marty Makary and biologics chief Vinay Prasad have sketched out the agency’s first real blueprint for ...
News Medical on MSN

Fondazione Telethon announces CHMP positive opinion for Waskyra™, a gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS)

Fondazione Telethon, an Italian biomedical charity committed to advancing research on rare and complex genetic diseases, announces the positive opinion issued by the Committee for Medicinal Products ...