News

FDA halts Rocket Pharma's gene therapy trial as patient dies
The U.S. FDA has paused Rocket Pharmaceuticals' mid-stage gene therapy trial after a patient died from serious complications, ...
Fierce Biotech7d
Rocket crashes as gene therapy patient dies, FDA imposes hold
The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, ...
Healio7d
FDA grants rare pediatric disease designation for Stargardt disease gene therapy
The FDA granted rare pediatric disease designation for OCU410ST, a gene therapy designed to treat ABCA4-associated ...
The American Journal of Managed Care4d
Hurdles Remain Despite Progress in Gene Therapy for DMD
Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...
6abc Action News2h
Pioneering gene editing therapy could give 5-year-old with rare, fatal disorder a new chance at life
A 5-year-old boy from Center City has faced rare odds since birth and now this warrior is in a real life race to rewrite his story.
uniQure's AMT-130 Gene Therapy Warrants An Upgrade
Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...
BioSpace18h
Rare Disease, Cell and Gene Therapy at Risk From Trump Tariffs, Industry Says
In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...
Science News6d
Personalized gene editing saved a baby, but the tech’s future is uncertain
The personalized CRISPR treatment could be the future of gene therapy, but hurdles remain before everyone has access.
BioSpace7d
Rocket’s Danon Disease Gene Therapy on Hold After Patient Death
Acute systemic infection caused the patient to develop fatal capillary leak syndrome, highlighting the unpredictability of ...
Rocket Pharmaceuticals: Despite FDA Clinical Hold On RP-A501, Prospects Remain Intact
FDA places clinical hold of RP-A501 for the treatment of patients with Danon disease due to a patient death. Click here to ...
Clinical Trials Arena on MSN7d
Patient dies in Rocket’s Phase II Danon disease gene therapy trial
The FDA ordered a clinical hold on Rocket Pharmaceuticals' Danon disease trial after the adverse event was first disclosed.