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, ...
Rocket Pharmaceuticals Stock Craters After Gene Therapy Patient Dies
Rocket Pharmaceuticals shares plummeted 60% Tuesday after the firm said it halted a study of a gene-therapy drug for Danon ...
FDA halts Rocket Pharma's gene therapy trial as complications lead to patient death
The U.S. FDA has paused a mid-stage gene therapy trial by Rocket Pharmaceuticals after a patient suffered serious ...
The American Journal of Managed Care2d
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 ...
Fierce Biotech5d
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, ...
Healio5d
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 ...
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 ...
BioSpace5d
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 ...
Ocugen's Retina Gene Therapy Drug Gets Rare Disease Designation From FDA
Ocugen's drug to treat retina diseases received a rare pediatric disease designation from U.S. regulators. The biotechnology said Tuesday that the Food and Drug Administration granted the designation ...
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 ...
Science News4d
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.