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 Biotech4d
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, ...
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 ...
The American Journal of Managed Care1d
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 ...
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 ...
BioSpace4d
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 ...
Fierce Biotech2d
Intellia's stock sinks on gene therapy patient's raised enzyme levels, but analysts keep faith
Intellia Therapeutics’ stock may have taken a hit in the wake of details of a grade 4 adverse event in a phase 3 trial, but ...
BioPharma Dive5h
Full Arvinas, Pfizer data confirm potential and limits of ‘Protac’ drug in breast cancer
While vepdegestrant outperformed a standard treatment option in a subtype of patients, the benefit on progression-free ...
BioSpace2d
Analysts Brush Off Intellia’s Adverse Event but Gene Therapy Safety Concerns Continue
While an adverse event reported in Intellia’s gene therapy trial was a “non-concern” for analysts, it follows a handful of ...
Science News3d
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.
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 ...
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 ...