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 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, ...
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 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 ...
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 ...
Fierce Biotech3d
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 ...
BioSpace3d
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 ...
Becker's Hospital Review4d
Gene therapy trial reports patient death, FDA enacts clinical hold
A New Jersey biotech company has paused a gene therapy trial after a participant had an unexpected adverse event and later died from a sudden, severe infection.  Rocket Pharmaceuticals said the ...
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.
Managed Healthcare Executive13d
Breakthrough Gene Therapy Saves Infant with Rare Metabolic Disease
A personalized gene editing therapy was successfully used to treat an infant with CPS1 deficiency, a disease that causes ...
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 ...