Fierce Biotech
After patient death, FDA lifts hold on 1 of 2 late-stage trials testing Intellia's CRISPR therapy
The hold followed Intellia’s voluntary pause in response to a grade 4 liver event—a case of Hy’s Law—in the ATTR-CM trial.
Yahoo
Pfizer says patient dies after receiving hemophilia drug in trial
Add Yahoo as a preferred source to see more of our stories on Google. FILE PHOTO: A Pfizer logo is displayed at a research facility in the La Jolla neighborhood of San Diego, California, U.S., ...
BioSpace
Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
GlobalData on MSN
FDA puts REGENXBIO gene therapy trials on hold after brain tumour
A five-year-old patient dosed with RGX-111 has developed a brain tumour four years after being dosed with the gene therapy.
MedPage Today
Gene Therapy Shows Signs of Delayed Progression in Fatal Neurodegenerative Disease
A single intravenous infusion of adeno-associated virus serotype 9 encoding beta-galactosidase (AAV9-GLB1) was associated with short-lived adverse events, increased beta-galactosidase levels, and ...
STAT
FDA halts gene therapy trials after brain tumor found in patient
The Food and Drug Administration paused trials for two experimental gene therapies from Regenxbio after one child developed a ...
Fierce Biotech
UniQure pauses Fabry gene therapy dosing in 2 groups after toxicities emerge
Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...
MedPage Today on MSN
FDA Halts Two Gene Therapy Trials After Child Develops Brain Tumor
Studies of Hunter and Hurler syndromes on hold ...