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.
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 brain tumor, the company announced Wednesday.
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 ...
Fierce Biotech
FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned'
After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...