News

FDA’s Reversal on Gene Therapy Helps My Son
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
DBusiness7h
Children’s Hospital Administers First Commercially Available Gene Therapy for Hemophilia B
A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...
Sarepta reportedly hired Trump-linked lobbying firm after gene therapy deaths
Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...
GEN - Genetic Engineering and Biotechnology News5d
Gene Therapy Companies Set Sights on Cardiovascular Disease
The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...
FDA Chief Says He Wants Gene Therapy Head Prasad to Return
US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...
Two boys died after a gene therapy. This family won’t give up hope.
The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...
IFLScience on MSN2d
World-First Gene Therapy Improves Vision For Man With Rare, Previously Untreatable Form Of Blindness
A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has ...
The American Journal of Managed Care6d
Progress and Challenges With Gene Therapy in Hereditary Hearing Loss
Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...
Sarepta will resume gene therapy shipments after FDA review of recent patient death
Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...
Fierce Biotech12d
'We will crack gene therapy': Elevidys fallout doesn't dent Roche's hopes for modality
Roche’s move came against a backdrop of wider industry retreat from gene therapy, with Pfizer pulling its FDA-approved ...
Fierce Pharma5d
Untangling Sarepta’s gene therapy fallout and a growing trust deficit
Over a whirlwind three weeks, Sarepta Therapeutics has faced tough safety questions around its commercial gene therapy ...
X-SCID gene therapy gives scientists a rare glimpse into early immune system
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune ...