News

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 ...
Sarepta reportedly hired Trump-linked lobbying firm after gene therapy deaths
Sarepta Therapeutics (NASDAQ:SRPT) has hired a lobbying firm with close ties to the Trump administration as regulatory ...
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 ...
GEN - Genetic Engineering and Biotechnology News6d
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, ...
DBusiness1d
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 ...
EurekAlert!2d
Breakthrough gene therapy for neovascular age-related macular degeneration shows promise in animal models and patients
A groundbreaking study titled "An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus ...
FDA’s Reversal on Gene Therapy Helps My Son
Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.
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 ...
Sarepta says it won't comply with FDA request to stop shipping gene therapy Elevidys
U.S. regulators asked Sarepta Therapeutics on Friday to voluntarily halt shipments of its Elevidys gene therapy after a ...
Live Science2y
Gene therapy: What is it and how does it work? - Live Science
Gene therapy has been headline news in recent years, in part due to the rapid development of biotechnology that enables doctors to administer such treatments. Broadly, gene therapies are ...
USA Today1y
FDA considers gene therapy for sickle cell disease: Cure at a cost.
Gene therapy ‒ which has the potential to cure, not just treat disease ‒ has been approved so far only for very rare blood, neuromuscular and eye diseases, though more than 589 clinical trials ...
Roche pauses shipments of Elevidys gene therapy outside US
Roche Holding AG said on Tuesday it has paused shipments of muscular disorder gene therapy Elevidys in some countries outside the U.S., echoing a similar decision by U.S. partner Sarepta Therapeutics ...