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 ...
Fashion Glamp on MSN3d
Revolutionary Gene Therapy Restores Hearing for Deaf Children
In a groundbreaking development that could herald a new era in the treatment of inherited deafness, a recent clinical trial ...
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, ...
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 ...
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.
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 ...
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 ...
Diorasis Therapeutics and Northway Biotech Announce Partnership for the Development and Manufacturing of AAV Gene Therapy for Glaucoma
Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...
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 ...
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 ...