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 ...
China Daily4h
China's gene therapy research making strides to restore sight for RP patients
Currently, GenAns Biotech is conducting investigator-initiated trials for several gene therapies, including GA001 for ...
DBusiness15h
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 ...
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, ...
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 ...
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 ...
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 ...
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 ...