In a groundbreaking development that could herald a new era in the treatment of inherited deafness, a recent clinical trial ...

A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, ...

US Food and Drug Administration Commissioner Marty Makary said he’s trying to persuade Vinay Prasad, the agency’s former head ...

Leveraging Northway Biotech’s 20+ years of CDMO expertise, Diorasis Therapeutics is advancing AAV gene therapy for glaucoma ...

The Revell family hoped a gene therapy could buy time for their sons, who have a rare and fatal disease. After two patients ...

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 ...

An expert discusses how clinics can successfully implement gene therapy workflows by establishing strong institutional protocols, assigning dedicated clinic coordinators, and using strategies like ...

Parents of boys with Duchenne muscular dystrophy weigh in on drug innovation and medical regulation.

Choosing gene therapy Gray was in and out of hospitals seven times during each of the two years before her gene therapy. Then 32, she explained to her doctor that she was too tired to go on.

Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root ...

Just getting selected to participate in the Bespoke Gene Therapy Consortium was a major hurdle. The eight diseases, including Talia's Charcot-Marie-Tooth disease type 4J or CMT4J, which causes ...