After a strong launch for its cancer gene therapy, Ferring Pharmaceuticals is setting the stage for long-term growth.

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

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

Sarepta Therapeutics (SRPT) stock in focus as report says company hired a Trump-linked lobbying firm after deaths linked to ...

A groundbreaking study titled "An Engineered Intravitreal Injection Retinal-Pigment-Epithelium-Tropic Adeno-Associated Virus ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development ...

A 26-year-old Downriver man seen at Children’s Hospital of Michigan in Detroit since infancy for a rare bleeding disorder in ...

For nearly three decades, Mayo Clinic researcher Christopher Evans, Ph.D., has pushed to expand gene therapy beyond its ...

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

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

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...

AAV gene therapy in early life provides lasting HIV protection in primates—offering hope for pediatric HIV intervention in ...