There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University’s College ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

There is no cure for the rare disease Hereditary Spastic Paraplegia (HSP), but researchers from Drexel University's College of Medicine and the UMass Chan Medical School have achieved ...

Nonprofit founder and CEO, Craig Martin, notes that the industry will likely see more alternative gene therapy commercialisation models in the future.

Editorial: Cutting-edge therapies exist, but the market cannot deliver them cheaply. Britain must build NHS capacity so that cures become collective goods, not expensive products ...

Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

For all of the political chaos that American science endured in 2025, aspects of this country’s research enterprise made it ...

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

AMD Phase 3 timing, Otsuka funding to 2028, and mixed 4D-710 data with financial risk. See why FDMT stock is a hold.

Cell and gene therapy (CGT) has evolved from a niche research pursuit into a central force in biopharmaceutical innovation. By engineering living cells and genetic material to repair or replace faulty ...

Major is one of the first pediatric patients in Florida to receive gene therapy for sickle cell disease since the FDA approved it for that use. But it was a long road. First, he had to undergo ...