FLT201 showed durable response and superior reduction of disease-causing substrate compared to ERT, underscoring its potential to improve upon standard of careLONDON, May 27, 2025 (GLOBE NEWSWIRE) -- ...

A new nasal spray-based gene therapy, using AAV.CPP.16 shows promise for treating lung diseases like COVID-19 and pulmonary ...

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV). To improve the AAV ...

A patient has died in a pivotal Phase II trial of Rocket Pharmaceuticals’ gene therapy for Danon disease after suffering a ...

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

US researchers have engineered a novel gene therapy to target the airway and lungs via a nasal spray. For gene therapy to ...

Find out if gene therapy has also find its way into skin diseases. With two approvals in the last 2 years, is it the start of ...

Developed by Sarepta Therapeutics, Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) ...

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's ...

While the medical world is melting down from the absolute apocalypse that is RFK Jr., it's good to celebrate that (at least ...

Researchers have developed a new viral delivery system designed efficiently to target the lungs and respiratory airways. It could help in tackling various respiratory illnesses including pulmonary fib ...

Biopharmas have been scaling back on AAV research for sometime now. Find out why and what's on the horizon for drug delivery ...