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) ...

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 ...

Researchers have developed a new gene therapy that can be delivered using a nasal spray, ensuring that the therapeutic is ...

Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and ...