It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

BETHESDA, Md. (July 16, 2025) – Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million ...

While the CRL derails a near-term clearance, one analyst described the rejection as more of “a speed bump to approval, rather ...

The firm said the agency cited chemistry, manufacturing, and controls issues in its complete response letter, but had no concerns about product quality.

A team of researchers from the University of Maryland School of Medicine (UMSOM) recently won a $250,000 prize from the ...

PS-002, Purespring’s lead precision nephrology programme, targets the complement pathway known to be a driver of IgA ...

Gene therapy can improve hearing in children and adults with congenital deafness or severe hearing impairment, a new study involving researchers at Karolinska Institutet reports.

Scientific organizations recently called for a 10-year ban on human germline editing. But will we ever be ready? And how would we even know?

A gene transfer approach to treating the bleeding disorder hemophilia B remains safe and effective long-term, as scientists from St. Jude Children's Research Hospital and University College London ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further innovations in safety, efficacy, and treatment strategies are needed.

Biotechnology New Gene Therapy Reverses Three Diseases With Shots to the Bloodstream The treatments rescued damaged blood and immune cells in newborn mice with just one shot.