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.

In his first 100 days as commissioner of the Food and Drug Administration, Marty Makary has made a handful of splashy ...

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.

A team of researchers from the San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET, Milan), led by Nadia Coltella and Luigi Naldini, has unveiled a powerful strategy to rejuvenate the ...

A new gene therapy has shown promising results in restoring hearing to children, teens, and even a young adult born with a rare form of inherited deafness.

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. Hearing improved ...

Deaf people have had their hearing restored thanks to groundbreaking gene therapy. Researchers found the state-of-the-art technique can improve hearing in children and adults with congenital ...