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

The U.S. Food and Drug Administration has declined to approve Ultragenyx Pharmaceutical's experimental gene therapy to treat ...

This is the first time such results have been achieved in both children and adult patients born with a specific type of ...

The deal gives AstraZeneca’s rare disease unit Alexion access to specialized capsids developed by the Japanese biotech JCR ...

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

The FDA granted orphan drug designation to Sineugene Therapeutics' SNUG01, a first-in-class experimental gene therapy for ...

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

A Northeast Ohio woman and a 22-year-old man from UAE are among the first patients to receive the innovative CRISPR treatment.

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

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 ...

Ferring Pharmaceuticals officially opened doors to its state-of-the-art gene therapy manufacturing facility in Parsippany ...