Clinical-stage biotechnology company Jaguar Gene Therapy has completed dosing in Cohort I of its first-in-human clinical ...

Rare disease drug developers struggle to survive in a biopharma investment market that prioritizes large patient populations.

Through an AI-enabled platform, the partnership between two non-profit groups seeks to bring overlooked cell and gene ...

The U.S. Court of Appeals for the Federal Circuit (CAFC) in a precedential decision on Friday reversed a district court’s ...

Real-world evidence links higher injection frequency to better VA outcomes and reduced CST fluctuations, reinforcing ...

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to treat the rare condition Fabry disease. In the phase 1/2 study, two ...

A patient in a clinical trial for a Regenxbio gene therapy developed a brain tumor, leading the FDA to place the study under a clinical hold. This pause has implications for another Regenxbio gene ...

New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be presented at WORLDSymposium™ 2026 NOVATO, Calif., Jan. 30, 2026 (GLOBE ...

Company expects up to six-month review period per FDA guidelines New longer-term clinical data demonstrating durable positive brain biochemical and clinical effect for as long as 8.5 years to be ...

The quest to restore hearing and balance function has long been a central challenge in medicine, particularly for genetic forms of inner ear disorders that have no effective cure. While treatments ...

AI-generated summary reviewed by our newsroom. Read our AI Policy. AskBio built an AAV gene-delivery platform and attracted investor frenzy in 2020 FDA approved first AAV therapy in 2017, validating ...

(RTTNews) - uniQure N.V. (QURE) today announced that a Type A meeting with the FDA has been scheduled to discuss the Biologics License Application (BLA) data package intended to support accelerated ...