Four-year data for AMT-130 show high-dose patients losing everyday function 61% more slowly than matched untreated patients, but a key composite score weakened. Here's what the results mean as the FDA reviews uniQure's application.

The FDA approved Ultragenyx's Fayuvi (UX111), an AAV9 gene therapy and the first treatment of any kind for Sanfilippo syndrome type A, often called childhood Alzheimer's. Here's how it works and what the data showed.

The FDA accepted Intellia's application for lonvo-z in hereditary angioedema with priority review, setting a March 10, 2027 decision date. It could be the first CRISPR therapy that edits genes inside the body.