Vertex Pharmaceuticals has discontinued all research on adeno-associated virus (AAV) vector technology, impacting partnerships with Affinia Therapeutics and Tevard Biosciences focused on Duchenne muscular dystrophy treatments.
Ultragenyx reported preliminary 2024 revenue of $555 million to $560 million, exceeding previous guidance, driven by strong sales of Crysvita and Dojolvi.
The FDA has granted priority review to Ultragenyx's Biologics License Application (BLA) for UX111, a gene therapy for Sanfilippo syndrome type A (MPS IIIA).