WASKYRA (etuvetidigene autotemcel), which received FDA approval in December 2025, represents a shift in how rare disease treatments reach the market. Developed by the Italian biomedical charity Fondazione Telethon, the therapy is being commercialized through a non-profit collaboration with Orphan Therapies. It is designed for pediatric patients aged six months and older, as well as adults, who carry a mutation in the WAS gene and require hematopoietic stem cell transplantation.

Wiskott-Aldrich Syndrome is an ultra-rare, life-threatening immunodeficiency marked by severe bleeding complications, recurrent infections, and an increased risk of malignancy. The treatment works by modifying a patient’s own stem cells ex vivo to introduce a functional copy of the WAS gene, effectively addressing the root cause of the condition. By selecting UCSF Benioff Children's Hospitals as the inaugural site, the organizations are beginning the rollout of a broader U.S. network intended to support patient access for those with ultra-rare genetic diseases.