UCSF Benioff Children's Hospitals First in US to Administer Gene Therapy WASKYRA®

Gene therapy WASKYRA® has become the first in the US approved to treat Wiskott-Aldrich Syndrome (WAS), offering hope for patients with this ultra-rare, life-threatening immunodeficiency. This therapy is now available at UCSF Benioff Children's Hospitals, recognized as the first US Qualified Treatment Center (QTC) for WASKYRA®. Developed by Fondazione Telethon and commercialized by Orphan Therapies, WASKYRA® marks a milestone in non-profit biotech collaboration.

Wiskott-Aldrich Syndrome, caused by mutations in the WAS gene, is characterized by severe immunological and bleeding complications. Given its rarity, the availability of WASKYRA® provides a crucial option for affected individuals, especially when traditional stem cell transplantation isn't feasible.

UCSF Benioff Children's Hospitals' designation as QTC sets a precedent for establishing a US clinical network, facilitating access to specialized treatments for rare genetic and immunological conditions. As more centers are expected to join, this initiative underscores an evolving model in translating research into accessible treatments.