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UCSF Benioff Children's Hospitals: A Breakthrough in Gene Therapy

In a groundbreaking development for pediatric medicine, UCSF Benioff Children's Hospitals have been recognized as the first U.S. Qualified Treatment Center (QTC) authorized to administer WASKYRA®, an innovative gene therapy designed for treating Wiskott-Aldrich Syndrome (WAS). This historic designation is a significant leap forward in providing specialized medical care for patients grappling with this rare, life-threatening immunodeficiency.

Understanding Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome is characterized by a triad of symptoms: thrombocytopenia (low platelet count), eczema, and recurrent infections. Caused by mutations in the WAS gene, this ultra-rare condition presents patients with severe health challenges, making it critical for them to receive timely and effective treatment. WASKYRA® (etuvetidigene autotemcel) signifies a new era for therapeutic options, being the first gene therapy approved in the U.S. aimed specifically at this condition.

A Pioneering Partnership

This achievement is not only a milestone for UCSF but also a testament to the collaboration between Fondazione Telethon, an Italian biomedical charity, and Orphan Therapies, a subsidiary of the Orphan Therapeutics Accelerator (OTXL). With over 35 years dedicated to researching rare diseases, Fondazione Telethon developed WASKYRA® and facilitated its journey to approval by the U.S. Food and Drug Administration (FDA) in December 2025. This therapy, built on a non-profit collaboration approach, is designed to provide patients with a much-needed lifeline.

Accessing WASKYRA®

The therapy is intended for pediatric patients aged six months and older, as well as adults who have a mutation in the WAS gene and for whom traditional hematopoietic stem cell transplantation is appropriate but not feasible due to the unavailability of a suitable HLA-matched donor. By utilizing a patient’s own stem cells, which are genetically modified to restore the production of the WAS protein, WASKYRA® addresses the underlying issue causing the syndrome.

The Impact of UCSF's Designation

UCSF Benioff Children's Hospitals' designation as the first QTC for WASKYRA® is a vital step toward the establishment of a nationwide clinical network focused on the provision of specialized care for patients with rare genetic disorders. According to Ilaria Villa, CEO of Fondazione Telethon, this partnership reflects the evolution of their mission to translate scientific research into tangible therapies that can reach those in need.

Beth White, Chief Commercial Officer of Orphan Therapies, emphasizes the importance of creating reliable access to critical treatments for ultra-rare conditions. The collaboration with Fondazione Telethon on WASKYRA® serves as a model for future initiatives designed to meet the challenges of delivering essential therapies to patients.

Looking Ahead

As WASKYRA® becomes commercially available, more treatment centers across the U.S. are anticipated to join the network in the upcoming months. This initiative not only highlights UCSF's cutting-edge expertise in treating rare genetic disorders but also marks an essential beginning for enhanced patient care in this specialized field. With innovative approaches and multidisciplinary collaboration at the forefront, UCSF Benioff Children's Hospitals is set to make strides in advancing treatments for Wiskott-Aldrich Syndrome and other rare genetic diseases.

For ongoing updates about WASKYRA® and treatment availability, interested parties can visit the dedicated website Waskyra.com for more information.