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UCSF Benioff Children's Hospitals Becomes First Qualified Center for WASKYRA®

In a landmark achievement, UCSF Benioff Children's Hospitals has been designated as the first Qualified Treatment Center (QTC) in the United States to administer WASKYRA® (etuvetidigene autotemcel), the pioneering gene therapy aimed at treating Wiskott-Aldrich Syndrome (WAS). This development represents a significant advancement in the fight against this ultra-rare genetic disorder that has debilitating consequences for those affected.

A Collaborative Milestone

This partnership marks a crucial step in improving access to innovative therapies for patients suffering from rare genetic diseases. The initiative was announced on September 28, 2026, through a collaboration between Fondazione Telethon, an Italian biomedical charity with decades of experience in genetic research, and Orphan Therapies, a non-profit biotech firm dedicated to enhancing availability of therapies for severe, rare conditions.

WASKYRA®, the first gene therapy approved in the United States for treating Wiskott-Aldrich Syndrome, was greenlit by the U.S. Food and Drug Administration (FDA) back in December 2025. This therapy signifies a monumental leap in the commercialization of advanced treatments, especially as it was developed through a non-profit collaboration.

Understanding Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome is a life-threatening immunodeficiency caused by mutations in the WAS gene. The disease typically manifests in early childhood and is characterized by a triad of symptoms: thrombocytopenia (low platelet count), eczema, and recurrent infections. Patients also face an increased risk of autoimmune diseases and malignancies, making early and effective treatment crucial.

UCSF's Expertise

UCSF Benioff Children's Hospitals stands as a leading institution in pediatric care, equipped with specialized knowledge in managing genetic and immunologic disorders. By becoming a Qualified Treatment Center, UCSF Health is set to enhance the clinical framework necessary for delivering WASKYRA® to eligible patients aged six months and older, as well as adults requiring this revolutionary gene therapy.

Ilaria Villa, the CEO of Fondazione Telethon, commented on the implications of this partnership, stating, "This milestone and partnership with Orphan Therapies reflect the ongoing and successful evolution of Fondazione Telethon's model, originally designed to bridge scientific research and tangible therapeutic opportunities for patients."

Future of Gene Therapy

The activation of UCSF Health as a QTC establishes not just immediate access to WASKYRA® for US patients, but also lays the groundwork for a broader clinical network tailored to serve individuals with ultra-rare conditions across the country. The collective aim is to ensure that those in need receive effective treatment promptly, overcoming existing barriers to access.

Beth White, Chief Commercial Officer of Orphan Therapies, emphasized the ethos behind this initiative, stating, "Our purpose is to provide reliable and sustainable commercial access to vital treatments for very rare conditions. Collaboration and innovation are key in addressing the prevalent market challenges that hinder the delivery of much-needed therapies to patients."

As the medical community continues to explore innovative solutions for complex genetic diseases like Wiskott-Aldrich Syndrome, UCSF Benioff Children's Hospitals finds itself at the forefront of a critical movement aimed at bridging the gap between groundbreaking research and accessible patient care. The introduction of WASKYRA® marks not just a new treatment option, but a beacon of hope for families facing the challenges of genetic disorders.