UCSF Benioff Children's Hospitals Launch First U.S. Approval for WASKYRA® Gene Therapy

Breakthrough in Gene Therapy: UCSF Benioff Children's Hospitals

The UCSF Benioff Children’s Hospitals have made a significant milestone in the treatment of rare genetic disorders by becoming the first center in the United States to be certified for the administration of WASKYRA®, a personalized gene therapy for Wiskott-Aldrich Syndrome (WAS). This unprecedented step marks a new era in medical innovation, enhancing therapeutic options for patients suffering from this rare immunodeficiency syndrome.

What is Wiskott-Aldrich Syndrome (WAS)?

The Wiskott-Aldrich Syndrome is an extremely rare and potentially life-threatening immunodeficiency disorder caused by mutations in the WAS gene. This illness is characterized by a triad of symptoms including thrombocytopenia (low platelet count), eczema, and recurrent infections. Patients with WAS often face complications such as autoimmune disorders and malignancies, making timely and effective treatment crucial.

WASKYRA® (etuvetidigene autotemcel) offers hope where conventional therapies may fall short. It has been specifically designed to provide a new treatment pathway for pediatric and adult patients with WAS who do not have a compatible donor for hematopoietic stem cell transplantation (HSCT).

The Partnership Leading the Charge

The introduction of WASKYRA® to the U.S. market is the result of a collaborative effort. The Fondazione Telethon (FT), a leading Italian biomedical charity dedicated to advancing research on rare genetic diseases, has partnered with Orphan Therapies, the commercial arm of Orphan Therapeutics Accelerator. This partnership aims to ensure sustainable access to innovative treatments for ultra-rare diseases.

Beth White, the Business Director of Orphan Therapies, emphasizes their commitment to finding alternative ways to deliver essential treatments for rare ailments. She notes that their collaboration with FT on WASKYRA® is a promising initial example of this approach.

WASKYRA® Approval and Availability

WASKYRA® received approval from the FDA in December 2025, becoming the first gene therapy to be commercially launched under a non-profit collaboration. This development is a significant breakthrough, especially for families affected by WAS, as it opens up a pathway to previously unavailable treatment options.

With UCSF Benioff Children’s Hospitals at the forefront of this initiative, the qualified treatment center's designation is the first step toward establishing a comprehensive network of specialized clinics across the U.S. These centers will enable patients to access WASKYRA® treatment, thus overcoming barriers linked to rare disease therapies.

UCSF: A Leader in Pediatric Care

UCSF Benioff Children’s Hospitals have built a reputation as one of the premier pediatric healthcare institutions in the U.S., particularly in managing complex genetic and immunological disorders. This recent designation reinforces UCSF’s commitment to providing cutting-edge treatment solutions for the most vulnerable patients, ensuring they receive necessary medical care.

Ilaria Villa, General Director of Fondazione Telethon, reflects on the transformative journey from scientific research to therapeutic reality, stating, “This important milestone and our partnership with Orphan Therapies illustrate the ongoing evolution of our model to turn scientific findings into concrete therapeutic opportunities for patients.”

As additional centers are anticipated to join the initiative in the upcoming months, the hope is that more patients with Wiskott-Aldrich Syndrome will have access to this life-changing therapy. The introduction of WASKYRA® represents a beacon of promise for families dealing with the challenges of rare genetic disorders, emphasizing the necessity of innovative collaborative approaches in healthcare.