UCSF Benioff Children's Hospitals Designated First U.S. Center for WASKYRA Gene Therapy
In a remarkable advancement for pediatric medicine, UCSF Benioff Children's Hospitals has been recognized as the first Qualified Treatment Center (QTC) in the United States to administer WASKYRA® (etuvetidigene autotemcel), a pioneering gene therapy designed specifically for Wiskott-Aldrich Syndrome (WAS). This significant milestone was announced jointly by Fondazione Telethon (FT), an Italian biomedical charity, and Orphan Therapies (OT), a non-profit biotech firm dedicated to enhancing access to treatments for ultra-rare conditions.
WASKYRA® is a breakthrough therapy that has been developed to address Wiskott-Aldrich Syndrome, a rare immunodeficiency disorder resulting from mutations in the WAS gene. Patients suffering from this syndrome face a host of challenges, including thrombocytopenia, recurrent infections, eczema, and a heightened risk of autoimmune diseases. Traditionally, hematopoietic stem cell transplantation (HSCT) has been the primary treatment option for these patients, but this procedure often necessitates a suitable human leukocyte antigen (HLA)-matched related stem cell donor, which can be difficult to obtain. With the launch of WASKYRA®, patients aged six months and older now have a commercially available treatment alternative when such donors are not available.
The approval of WASKYRA® by the U.S. Food and Drug Administration (FDA) in December 2025 marked the first instance of a gene therapy being expressly designed to treat Wiskott-Aldrich Syndrome in the U.S. This therapy's distinction lies not only in its innovative approach but also in its journey to commercialization, with WASKYRA® being produced and marketed entirely through a collaborative, non-profit effort. The partnership between Fondazione Telethon and Orphan Therapies underscores a novel business model aimed at overcoming market barriers that often prevent urgent therapies from reaching those in need.
The designation of UCSF Benioff Children's Hospitals as a QTC is a critical step forward, paving the way for the establishment of a clinical network across the U.S. targeting specialized centers capable of offering WASKYRA® and potentially other emerging therapies. The experts at UCSF Health have specialized training in managing complex genetic and immunologic disorders, making them an ideal provider for this advanced treatment.
Ilaria Villa, CEO of Fondazione Telethon, stated, "This milestone reflects the ongoing evolution of our model, designed to translate scientific research into tangible therapeutic opportunities. After decades of significant research, we recognized the necessity for collaborative solutions to challenges that impede patient access to rare disease treatments."
Beth White, Chief Commercial Officer of Orphan Therapies, emphasized the commitment to establishing sustainable commercial avenues for these vital treatments. "Our approach aims to deliver effective therapies despite existing systemic challenges in healthcare accessibility. The collaboration with Fondazione Telethon on WASKYRA® serves as a model we intend to replicate across therapies for ultra-rare diseases."
The conclusion of this initiative is set against the backdrop of a broader commitment to innovation in pediatric healthcare. As future centers are anticipated to follow in UCSF's footsteps, the landscape for managing conditions like Wiskott-Aldrich Syndrome is set for substantial transformation. The integration of WASKYRA® not only highlights the potential within the realm of gene therapy but embodies a beacon of hope for countless families navigating the complexities of rare genetic disorders.
Looking ahead, this partnership signals a progressive shift towards more integrated and accessible care pathways for patients facing ultra-rare conditions, shifting the paradigm from traditional therapies to innovative treatments like gene therapy that can potentially improve the quality of life for many.
WASKYRA® is a breakthrough therapy that has been developed to address Wiskott-Aldrich Syndrome, a rare immunodeficiency disorder resulting from mutations in the WAS gene. Patients suffering from this syndrome face a host of challenges, including thrombocytopenia, recurrent infections, eczema, and a heightened risk of autoimmune diseases. Traditionally, hematopoietic stem cell transplantation (HSCT) has been the primary treatment option for these patients, but this procedure often necessitates a suitable human leukocyte antigen (HLA)-matched related stem cell donor, which can be difficult to obtain. With the launch of WASKYRA®, patients aged six months and older now have a commercially available treatment alternative when such donors are not available.
The approval of WASKYRA® by the U.S. Food and Drug Administration (FDA) in December 2025 marked the first instance of a gene therapy being expressly designed to treat Wiskott-Aldrich Syndrome in the U.S. This therapy's distinction lies not only in its innovative approach but also in its journey to commercialization, with WASKYRA® being produced and marketed entirely through a collaborative, non-profit effort. The partnership between Fondazione Telethon and Orphan Therapies underscores a novel business model aimed at overcoming market barriers that often prevent urgent therapies from reaching those in need.
The designation of UCSF Benioff Children's Hospitals as a QTC is a critical step forward, paving the way for the establishment of a clinical network across the U.S. targeting specialized centers capable of offering WASKYRA® and potentially other emerging therapies. The experts at UCSF Health have specialized training in managing complex genetic and immunologic disorders, making them an ideal provider for this advanced treatment.
Ilaria Villa, CEO of Fondazione Telethon, stated, "This milestone reflects the ongoing evolution of our model, designed to translate scientific research into tangible therapeutic opportunities. After decades of significant research, we recognized the necessity for collaborative solutions to challenges that impede patient access to rare disease treatments."
Beth White, Chief Commercial Officer of Orphan Therapies, emphasized the commitment to establishing sustainable commercial avenues for these vital treatments. "Our approach aims to deliver effective therapies despite existing systemic challenges in healthcare accessibility. The collaboration with Fondazione Telethon on WASKYRA® serves as a model we intend to replicate across therapies for ultra-rare diseases."
The conclusion of this initiative is set against the backdrop of a broader commitment to innovation in pediatric healthcare. As future centers are anticipated to follow in UCSF's footsteps, the landscape for managing conditions like Wiskott-Aldrich Syndrome is set for substantial transformation. The integration of WASKYRA® not only highlights the potential within the realm of gene therapy but embodies a beacon of hope for countless families navigating the complexities of rare genetic disorders.
Looking ahead, this partnership signals a progressive shift towards more integrated and accessible care pathways for patients facing ultra-rare conditions, shifting the paradigm from traditional therapies to innovative treatments like gene therapy that can potentially improve the quality of life for many.
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