UCSF Benioff Children's Hospitals Named First US Qualified Treatment Center for WASKYRA Gene Therapy in Wiskott-Aldrich Syndrome
核心洞察
UCSF Benioff Children's Hospitals (搜索) has been designated the first US Qualified Treatment Center contracted to administer WASKYRA (搜索) (etuvetidigene autotemcel (搜索)) to eligible patients with Wiskott-Aldrich Syndrome (搜索).
WASKYRA (搜索), approved by the FDA in December 2025, is the first gene therapy available in the US for Wiskott-Aldrich Syndrome (搜索) and the first advanced therapy commercialized entirely through a non-profit collaboration.
The autologous hematopoietic stem cell-based therapy is indicated for patients aged six months and older with a WAS gene (搜索) mutation and no suitable HLA-matched related stem cell donor.
Fondazione Telethon and Orphan Therapies (搜索) announced the designation of UCSF Benioff Children's Hospitals (搜索) as the first US Qualified Treatment Center (QTC) contracted to administer WASKYRA (搜索) (etuvetidigene autotemcel (搜索)), the first gene therapy approved in the United States for Wiskott-Aldrich Syndrome (搜索) (WAS). The designation establishes commercial availability of the therapy for eligible patients in the US and marks the first step toward building a national network of specialized clinical centers.
WASKYRA (搜索) received FDA approval in December 2025. It is the first gene therapy available to treat Wiskott-Aldrich Syndrome (搜索) and the first advanced therapy to be commercialized entirely through a non-profit collaboration. Fondazione Telethon, an Italian biomedical charity with more than 35 years of work in rare and complex genetic diseases, developed the therapy and submitted it for regulatory approval. Orphan Therapies (搜索), the commercial subsidiary of the non-profit Orphan Therapeutics Accelerator (搜索), is the exclusive US commercialization partner.
Indication and Patient Population
WASKYRA (搜索) is approved for pediatric patients aged six months and older and for adults with Wiskott-Aldrich Syndrome (搜索) who have a mutation in the WAS gene (搜索) and for whom hematopoietic stem cell transplantation (HSCT) is appropriate, but for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available.
Wiskott-Aldrich Syndrome (搜索) is an ultra-rare, life-threatening immunodeficiency caused by mutations in the WAS gene (搜索). The disease is characterized by thrombocytopenia and bleeding complications, recurrent infections, eczema, immune dysregulation, and an increased risk of autoimmune disease and malignancy.
The therapy uses a patient's own stem cells, genetically modified ex vivo to introduce a functional copy of the WAS gene (搜索), with the goal of restoring expression of WAS protein and addressing the underlying cause of disease.
Building a US Treatment Network
UCSF Benioff Children's Hospitals (搜索) are among the nation's foremost pediatric hospitals and have extensive expertise in caring for patients with rare genetic and immunologic disorders. The activation of UCSF Health as a QTC represents the first step toward a US clinical network of highly specialized centers intended to support patient access, with additional centers expected to be added in the coming months.
"This milestone and partnership with Orphan Therapies (搜索) reflect the ongoing and successful evolution of Fondazione Telethon's model, which was initially designed to translate scientific research into tangible therapeutic opportunities for patients," said Ilaria Villa, CEO of Fondazione Telethon. "After decades of groundbreaking research, we recognized the need for novel, collaborative solutions to market-based challenges that keep urgently needed treatments from reaching people affected by rare genetic diseases."
Beth White, Chief Commercial Officer of Orphan Therapies (搜索), framed the arrangement as a template for the organization's broader strategy. "Our purpose is to provide reliable and sustainable commercial access to vital treatments for very rare conditions," White said. "To do so, we find new ways to work within existing systems to deliver for patients. Our partnership with FT on WASKYRA (搜索) is an initial proof point for an approach that we intend to apply across a range of therapies for ultra-rare conditions."
Additional information about WASKYRA (搜索), including safety information, full prescribing information, and updates on the US treatment network, will be available on the dedicated website Waskyra.com.