San Francisco-based UCSF Benioff Children’s Hospitals has become the first U.S. qualified treatment center contracted to administer Waskyra (etuvetidigene autotemcel) to eligible patients with Wiskott-Aldrich syndrome.
The designation makes Waskyra commercially available to eligible patients in the U.S., according to a Sept. 28 news release. More treatment centers are expected to be added in the coming months.
The FDA approved Waskyra in December 2025 as the first gene therapy to treat Wiskott-Aldrich syndrome. It is approved for patients 6 months and older who have a mutation in the WAS gene, for whom hematopoietic stem cell transplantation is appropriate and who do not have a suitable human leukocyte antigen-matched related stem cell donor.
