UCSF can now offer an approved gene therapy for Wiskott-Aldrich syndrome

UCSF Benioff Children's Hospitals is the first center in the United States qualified to give Waskyra, the gene therapy the Food and Drug Administration approved in December 2025 for Wiskott-Aldrich syndrome, the UCSF Department of Pediatrics reported on October 20, 2026. The treatment, also called etuvetidigene autotemcel, uses a patient's own blood-forming stem cells. Those cells are collected, given a working copy of the WAS gene, and returned after chemotherapy. No donor is required. The syndrome is a rare genetic disorder that weakens the immune system and the blood's ability to clot. Children can have severe infections, bleeding, and eczema.

In the studies that supported approval, 27 patients with severe disease received the therapy. Severe infections fell by 93 percent, and bleeding events fell by 60 percent, during the first year. Kristin Shimano, a UCSF professor of pediatrics, said that for many families the counts and the immune function move much closer to normal. The label is for patients 6 months and older, children and adults, who have a WAS mutation, for whom a stem-cell transplant is appropriate, and who do not have a suitable matched related donor. Chris Dvorak's team expects to collect cells from UCSF's first patient before the end of the year. Those year-one percentages come from the approval studies, not from anyone treated at UCSF yet. The department points readers to the prescribing information for safety details. A drop in infections and bleeding over one year, in 27 people, is not a statement about every patient or about decades of follow-up. Fondazione Telethon developed the therapy. Orphan Therapies is the U.S. partner. Other centers are expected to qualify after UCSF.