UCSF becomes first US center to give nonprofit gene therapy Waskyra

Fondazione Telethon and its commercial partner Orphan Therapies announced on 28 September 2026 that UCSF Benioff Children’s Hospitals is the first qualified treatment center in the United States contracted to administer Waskyra (etuvetidigene autotemcel).

Waskyra treats Wiskott-Aldrich syndrome, an ultra-rare inherited immunodeficiency caused by mutations in the WAS gene. The therapy takes a patient’s own blood stem cells, adds a working copy of the gene outside the body, and returns the corrected cells by transplant. The US Food and Drug Administration approved it in December 2025 for patients aged six months and older who lack a suitable HLA-matched related stem cell donor.

The partners describe Waskyra as the first advanced therapy commercialized entirely through a nonprofit collaboration; Fondazione Telethon is an Italian medical research charity. The announcement does not state a price.

The designation turns an approval into an available treatment: until a contracted center exists, a gene therapy cleared by the regulator cannot reach a US patient.