UCSF Benioff Named First WASKYRA Treatment Center

The hospital is the first site in the U.S. authorized to administer the gene therapy for Wiskott-Aldrich Syndrome.

Updated on Sept. 28, 2026 in Biotech

UCSF Benioff Named First WASKYRA Treatment Center

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UCSF Benioff Children's Hospitals has been designated as the first Qualified Treatment Center for WASKYRA, a recently approved gene therapy. The drug provides a treatment option for pediatric patients with Wiskott-Aldrich Syndrome who lack a matched donor.

Why it matters

This designation provides a critical path for patients with rare genetic conditions to access specialized gene therapies. The initiative helps overcome market barriers that have historically limited the availability of life-saving treatments for rare diseases.

WASKYRA is a gene therapy designed for patients aged six months and older who have a mutation in the WAS gene. It is intended for individuals where a human leukocyte antigen-matched related donor is currently unavailable.

The players

UCSF Benioff Children's Hospitals

These hospitals provide comprehensive pediatric care and are a leading academic medical institution in San Francisco.

Orphan Therapies

This company serves as the exclusive United States commercialization partner for the WASKYRA gene therapy.

Fondazione Telethon

Based in Rome, Italy, this organization has focused on advancing medical research for genetic diseases for 35 years.

The details

The treatment is now commercially available in the United States through a network of specialized facilities. Fondazione Telethon developed the therapy and successfully submitted it for FDA approval in December 2025.

The FDA approved the gene therapy WASKYRA in December 2025.
UCSF Benioff Children's Hospitals became the first QTC on September 28, 2026.
Additional treatment centers will be added to the network in the coming months.

The Tech Race

The introduction of WASKYRA follows the framework established by the FDA Orphan Drug Act to incentivize research into rare medical conditions. This marks a shift toward localized, specialized administration models that replace broader, less targeted clinical approaches.

Eligible patients aged six months and older gain local access to a specialized therapy previously unavailable in the region. Families will need to coordinate with clinical specialists to determine if their child meets the specific HLA-matched donor criteria.

The takeaway

This development highlights the growing importance of specialized clinical networks in the delivery of modern genetic medicine. Patients and families should consult with their healthcare providers to monitor the expansion of treatment sites.