UCSF Named First Qualified Center for WASKYRA Therapy
The FDA-approved gene therapy for Wiskott-Aldrich Syndrome is now commercially available in the United States.
Updated on Sept. 28, 2026 in Biotech
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UCSF Benioff Children's Hospitals has been designated the first Qualified Treatment Center for WASKYRA, a gene therapy approved in December 2025 to treat Wiskott-Aldrich Syndrome. The therapy is now commercially available for patients aged six months and older who lack a matched related donor.
Why it matters
This development marks the start of a phased U.S. rollout for the treatment, which addresses critical access barriers for patients with rare genetic mutations. The rollout aims to provide specialized care through a dedicated network of clinical centers.
WASKYRA is authorized for use in patients aged 6 months and older who possess a mutation in the WAS gene. This therapy is specifically indicated for those without an available human leukocyte antigen-matched related donor.
The players
UCSF Benioff Children's Hospitals
A premier pediatric healthcare network in San Francisco that specializes in advanced genetic and complex clinical treatments.
Orphan Therapies
A Cambridge-based biotechnology firm serving as the exclusive U.S. commercialization partner for rare disease therapies.
Fondazione Telethon
A Rome-based research foundation with 35 years of experience focused on advancing treatments for rare genetic diseases.
The details
WASKYRA acts as a gene therapy targeting Wiskott-Aldrich Syndrome, a rare immunodeficiency disorder caused by a mutation in the WAS gene that impairs the production of functional blood cells. The therapy is administered through specialized centers designed to handle the complexities of genetic interventions. Fondazione Telethon, an Italian biomedical research organization, developed the treatment over a 35-year research tenure before Orphan Therapies secured the exclusive U.S. commercialization rights.
Timeline
December 2025: The FDA officially approved the WASKYRA gene therapy.
September 28, 2026: UCSF Benioff Children's Hospitals became the first QTC.
Coming months: Additional treatment centers will be added to the U.S. network.
The Tech Race
This rollout represents the commercial transition of Fondazione Telethon's 35-year research program into the U.S. clinical landscape. It follows the FDA's regulatory pathway to address rare genetic conditions through specialized, decentralized treatment networks.
Patients aged six months and older meeting the genetic eligibility criteria can now access the treatment at the San Francisco-based center. Future access will depend on the geographic expansion of the specialized clinical network currently being established by Orphan Therapies.
The takeaway
The deployment of WASKYRA highlights the shift toward centralized administration for complex gene therapies in the United States. Families should watch for upcoming announcements regarding the rollout of further clinical treatment centers in other regions.
What happens next
Additional clinical sites are expected to be announced in the coming months as the commercial network expands.
Further reading
For more on local innovation in clinical genetics, visit the Biotech section.
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