UCSF Named First US Center for Rare Gene Therapy
Patients aged six months and older can now access WASKYRA treatment for Wiskott-Aldrich Syndrome.
Updated on Sept. 28, 2026 in Biotech
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Families seeking treatment for Wiskott-Aldrich Syndrome now have a local option at UCSF Benioff Children's Hospitals. The facility has been named the first Qualified Treatment Center in the United States to offer the gene therapy WASKYRA.
Why it matters
This designation provides a new path for pediatric patients who lack a human leukocyte antigen-matched related donor. The partnership aims to resolve long-standing market challenges that have limited access to rare disease therapies.
UCSF Benioff Children's Hospitals is now the first center in the U.S. to offer this therapy, which is approved for children as young as six months. While the facility is active, the timing for the expansion of the broader treatment network remains unknown.
The players
UCSF Benioff Children's Hospitals
The local medical institution now serving as the first authorized site for this rare disease therapy.
Orphan Therapies
The Cambridge-based firm serving as the exclusive U.S. commercialization partner for the drug.
Fondazione Telethon
The Rome-based research organization that developed the therapy and submitted it for federal approval.
The details
The FDA approved WASKYRA in December 2025 to treat patients with mutations in the WAS gene. Developed by Fondazione Telethon and commercially managed by Orphan Therapies, the treatment is now available at UCSF for those without a matched donor. More treatment sites are expected to join the network in the coming months.
Timeline
December 2025: FDA granted approval for WASKYRA gene therapy.
September 28, 2026: UCSF Benioff Children's Hospitals was named the first Qualified Treatment Center.
Coming months: Additional clinical centers are slated to join the treatment network.
Across the Bay
This designation follows the precedent set by the FDA Orphan Drug Act framework by facilitating the commercial rollout of a specialized treatment for a rare condition. The move marks a shift in how medical research is scaled from development to clinical practice in the Bay Area.
Families with patients diagnosed with Wiskott-Aldrich Syndrome now have a local, authorized site for gene therapy administration. Eligibility is strictly limited to patients aged six months and older who do not have an antigen-matched donor.
The takeaway
Patients or families interested in this therapy should consult with their care team about eligibility requirements regarding the WAS gene mutation. Watch for future announcements as Orphan Therapies expands the network to additional clinical locations across the country.
Further reading
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