Cambridge, Mass.– Fondazione Telethon and Orphan Therapies have designated UCSF Benioff Children’s Hospitals as the first U.S. Qualified Treatment Center authorized to administer WASKYRA, a gene therapy for Wiskott-Aldrich Syndrome.
The designation makes WASKYRA commercially available to eligible patients in the United States, with additional treatment centers expected to be added in the coming months.
WASKYRA, or etuvetidigene autotemcel, is approved for pediatric patients aged six months and older and adults with Wiskott-Aldrich Syndrome who have a mutation in the WAS gene, are appropriate candidates for hematopoietic stem cell transplantation and do not have a suitable HLA-matched related stem cell donor.
Orphan Therapies is the exclusive U.S. commercialization partner for WASKYRA, which was developed by Fondazione Telethon and submitted to the U.S. Food and Drug Administration for approval. The therapy was approved by the FDA in December 2025 and is the first gene therapy approved in the United States for Wiskott-Aldrich Syndrome.
Wiskott-Aldrich Syndrome is an ultra-rare, life-threatening immunodeficiency caused by mutations in the WAS gene. The condition can cause thrombocytopenia and bleeding, recurrent infections, eczema, immune dysregulation and an increased risk of autoimmune disease and cancer.
UCSF Benioff Children’s Hospitals have experience treating patients with rare genetic and immunologic disorders. Their designation as a Qualified Treatment Center marks the first step in developing a U.S. network of specialized centers to administer the therapy.
“This milestone and partnership with Orphan Therapies reflect the ongoing and successful evolution of Fondazione Telethon’s model, which was initially designed to translate scientific research into tangible therapeutic opportunities for patients,” said Ilaria Villa, CEO of Fondazione Telethon.
“After decades of groundbreaking research, we recognized the need for novel, collaborative solutions to market-based challenges that keep urgently needed treatments from reaching people affected by rare genetic diseases,” Villa said.
“Our purpose is to provide reliable and sustainable commercial access to vital treatments for very rare conditions,” said Beth White, Chief Commercial Officer of Orphan Therapies. “Our partnership with FT on WASKYRA is an initial proof point for an approach that we intend to apply across a range of therapies for ultra-rare conditions.”
