#USA #San Francisco #Fondazione Telethon #WASKYRA® #UCSF Hospitals

UCSF Benioff Children's Hospitals Make History with WASKYRA®

In a groundbreaking development for pediatric medicine, UCSF Benioff Children's Hospitals have been designated as the first Qualified Treatment Center (QTC) in the United States to administer WASKYRA® (etuvetidigene autotemcel), the first gene therapy officially approved for the treatment of Wiskott-Aldrich syndrome (WAS). This historical achievement was announced in collaboration with Fondazione Telethon, an esteemed Italian biomedical charity, and Orphan Therapies, a nonprofit dedicated to advancing therapies for rare diseases.

What is WASKYRA®?

WASKYRA® is a state-of-the-art gene therapy designed specifically for patients suffering from Wiskott-Aldrich syndrome, a rare and potentially deadly immunodeficiency disorder. The syndrome is caused by mutations in the WAS gene and presents a range of serious health issues, including thrombocytopenia, recurrent infections, eczema, and increased susceptibility to autoimmune diseases and cancers.

Approved by the FDA in December 2025, WASKYRA® offers a new hope for pediatric patients who do not have a compatible human leukocyte antigen (HLA)-matched stem cell donor. This gene therapy aims to correct the underlying genetic defect, potentially transforming the lives of those affected by this serious condition.

The Role of UCSF Benioff Children's Hospitals

UCSF Benioff Children's Hospitals are recognized as one of the leading pediatric healthcare providers in the country, particularly in the field of genetic and immunologic disorders. Their designation as a Qualified Treatment Center allows them to provide access to WASKYRA® for eligible patients across the United States. In doing so, they are not only paving the way for innovative treatments but also working towards establishing a national network of specialized centers focused on rare diseases.

Ilaria Villa, the CEO of Fondazione Telethon, expressed her enthusiasm for this partnership, stating, "This milestone and our collaboration with Orphan Therapies reflect the continuous evolution of the Fondazione Telethon model, initially designed to turn groundbreaking scientific research into accessible therapeutic opportunities for patients. Decades of pioneering research highlighted the necessity for collaborative solutions to overcome market challenges that prevent urgently needed treatments from reaching individuals affected by rare genetic diseases."

Beth White, the Business Director of Orphan Therapies, underscored the organization's mission to ensure reliable and sustainable access to vital therapies for extremely rare diseases. She noted, "Our collaboration with FT on WASKYRA® is a foundational test of our approach, which we strive to extend to a variety of therapies for ultrarare conditions. We are committed to finding innovative ways to operate within existing systems to benefit affected patients."

With WASKYRA® now commercially available and the induction of UCSF Benioff Children's Hospitals as a QTC, the future looks promising for patients diagnosed with Wiskott-Aldrich syndrome. As the first step toward a national integration of specialized clinical centers, this initiative could redefine treatment accessibility and outcomes for those afflicted by rare genetic conditions.

The administration of WASKYRA® marks a significant advancement in gene therapy that combines the latest scientific research with compassionate patient care. With these hospitals taking the lead in delivering such transformative treatments, the hope is to see a brighter future for many children and families grappling with the implications of rare genetic disorders.