#USA #San Francisco #Fondazione Telethon #Waskyra #UCSF Benioff

UCSF Benioff Children's Hospitals Achieve a Milestone in Gene Therapy

In a groundbreaking development within the realm of pediatric treatment, UCSF Benioff Children's Hospitals has been designated as the first qualified treatment center in the United States authorized to administer the innovative gene therapy, WASKYRA®. This therapy aims to treat children suffering from Wiskott-Aldrich syndrome (WAS), a rare genetic disorder that poses serious health risks.

WASKYRA® is the first gene therapy approved in the U.S. for the treatment of WAS. The therapy is being offered through a collaboration with the Fondazione Telethon, a distinguished Italian biomedical charity known for advancing research into rare genetic diseases for over 35 years, and its commercial partner, Orphan Therapies.

What is Wiskott-Aldrich Syndrome?

Wiskott-Aldrich syndrome is a serious immunodeficiency disorder caused by mutations in the WAS gene. Patients with WAS typically experience symptoms including low platelet counts (thrombocytopenia), increased risk of bleeding, recurrent infections, eczema, and a higher propensity for autoimmune diseases and malignancies. The life-threatening nature of the syndrome necessitates effective treatments, making the approval of WASKYRA® a pivotal moment for affected families.

Groundbreaking Approval and Treatment Availability

In December 2025, the U.S. Food and Drug Administration (FDA) approved WASKYRA® to meet the urgent needs of patients with WAS. This innovative treatment has been developed with patient accessibility at its core. UCSF Benioff is now equipped to provide this therapy to eligible pediatric patients as young as six months old, as well as adults who exhibit the specific gene mutation and do not have a suitable HLA-matched donor for hematopoietic stem cell transplantation (HSCT).

This initiative not only provides hope to WAS patients but also sets a precedent for future collaborations aimed at enhancing access to vital therapies for extremely rare diseases. The partnership signifies a monumental step towards ensuring patients with WAS receive potentially life-saving treatment within medical infrastructure that can handle their specialized needs.

Expanding Access to Innovative Therapies

The collaboration between UCSF Benioff, Fondazione Telethon, and Orphan Therapies reflects a shift in how gene therapies are developed and distributed. “The approval of UCSF Health as a Qualified Treatment Center (QTC) is a groundbreaking step,” remarked Ilaria Villa, CEO of Fondazione Telethon. This strategic alliance demonstrates a commitment to addressing the market challenges that have previously impeded access to critical treatments for those suffering from rare genetic disorders.

Beth White, Chief Commercial Officer of Orphan Therapies, accentuated the importance of establishing a sustainable commercial access route for therapies catering to very rare conditions. “Finding new ways to navigate existing frameworks is essential. The partnership regarding WASKYRA® exemplifies our innovative approach,” she stated.

The Road Ahead

As UCSF Benioff Children's Hospitals leads the way as the first treatment center for WASKYRA®, the healthcare community anticipates that more centers will join this groundbreaking initiative in the upcoming months, forming a robust network of specialized clinical centers across the U.S. This nationwide effort will be critical in ensuring timely access to cutting-edge therapies for other patients grappling with rare and life-threatening conditions.

In summary, the launch of WASKYRA® at UCSF Benioff not only marks a significant advancement in gene therapy but also illustrates an evolving model for collaboration between medical, commercial, and philanthropic entities. Together, these partners aim to transform research breakthroughs into tangible therapeutic options for patients who need them most.