FDA approval of the Biologics License Application (BLA) for Waskyra, the first gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).

The ESGCT would like to congratulate Fondazione Telethon and HSR-TIGET on the milestone achievement of the FDA approval of WAS ex vivo gene therapy.

Please see below their statement:

We are honoured to share a moment of profound significance for us, for patients, families, and the scientific community. 

The U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, the first gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-wiskott-aldrich-syndrome

This milestone marks the culmination of decades of research, perseverance, and collaboration driven by Telethon's mission: to transform the lives of those affected by rare diseases through the power of science and compassion.

Our heartfelt gratitude goes to the researchers, clinicians, partners, and — above all — the patients and families whose trust and courage made this achievement possible.