Around the World: Global Seminar Series Australasia - Thursday 26 February 2026
Hosted as part of the AROUND THE WORLD SERIES in collaboration with the Coalition of International Gene Therapy Societies.
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
