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
2025-SEVILLE
The 32nd ESGCT Annual Congress took place in collaboration with SETGYC from 7-10 October 2025 at Fibes in Seville. Congress delegates can log in to their online registration portal to access the Congress abstract book, online programme, and Certificate of Attendance. Login here > Abstracts published by Human Gene Therapy will be available soon. Event Statistics (Click to enlarge) We are very grateful for the support of all our sponsors and partners.
