Up to 13 Years of Follow-up: Integrated Analysis of Etuvetidigene Autotemcel Shows Sustained Clinical Benefit in WAS Patients
During the EBMT Annual Meeting, Dr. Francesca Ferrua from the IRCCS San Raffaele Scientific Institute presented an integrated analysis of the lentiviral hematopoietic stem and progenitor cell (HSPC) gene therapy, etuvetidigene autotemcel (ototemcel), for the treatment of Wiskott-Aldrich Syndrome (WAS). The report highlighted remarkable efficacy and safety outcomes with a follow-up period extending up to 13 years.









