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ASGCT 2021 | AVR-RD-01: ex vivo lentiviral gene therapy for Fabry disease

Mark Thomas, MD, Royal Perth Hospital, Perth, Australia, provides an overview of the current treatment landscape for Fabry disease and the rationale behind the application of gene therapy technologies and their potential to offer a one-time curative therapy for Fabry disease. He describes updated results from the ongoing Phase I and II FAB-GT clinical trials (NCT03454893) of AVR-RD-01, the first investigational ex vivo lentiviral gene therapy for Fabry disease. The results presented at ASGCT 2021 indicate sustained enzyme activity and a 100% globotriaosylceramide (Gb3) kidney clearance at one-year in the most recent Phase II study patient. This interview took place during the American Society for Cell & Gene Therapy 24th Annual Meeting 2021.


Mark Thomas is a principal investigator with AVROBIO, and has also received departmental research support or consultancy fees from Sanofi-Genzyme, Takeda-Shire, Amicus & Idorsia, but hold no stocks or equity.