Educational content on VJRegenMed is intended for healthcare professionals only. By visiting this website and accessing this information you confirm that you are a healthcare professional.

Share this video  

ESGCT 2021 | The safety of AVR-RD-01 gene therapy for Fabry disease

Mark Thomas, MD, Royal Perth Hospital, Perth, Australia, provides an update on the ongoing combined Phase I and II FAB-GT clinical trials (NCT03454893) of AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease (FD). In these studies, two conditioning agents, low-dose melphalan or busulfan, were used. Interim safety data from five patients with FD receiving enzyme replacement therapy (ERT) the Phase I study and eight treatment-naïve patients in the Phase II study indicates a favorable safety profile for AVR-RD-01, with no adverse events, serious adverse events or allergy development related to the investigational product reported to date. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.