Fabry Disease – VJRegenMed

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

admin_vjr_user — Wed, 22 Dec 2021 18:29:34 +0000

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.

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

admin_vjr_user — Fri, 14 May 2021 14:11:25 +0000

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.