Endocrinology – VJRegenMed

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.

Renin producing human kidney organoids

admin_vjr_user — Wed, 19 Aug 2020 16:20:19 +0000

Anushka Shankar, MD, MSc, of Erasmus University Rotterdam, Rotterdam, the Netherlands, discusses the development of human kidney organoids from stem cells, that can restore the endocrine system, including renin production. Furthermore, Dr. Shankar outlines the next steps in research, including determining how physiological interactions may affect renin production. This interview was recorded via an online conference call with The Video Journal of Regenerative Medicine (VJRegenMed).

Next steps in kidney organoid development

admin_vjr_user — Wed, 19 Aug 2020 16:20:19 +0000

Anushka Shankar, MD, MSc, of Erasmus University Rotterdam, Rotterdam, the Netherlands, outlines the next steps in developing functional kidney organoids including vascularization, and furthermore highlights the importance of safety analysis before these novel therapies can be used in routine clinical practice. This interview was recorded via an online conference call with The Video Journal of Regenerative Medicine (VJRegenMed).