https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Thu, 26 Aug 2021 17:53:56 +0000 en-US hourly 1 https://wordpress.org/?v=6.5.2 https://d2xz56kaqxj8if.cloudfront.net/wp-content/uploads/2023/09/12102509/VJR-Favicon.png https://mirror.vjregenmed.com 32 32 https://mirror.vjregenmed.com/video/wvhyubjwzb4-avr-rd-01-ex-vivo-lentiviral-gene-therapy-for-fabry-disease/ Fri, 14 May 2021 14:11:25 +0000 http://13.40.107.223/video/wvhyubjwzb4-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.

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