https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Tue, 04 Jan 2022 17:55:04 +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/gzokksnhsq0-lys-saf302-aav-gene-therapy-for-mps-iiia/ Wed, 22 Dec 2021 18:29:28 +0000 http://13.40.107.223/video/gzokksnhsq0-lys-saf302-aav-gene-therapy-for-mps-iiia/ Michael Hocquemiller, PhD, Lysogene, Paris, France, provides an overview of the ongoing Phase II/III AAVance (NCT03612869) study of LYS-SAF302 in patients with Mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo type A. LYS-SAF302 is an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying functional human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA. This study aims to evaluate the efficacy in improving the neurodevelopmental state of MPS IIIA patients following one-time intracerebral administration of LYS-SAF302. Cerebrospinal fluid biomarker data obtained at one-year follow up supports the biological activity and therapeutic potential of LYS-SAF302. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

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