LYS-GM101 – VJRegenMed https://mirror.vjregenmed.com The Video Journal of Regenerative Medicine Tue, 04 Jan 2022 17:55:02 +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 LYS-GM101 – VJRegenMed https://mirror.vjregenmed.com 32 32 LYS-GM101 AAV gene therapy for GM1 gangliosidosis https://mirror.vjregenmed.com/video/z3jg5fdbgrc-lys-gm101-aav-gene-therapy-for-gm1-gangliosidosis/ Thu, 23 Dec 2021 16:24:52 +0000 http://13.40.107.223/video/z3jg5fdbgrc-lys-gm101-aav-gene-therapy-for-gm1-gangliosidosis/ GM1 gangliosidosis is a rare autosomal lysosomal storage disorder caused by mutations in the GLB1 gene which encodes β-galactosidase (Beta-gal), resulting in toxic accumulation of GM1 ganglioside predominantly in the central nervous system (CNS). Michael Hocquemiller, PhD, Lysogene, Paris, France, describes ongoing research into LYS-GM101, an adeno-associated virus serotype rh.10 (AAVrh.10) vector carrying GLB1 cDNA, administered via a single injection into the cisterna magna. Following promising results obtained in pre-clinical studies in mouse, cat and non-human primate disease models, an open-label two-stage adaptive design study (NCT04273269) is currently underway which will evaluate the safety and efficacy of LYS-GM101 in patients with GM1 gangliosidosis. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.

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