Michael Hocquemiller – VJRegenMed

LYS-GM101 AAV gene therapy for GM1 gangliosidosis

admin_vjr_user — Thu, 23 Dec 2021 16:24:52 +0000

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.

LYS-SAF302 AAV gene therapy for MPS IIIA

admin_vjr_user — Wed, 22 Dec 2021 18:29:28 +0000

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.

ESGCT 2021 highlights: AAV vector capsid modification for CNS disorders

admin_vjr_user — Wed, 22 Dec 2021 18:29:27 +0000

Michael Hocquemiller, PhD, Lysogene, Paris, France, discuses his highlights from the 2021 European Society of Gene & Cell Therapy (ESGCT) Congress. He notes that ongoing research into adeno-associated virus (AAV) vector capsid modification is of particular importance in gene therapy for central nervous system (CNS) disorders. The ability to enhance the tropism of AAV vectors could allow more efficient targeting of the CNS following intravenous injection, thus allowing the administration of lower vector doses which would reduce challenges associated with toxicity and vector manufacturing. This interview took place at the ESGCT Virtual Congress 2021.