LYS-GM101 / Lysogene - LARVOL DELTA

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=5 | Terminated | Sponsor: LYSOGENE | N=16 ➔ 5 | Trial completion date: Jan 2030 ➔ May 2023 | Active, not recruiting ➔ Terminated | Trial primary completion date: Jan 2026 ➔ May 2023; The clinical trial is closed due to Lysogene's cessation of activities. This study closure is not due to safety reasons.

Enrollment change • Gene therapy • Trial completion date • Trial primary completion date • Trial termination • Viral vector • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates. (PubMed, Mol Ther Methods Clin Dev) - "Histopathologic examination revealed asymptomatic adverse changes in the sensory pathways of the spinal cord and dorsal root ganglia in both sexes and at both doses. Taken as a whole, these pre-clinical data support the initiation of a clinical study with LYS-GM101 for the treatment of GM1 gangliosidosis."

Journal • Preclinical • CNS Disorders • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • GLB1

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2 | N=16 | Active, not recruiting | Sponsor: LYSOGENE | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2025 ➔ Jan 2030 | Trial primary completion date: Jun 2025 ➔ Jan 2026

Enrollment closed • Trial completion date • Trial primary completion date • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases

A Study of Intracisternal Administration of LYS-GM101 in Children with Infantile GM1 Gangliosidosis: Preliminary Results of the Safety Cohort (ASGCT 2022) - P1/2 | "The safety cohort data will be reviewed by an independent Data Safety Monitoring Board before initiating the confirmatory phase (Stage 2) with the objective to enroll at least 12 additional patients with early or late infantile GM1 gangliosidosis. Preliminary safety and biomarker data from the first enrolled subjects will be presented during this conference."

Clinical • CNS Disorders • Gene Therapies • Genetic Disorders • Lysosomal Storage Diseases • GLB1

[VIRTUAL] Intracisternal delivery of an AAV gene therapy candidate for the treatment of GM1 Gangliosidosis (ESGCT 2021) - P1/2 | "LYSGM101 is an AAVrh.10 based gene therapy vector carrying the GLB1 cDNA, administered via a onetime injection into the cisterna magna...The trial will enroll 16 patients with a diagnosis of early or late infantile GM1 gangliosidosis at sites in the US and Europe. This talk will focus on nonclinical pharmacology and toxicology studies and elaborate on the current status of the ongoing clinical trial."

CNS Disorders • Gene Therapies • Lysosomal Storage Diseases

"Lysogene Announces a Presentation of its LYS-GM101 Program at the ESGCT 2021 https://t.co/UqdRiwafMs" (@NewsFromBW)

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2; N=16; Recruiting; Sponsor: LYSOGENE; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Gene Therapies • MRI

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis (clinicaltrials.gov) - P1/2; N=16; Not yet recruiting; Sponsor: LYSOGENE; Initiation date: Apr 2020 ➔ Mar 2021

Clinical • Trial initiation date • Gene Therapies