A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

NCTID	NCT04273269 (View at clinicaltrials.gov)
Description	LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system. This study will assess, in a 2-stage adaptive-design, the safety and efficacy of treatment in subjects with infantile GM1 gangliosidosis. (Show More)
Development Status	Inactive
Indication	GM1 Gangliosidosis
Disease Ontology Term	DOID:3322
Compound Name	LYS-GM101
Sponsor	LYSOGENE
Funder Type	Industry
Recruitment Status	Terminated
Enrollment Count	5
Results Posted	Not Available

Target Gene/Variant	GLB1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisternal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVrh.10
Editor Type	none
Dose 1	8E12 vg/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	3
Locations	United States,United Kingdom,France

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	2 participants died due to progression of disease, little evidence of potential efficacy