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Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis


Lysogene Announces First Patient Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
Ongoing enrollment of a total of 16 patients in the UK, US and France
Regulatory News:
Lysogene (Paris:LYS) (FR0013233475 LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today announced dosing of the first patient, at Royal Manchester Children s Hospital, part of Manchester University NHS Foundation Trust, in the global adaptative-design clinical trial with LYS-GM101 (NCT04273269), a gene therapy for the treatment of GM1 gangliosidosis.
This trial is an interventional, multi-center, single-arm, two-stage adaptive-design study evaluating the intracisternal delivery of a recombinant adeno-associated virus vector serotype rh.10 (AAVrh.10) carrying the human ß-galactosidase gene (GBL1). The clinical trial includes a safety phase and a confirmatory efficacy phase. The trial will enroll 16 patients wi ....

United Kingdom , United States , Simon Jones , Karen Aiach , Manchester University , European Union , University Of Manchester , University Dental Hospital Of Manchester , Sarepta Therapeutics Inc , Foundation Trust , Second Lysogene , Royal Manchester Children , Founder Chairman , Chief Executive Officer , Chief Investigator , Paediatric Inherited Metabolic Disease , Senior Lecturer , Rare Pediatric Disease , Casimir Trials , Sarepta Therapeutics , Manchester Royal Infirmary , Saint Mary , Manchester Royal Eye Hospital , University Dental Hospital , Trafford General , Altrincham Hospital ,