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

Lysogene Announces First Patient in the United States Dosed with LYS-GM101 Investigational Gene Therapy for the Treatment of GM1 Gangliosidosis
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Lysogene Announces FDA Fast Track Designation for LYS-GM101 Gene Therapy for the Treatment of GM1 Gangliosidosis

Lysogene Announces FDA Fast Track Designation for LYS-GM101 Gene Therapy for the Treatment of GM1 Gangliosidosis
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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 ....

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