Last update 08 Nov 2024

LYS-GM101

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Lixmabegagene Relduparvovec
Target
β-galactosidase
Mechanism
β-galactosidase modulators(Beta-galactosidase modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication-
Inactive Indication
Gangliosidosis, GM1
Originator Organization
Lysogene SALysogene SA
Active Organization-
Inactive Organization
Lysogene SALysogene SA
Drug Highest PhaseDiscontinuedPhase 1/2
First Approval Date-
RegulationOrphan Drug (US), Rare Pediatric Disease (US), Fast Track (US)
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Related

1
Clinical Trials associated with LYS-GM101
NCT04273269 / TerminatedPhase 1/2
An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human β-Galactosidase cDNA for Treatment of GM1 Gangliosidosis
100 Clinical Results associated with LYS-GM101
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100 Translational Medicine associated with LYS-GM101
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100 Patents (Medical) associated with LYS-GM101
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100 Deals associated with LYS-GM101
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Gangliosidosis, GM1Phase 2
US
Lysogene SALysogene SA
11 May 2021
Gangliosidosis, GM1Phase 2
FR
Lysogene SALysogene SA
11 May 2021
Gangliosidosis, GM1Phase 2
GB
Lysogene SALysogene SA
11 May 2021
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Clinical Result

Indication
Phase
Evaluation
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Clinical Trial

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Approval

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Regulation

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