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Lixmabegagene relduparvovec - LYSOGENE

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Drug Profile

Lixmabegagene relduparvovec - LYSOGENE

Alternative Names: LYS-GM101

Latest Information Update: 22 Jun 2023

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At a glance

  • Originator LYSOGENE
  • Class Gene therapies
  • Mechanism of Action Beta-galactosidase replacements; Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Gangliosidoses
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Gangliosidoses

Most Recent Events

  • 07 Jun 2023 IDEAYA Biosciences terminates phase-I/II trial in Gangliosidoses (In children, In infants, In neonate) in France due to Lysogene's cessation of activities (Intracisternal) (NCT04273269)
  • 11 Feb 2022 Adverse events data from a phase I/II trial in infantile GM1 Gangliosidosis (In children, In infants, In neonates) released by LYSOGENE
  • 29 Dec 2021 Medicines and Healthcare products Regulatory Agency (MHRA) awards Lixmabegagene relduparvovec investigational gene therapy an ‘Innovation Passport’ for the treatment of GM1 gangliosidosis under the United Kingdom’s (UK) Innovative Licensing and Access Pathway (ILAP)
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