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M 012 - M6P Therapeutics

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Drug Profile

M 012 - M6P Therapeutics

Alternative Names: AAV-GBA-S1S3; M-012 - M6P Therapeutics

Latest Information Update: 09 Feb 2023

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At a glance

  • Originator M6P Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Glucosylceramidase replacements
  • Orphan Drug Status

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

    Yes - Gaucher's disease
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Preclinical Gaucher's disease

Most Recent Events

  • 09 Feb 2023 M6P Therapeutics plans to file IND for Gaucher's disease in Q1 2025 (M6P Therapeutics website, February 2023)
  • 11 Oct 2022 Pharmacodynamics data from a preclinical trial in Gaucher disease released by M6P Therapeutics
  • 19 Jul 2021 M6P Therapeutics plans a clinical trial for Gaucher's disease in 2022

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