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M 002

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Drug Profile

M 002

Alternative Names: AAV-S1S3; M-002

Latest Information Update: 28 Feb 2025

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At a glance

  • Originator M6P Therapeutics
  • Class 3-ring heterocyclic compounds; Amides; Gene therapies; Small molecules
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Metabolic bone diseases
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • No development reported Metabolic bone diseases

Most Recent Events

  • 28 Feb 2025 No recent reports of development identified for preclinical development in Metabolic bone diseases in USA (Parenteral)
  • 09 Feb 2023 M6P Therapeutics plans to file IND in Metabolic bone disorders by Q3 2024 (M6P Therapeutics website, February 2023)
  • 17 May 2021 Pharmacodynamics data from a preclinical studies in Mucolipidosis type II ([metabolic bone diseases) presented at the American Society of Gene & Cell Therapy (ASGCT-2021)

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