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M 002

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Drug Profile

M 002

Alternative Names: AAV-S1S3; M-002

Latest Information Update: 18 Jul 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

  • Preclinical Metabolic bone diseases

Most Recent Events

  • 18 Jul 2025 M 002 is still in preclinical development in Metabolic bone diseases in USA (Parenteral) (M6P therapeutics pipeline, July 2025)
  • 18 Jul 2025 M6P Therapeutics plans to file IND in Metabolic bone disorders by third quarter of 2026 (M6P Therapeutics pipeline, July 2025)
  • 18 Jul 2025 Pharmacodynamics data from a preclinical studies in Mucolipidosis type II released by M6P Therapeutics

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