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M 033

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Drug Profile

M 033

Alternative Names: Lenti-GalC-S1S3; M-033

Latest Information Update: 18 Jul 2025

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At a glance

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

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

    No
  • New Molecular Entity No
  • Available For Licensing Yes

Highest Development Phases

  • Research Globoid cell leukodystrophy

Most Recent Events

  • 18 Jul 2025 M 033 is still in early research development in Globoid-cell-leukodystrophy in USA (Parenteral) (M6P Therapeutics pipeline, July 2025)
  • 28 Mar 2025 No recent reports of development identified for early research development in Globoid-cell-leukodystrophy in USA (Parenteral)
  • 09 Feb 2023 M 033 receives Rare pediatric drug for Enzyme replacement therapy status for in USA prior to February 2023 (M6P Therapeutics website, February 2023)

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