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M 043

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Drug Profile

M 043

Alternative Names: AAV-NAGLU-S1S3; Lenti-NAGLU-S1S3; M-043; Sanfilippo B (MPSIIIB)

Latest Information Update: 09 Feb 2023

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At a glance

  • Originator M6P Therapeutics
  • Class Gene therapies
  • Mechanism of Action Alpha N acetyl D glucosaminidase 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

  • Preclinical Mucopolysaccharidosis III

Most Recent Events

  • 09 Feb 2023 M 043 receives Rare pediatric drug for Enzyme replacement therapy status for in USA prior to February 2023 (M6P Therapeutics website, February 2023)
  • 04 Feb 2021 M 043 is available for licensing as of 03 Feb 2021
  • 03 Feb 2021 Preclinical trials in Mucopolysaccharidosis III in USA (Parenteral) (M6P Therapeutics pipeline, February 2021)

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