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NGN 201

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Drug Profile

NGN 201

Alternative Names: NGN-201

Latest Information Update: 16 Dec 2022

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At a glance

  • Originator Neurogene Inc
  • 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

Highest Development Phases

  • Preclinical Globoid cell leukodystrophy

Most Recent Events

  • 13 Dec 2022 Preclinical trials in Globoid cell leukodystrophy in USA (unspecified route) before December 2022 (Neurogene website, December 2022)

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