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NGN 401

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Drug Profile

NGN 401

Alternative Names: NGN-401

Latest Information Update: 20 Jun 2024

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At a glance

  • Originator Neurogene Inc
  • Class Gene therapies
  • Mechanism of Action Gene transference; Methyl-CpG-binding protein 2 gene replacements
  • Orphan Drug Status

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

    Yes - Rett syndrome
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Rett syndrome

Most Recent Events

  • 18 Jun 2024 Adverse events data from a phase I/II trial in Rett syndrome released by Neurogene
  • 03 Jun 2024 The US FDA selects NGN 401 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) pilot program
  • 10 May 2024 Australian Therapeutic Goods Administration approves IND application for NGN 401 in Rett syndrome
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