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GNE-Lipoplex gene therapy - Gradalis

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Drug Profile

GNE-Lipoplex gene therapy - Gradalis

Latest Information Update: 18 Sep 2023

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At a glance

  • Originator Gradalis
  • Class Gene therapies
  • 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.

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Nonaka distal myopathy

Most Recent Events

  • 09 Aug 2023 Gradalis plans to file IND application for GNE Lipoplex gene therapy
  • 31 Jul 2023 Preclinical trials in Nonaka distal myopathy in USA (Parenteral)
  • 31 Jul 2023 Gradalis plans a phase I trial for Nonaka distal myopathy in 2024

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