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ASAH1 gene therapy - Genethon

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Drug Profile

ASAH1 gene therapy - Genethon

Latest Information Update: 04 Dec 2024

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At a glance

  • Originator Genethon
  • Class Gene therapies
  • Mechanism of Action Ceramidase 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 Farber lipogranulomatosis; Spinal muscular atrophy

Most Recent Events

  • 21 Oct 2024 Preclinical trials in Farber lipogranulomatosis in France (Parenteral), before October 2024 (Genethon pipeline, October 2024)
  • 21 Oct 2024 Preclinical trials in Spinal muscular atrophy in France (Parenteral), before October 2024 (Genethon pipeline, October 2024)
  • 21 Oct 2024 Genethon plans a clinical trial for Farber lipogranulomatosis and Spinal muscular atrophy (Genethon website, October 2024)

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