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CLN1 gene therapy - Amicus Therapeutics

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Drug Profile

CLN1 gene therapy - Amicus Therapeutics

Alternative Names: CLN1 Batten Disease - Amicus Therapeutics

Latest Information Update: 28 Feb 2025

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At a glance

  • Originator Nationwide Children's Hospital; Ohio State University
  • Developer Amicus Therapeutics; Nationwide Children's Hospital
  • 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

  • No development reported Neuronal ceroid lipofuscinosis

Most Recent Events

  • 28 Feb 2025 No recent reports of development identified for preclinical development in Neuronal ceroid lipofuscinosis in USA (Intrathecal)
  • 13 Jan 2021 Preclinical trials in Neuronal ceroid lipofuscinosis in USA (Intrathecal)
  • 08 Feb 2019 Early research in Neuronal ceroid lipofuscinosis in USA (Parenteral) before February 2019 (Amicus Therapeutics Pipeline)

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