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CLN 301

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Drug Profile

CLN 301

Alternative Names: AAV-CLN3-Amicus Therapeutics; AAV9 CLN3; AT GTX 502; CLN-301; CLN3 gene therapy; CLN3 gene therapy- Amicus Therapeutics/Nationwide-Children's-Hospital/Ohio-State-University; CLN3-Batten disease-Amicus therapeutics

Latest Information Update: 05 May 2026

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At a glance

  • Originator Nationwide Children's Hospital; Ohio State University
  • Developer Amicus Therapeutics
  • Class Gene therapies
  • Mechanism of Action CLN3 protein expression stimulants; Gene transference
  • Orphan Drug Status

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

    Yes - Neuronal ceroid lipofuscinosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Neuronal ceroid lipofuscinosis

Most Recent Events

  • 27 Apr 2026 Amicus Therapeutics has been acquired by BioMarin Pharmaceutical
  • 31 Jan 2024 Amicus Therapeutics terminates its license for CLN 301
  • 25 Feb 2021 AAV9 CLN3 is still in phase I/II trials for Neuronal ceroid lipofuscinosis (In children) in USA (Intrathecal) (NCT03770572)

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