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AAV9 CLN3

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Drug Profile

AAV9 CLN3

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

Latest Information Update: 02 Oct 2021

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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

  • 25 Feb 2021 AAV9 CLN3 is still in phase I/II trials for Neuronal ceroid lipofuscinosis (In children) in USA (Intrathecal) (NCT03770572)
  • 08 Feb 2021 Amicus Therapeutics plans to submit IND application for a clinical trial, in the second half of 2021
  • 08 Feb 2021 Efficacy and adverse events data from a phase I/IIa trial in Neuronal ceroid lipofuscinosis presented at the at the 17th Annual WORLDSymposium™ 2021 (WORLDSymposium-2021)
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