Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

AAV9 CLN3

Next Previous
X
Search
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

Price :

$50 *

Note:
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

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)

You need to be a logged in or subscribed to view this content Request demo

If your organization or you do not have a subscription, try one of the following:
  • Contacting your organization’s admin about adding this content to your AdisInsight subscription
  • Buying a PDF version of any individual profile
  • Request a free trial
If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top