Research programme: amyotrophic lateral sclerosis gene therapy - Treeway

Drug Profile

Research programme: amyotrophic lateral sclerosis gene therapy - Treeway

Alternative Names: AAV5-GDNF gene therapy; ALS gene theapy - uniQure/Treeway; TW 002

Latest Information Update: 26 Jan 2016

Price : $50 *
* 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. Adis is an information provider. We do not sell or distribute the pharmaceutical compounds written about in this database.

At a glance

  • Originator uniQure
  • Developer Treeway
  • Class Gene therapies; Nerve growth factors
  • Mechanism of Action Gene silencing; Gene transference; Glial cell line-derived neurotrophic factor agonists
  • Orphan Drug Status

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

  • New Molecular Entity No

Highest Development Phases

  • Preclinical Amyotrophic lateral sclerosis

Most Recent Events

  • 26 Jan 2016 Preclinical trials in Amyotrophic lateral sclerosis in Netherlands (Parenteral)
  • 05 Jan 2016 Treeway plans the proof of concept clinical trial for Amyotrophic lateral sclerosis (Treeway pipeline, January 2016)
  • 15 Jan 2015 Early research in Amyotrophic lateral sclerosis in Netherlands (Parenteral)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top