Drug Name: TW002
Company: Treeway
Location: Netherlands
Drug Type: Gene Therapy
Conditions: ALS
Mechanism Type: Neuroprotection
Mechanism: TW002 is a gene therapy for ALS, which uses AAV5 vectors to administer glial cell line-derived neurotrophic factor (GDNF) in order to provide neurotrophic support for motor neurons.
U.S. Status for ALS: Preclinical

[1] Treeway Pipeline. Treeway, 2015. Accessed 11 Mar 2016 from
[2] Treeway to collaborate on developing AAV5-GDNF gene therapy for amyotrophic lateral sclerosis (ALS). FirstWord Pharma, 8 Dec 2015. Accessed 11 March 2016 from
[3] UniQure exclusively licensed this technology to Treeway, and the companies are collaborating on its development for ALS. PR Newswire, 14 Jan 2015. Accessed 11 Mar 2016 from

Last updated March 11th, 2016

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