Drug Type: Gene Therapy
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
 Treeway Pipeline. Treeway, 2015. Accessed 11 Mar 2016 from http://www.treeway.nl/pipeline.
 Treeway to collaborate on developing AAV5-GDNF gene therapy for amyotrophic lateral sclerosis (ALS). FirstWord Pharma, 8 Dec 2015. Accessed 11 March 2016 from http://www.firstwordpharma.com/node/1338949?tsid=33#axzz42fVwf3bf.
 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 http://www.prnewswire.com/news-releases/treeway-announces-license-and-collaboration-agreement-with-uniqure-to-develop-a-gene-therapy-for-amyotrophic-lateral-sclerosis-als-288529301.html.
Last updated March 11th, 2016