Drug Type: Gene Therapy
Conditions: ALS, Parkinson’s disease
Mechanism Type: Neuroprotection
Mechanism: The approach aims to help protect motor neurons in ALS in part, by stimulating axonal outgrowth and protecting against glutamate-induced excitotoxicity.
U.S. Status for ALS: Preclinical
 Intraspinal delivery of AAV2-NRTN for ALS – a dose-ranging study of safety, tolerability, biodistribution, and efficacy. Gross, SK et al. 2017 Meeting of the Society of Neuroscience.
 The ongoing pursuit of neuroprotective therapies in Parkinson disease. Athauda D, Foltynie T. Nat Rev Neurol. 2015 Jan;11(1):25-40.
Last updated February 15th, 2018