Not Disclosed

Drug Name: Not Disclosed
Company: AveXis
Location: US-Ohio
Website: https://avexis.com/
Drug Type: Gene Therapy
Conditions: ALS
Mechanism Type: SOD1-Lowering Therapy
Mechanism: This experimental gene therapy aims to protect motor neurons in SOD1 ALS by reducing levels of misfolded SOD1.
U.S. Status for ALS: Preclinical

References:
[1] Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Foust KD, et al. Mol Ther. 2013 Dec;21(12):2148-59.
[2] AveXis and REGENXBIO Announce New Exclusive Worldwide Licenses for the Treatment of Two Rare Neurological Monogenic Disorders Using NAV AAV9 Vector. Accessed 15 Feb 2018 from http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2279618.

Last updated February 15th, 2018

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