Not Disclosed

Drug Name: Not Disclosed
Company: AveXis
Location: US-Ohio
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

[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

Last updated February 15th, 2018

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