Drug Name: VY-SOD101
Company: Voyager Therapeutics
Location: Cambridge, MA
Website: http://voyagertherapeutics.com/programs.php
Drug Type: Gene Therapy
Conditions: SOD1 ALS
Mechanism Type: SOD1-Lowering Therapy
Mechanism: VY-SOD101 is an adeno-associated viral (AAV)-based gene therapy that aims to slow progression of SOD1 ALS by reducing levels of ALS-linked mutant SOD1. SOD1 mutations account for approximately 20% of familial forms of ALS.
U.S. Status for ALS: Preclinical

[1] Voyager Therapeutics Highlights Programs in Parkinson’s Disease and ALS; Announces Expansion of CNS Pipeline at R&D Day. Globe Newswire. April 29, 2016.

Last updated March 8th, 2017

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