Company: miRagen Therapeutics
Drug Type: Gene Therapy
Mechanism Type: Immunomodulation
Mechanism: MRG-107 is an oligonucleotide drug that targets miR-155, a microRNA that is elevated in the spinal cord of patients with ALS, and in ALS mouse models. It is emerging as a key player in neuroinflammation and immune regulation, and therefore is a therapeutic target of interest in ALS.
U.S. Status for ALS: Preclinical
 miRagen Therapeutics – Pipeline. miRagen Therapeutics, 2016. Accessed 11 Mar 2016 from http://miragentherapeutics.com/pipeline/.
 Targeting miR-155 restores abnormal microglia and attenuates disease in SOD1 mice. Butovsky, O et al. Ann Neurol. 2015 Jan; 77(1): 75–99.
 Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice. Koval, ED et al. Hum Mol Genet. 2013 Oct 15;22(20):4127-35.
Last updated March 11th, 2019