Drug Type: Gene Therapy
Conditions: ALS, Glaucoma
Mechanism Type: Neuroprotection, Astrocyte-Mediated Toxicity
Mechanism: This gene therapy aims to reduce TGFß signaling via the delivery of an antisense oligonucleotide targeting TGFß receptor type-II (TGFßRII). The approach may be protect motor neurons in ALS, according to preclinical studies, in part, by reducing astrocyte-mediated toxicity.
U.S. Status for ALS: Preclinical
 In vivo safety and efficacy evaluation of a novel 3rd generation antisense oligonucleotide against TGFßRII to treat amyotrophic lateral sclerosis. Peters, S et al. 2017 Annual Meeting of the Society of Neuroscience.
 Reactive Astrocytes Promote ALS-like Degeneration and Intracellular Protein Aggregation in Human Motor Neurons by Disrupting Autophagy through TGF-β1. Tripathi, P. et al. Stem Cell Reports. 2017 Aug 8;9(2):667-680.
Last updated February 15th, 2018