ABS-0871

ABS-0871 is a novel, orally administered small molecule inhibitor of the transient receptor potential cation channel subfamily V member 4 (TRPV4). It is being developed by Actio Biosciences for the treatment of Charcot-Marie-Tooth disease type 2C (CMT2C), a severe hereditary neuropathy caused by mutations in the TRPV4 gene. These mutations lead to overactivity of the TRPV4 protein, resulting in progressive muscle weakness, vocal cord paralysis, respiratory complications, and other debilitating symptoms. By inhibiting TRPV4 activity, ABS-0871 aims to address the underlying genetic cause of CMT2C and improve patient outcomes. The drug has received both orphan drug designation and rare pediatric disease designation from the FDA and is currently in Phase 1 clinical trials for CMT2C. Preclinical studies have shown that ABS-0871 improves motor function and mobility in models of TRPV4-related diseases[1][2][3][5][6][7].