Regulus Therapeutics Inc. (Nasdaq:RGLS) is a biopharmaceutical company structured around discovery and development of innovative medicines targeting microRNAs. The firm's principals have leveraged in-house oligonucleotide drug discovery and development expertise to develop a well-balanced microRNA therapeutics pipeline complemented by a maturing microMarkersSM biomarkers platform. The firm has a strong IP base in place to retain its leadership in the microRNA field. Underdevelopment are RG-101, a GalNAc-conjugated anti-miR targeting microRNA-122 for the treatment of chronic hepatitis C virus infection, and RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport syndrome, a life-threatening kidney disease driven by genetic mutations with no approved therapy. Also of focus is RG-125, a GalNAc-conjugated anti-miR targeting microRNA-103/107 for the treatment of NASH in patients with type 2 diabetes/pre-diabetes. In a strategiic relationship AstraZeneca, Regulas entered Phase I clinical development and is is also advancing several programs toward clinical development in renal, hepatic and central nervous systems diseases, both independently and with strategic alliance partners: Sanofi and AstraZeneca. In Jan 2022, firm announced successful completion of pre-IND meeting with FDA re. the firm's next generation compound RGLS8429 as a potential treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
