RGLS

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing drugs targeting microRNAs to treat diseases. The Company is leveraging its oligonucleotide drug discovery and development to develop a pipeline complemented by a rich intellectual property estate in the microRNA field. The Company’s lead product candidate includes RGLS8429. The Company is developing single-stranded oligonucleotides, which are chemically synthesized chains of nucleotides that are complementary to (thereby pairing with) the target microRNA. RGLS8429 is an anti-miR next-generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD). RGLS8429 maintains beneficial attributes, such as preferential kidney exposure and similar PK profile, miR-17 inhibition potency and duration of action in the kidney, equal potency in vitro and in vivo efficacy studies.