Therapeutic Areas

Regulus is developing single-stranded oligonucleotides, which are chemically synthesized chains of nucleotides that are mirror images of specific target microRNAs. We incorporate proprietary chemical modifications to enhance drug properties such as potency, stability and tissue distribution. We refer to these chemically modified oligonucleotides as anti-miRs. Each anti-miR is designed to bind with and inhibit a specific microRNA target that is up-regulated in a cell and that is involved in the disease state. In binding to the microRNA, anti-miRs correct the dysregulation and return diseased cells to their healthy state. We have demonstrated therapeutic benefits of our anti-miRs in at least 20 different preclinical models of human diseases. Regulus is currently optimizing anti-miRs in several distinct programs, both independently and with our strategic alliance partners, AstraZeneca and Sanofi.

Initial microRNA Development Programs
microRNA Target anti-miR Program Commercial Rights
miR-122 RG-101 for Hepatitis C Virus Infection Regulus
miR-21 RG-012 for Kidney Fibrosis
Hepatocelluar Carcinoma
miR-103/107 RG-125 for NASH in patients with type 2 diabetes/pre-diabetes
miR-10b Glioblastoma Regulus
miR-221 Hepatocelluar Carcinoma Sanofi

Hepatitis C Virus 

miR-122 is a liver-expressed microRNA that has been shown to be a critical endogenous “host factor” for the replication of HCV, and anti-miRs targeting miR-122 have been shown to block HCV infection.


We have identified microRNA signatures in several different models of fibrotic disease. These data suggest that microRNAs drive the pathogenesis of fibrotic disease.


Many if not all cancers display abnormal expression of microRNAs. This dysregulation has been shown to promote tumor progression and metastasis. Treating cancers with microRNA therapeutics offers a promising new approach to many different cancers.


microRNAs have been shown to be key regulators of glucose and lipid metabolism. The modulation of specific microRNAs is a promising strategy to treat metabolic disease.


In addition to the select drug discovery and development programs highlighted above, Regulus has a significant internal exploratory effort focused on new target identification and assay development. In addition to internal discovery efforts, Regulus also leverages its extensive network of leading academic collaborators to discover new microRNAs and support microRNA discovery efforts that feed the Company’s pipeline.