Regulus Therapeutics, Inc. (NASDAQ:RGLS) is a biopharmaceutical company based in San Diego, California, focused on the discovery and development of innovative therapeutics targeting microRNAs. By modulating microRNA pathways, Regulus aims to address the root causes of disease at the genetic regulation level, with programs spanning liver disease, renal disorders and oncology. The company leverages its proprietary microRNA platform to identify and optimize oligonucleotide drug candidates that can either inhibit or mimic specific microRNAs involved in disease pathology.
At the core of Regulus’s pipeline are several lead candidates, including RG-012 for the treatment of Alport syndrome, a rare genetic kidney disease, and RGLS4326 for autosomal dominant polycystic kidney disease (ADPKD). Earlier-stage programs target fibrosis and metabolic disorders, and the company has broad collaborations with global pharmaceutical partners to validate and advance these therapeutic candidates. Regulus combines in-house discovery capabilities with preclinical and clinical development expertise to progress compounds from target identification through human trials.
Founded in 2007, Regulus has built a robust intellectual property portfolio covering multiple microRNA targets and delivery technologies. Its early collaborations with major pharmaceutical companies underscored the strategic value of microRNA-based drug discovery. Operating out of the biotechnology cluster in San Diego, Regulus benefits from proximity to leading academic institutions and specialized contract research organizations, supporting both innovation and execution across its research and development activities.
Regulus Therapeutics serves patient populations worldwide through its development programs and strategic alliances. While the company’s primary clinical activities and trial sites are in North America, it maintains global research collaborations and regulatory engagements to support future market entry in Europe and Asia. Regulus is led by an experienced management team with deep expertise in RNA-based drug discovery, clinical development and commercial strategy, positioning the company to advance novel therapies for unmet medical needs.
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