Regulus Therapeutics

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company that directs its efforts toward discovering and developing medicines that target microRNAs, which are small, non-coding RNA molecules involved in regulating gene expression. The company was established in September 2007 as a joint venture between two RNA-focused firms, Alnylam Pharmaceuticals and Isis Pharmaceuticals (now Ionis Pharmaceuticals). This formation combined the intellectual property and technological expertise of its parent companies, creating a significant force in the microRNA therapeutics space. Kleanthis G. Xanthopoulos was the founding President and CEO. Regulus went public in October 2012 with an IPO on the NASDAQ, raising approximately $80.9 million to advance its pipeline.

The company's business model is centered on high-risk, high-reward research and development, which is typical for a clinical-stage entity. As such, revenue has been primarily generated through strategic collaborations and license agreements with larger pharmaceutical partners, including upfront payments, milestone payments, and potential future royalties. Significant alliances have been formed with companies like GlaxoSmithKline, Sanofi, and AstraZeneca for the development of microRNA therapeutics in areas such as inflammatory diseases and fibrosis. A substantial portion of the company's capital is reinvested into its R&D programs to advance its pipeline through preclinical and clinical trials.

Regulus focuses on developing treatments for genetically based orphan diseases with high unmet medical needs. Its core technology involves identifying microRNAs linked to specific diseases and then designing and synthesizing oligonucleotide therapeutics, or "anti-miRs," to modulate their activity. The company's lead product candidate is farabursen (also known as RGLS8429), a next-generation oligonucleotide designed to inhibit miR-17 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD). ADPKD is a genetic disorder that causes cysts to grow in the kidneys, which can lead to kidney failure. Farabursen has shown in preclinical models and early clinical trials to improve kidney function and size. The drug has demonstrated a favorable safety and pharmacokinetic profile in Phase 1 clinical trials. In April 2025, Novartis announced an agreement to acquire Regulus, a deal centered on the potential of farabursen. As of June 2025, Regulus operates as a subsidiary of Novartis AG.

Keywords: microRNA therapeutics, oligonucleotide, RNA interference, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, autosomal dominant polycystic kidney disease, ADPKD, farabursen, RGLS8429, anti-miR, orphan diseases, genetic kidney disease, clinical-stage biopharmaceutical, gene expression, Novartis, drug discovery, RNA-targeted therapies, San Diego biotechnology, fibrotic diseases, rare disease therapeutics