Regulus Therapeutics (NASDAQ:RGLS), Inc. (NASDAQ:RGLS) is a clinical-stage biopharmaceutical company dedicated to developing novel therapeutics that target microRNAs, small non-coding RNA molecules known to regulate gene expression. Founded in 2007 as a joint venture between Alnylam Pharmaceuticals and Ionis Pharmaceuticals, Regulus has built a proprietary platform that enables the discovery and development of microRNA-targeted drugs. Headquartered in San Diego, California, the company’s research efforts focus on delivering innovative treatment options for diseases with significant unmet medical needs.
The company’s pipeline comprises several internally discovered and partnered programs across key therapeutic areas, including renal disease, metabolic disorders, and infectious diseases. Lead candidates such as RG-012, designed to inhibit microRNA-21 in patients with Alport syndrome, and RGLS8429, targeting microRNA-17 for the treatment of focal segmental glomerulosclerosis (FSGS), exemplify Regulus’s focus on precision molecular medicine. Additional pipeline assets investigate microRNA mechanisms in autosomal dominant polycystic kidney disease (ADPKD) and cardiometabolic indications, reflecting a diversified approach to microRNA modulation.
Regulus operates on a hybrid model of in-house research and strategic collaborations, leveraging partnerships to advance its microRNA platform. The company has forged alliances with leading pharmaceutical organizations and academic institutions to access complementary expertise and accelerate clinical development. These collaborations have expanded Regulus’s global reach, enabling it to conduct multi-regional clinical trials and facilitate regulatory interactions across North America, Europe, and Asia Pacific.
Under the leadership of President and Chief Executive Officer Jonathan H. Genzen, Regulus Therapeutics is guided by a team of industry veterans with extensive experience in RNA biology, drug development, and commercialization. The company’s board and scientific advisory panels include recognized experts in microRNA research and clinical development, supporting its mission to translate pioneering science into transformative therapies. Through its commitment to microRNA innovation, Regulus aspires to deliver novel treatment paradigms that address both rare and prevalent diseases worldwide.
