RGLS Profile
Regulus Therapeutics Inc., based in San Diego, California, is a clinical-stage biopharmaceutical company specializing in the development of innovative therapies targeting microRNAs to address a range of complex diseases. The company's primary focus is on advancing its drug candidates through clinical trials, aiming to harness the potential of microRNA modulation to treat various conditions. Regulus’s lead product candidates include RG-012 and RGLS8429, each targeting specific microRNAs with the goal of addressing severe and chronic diseases.
RG-012 is an anti-miR therapy designed to target miR-21 and is currently undergoing Phase II clinical trials for the treatment of Alport syndrome, a rare and life-threatening genetic kidney disease characterized by progressive kidney failure. This therapeutic approach aims to mitigate the disease's progression by interfering with disease-related microRNA pathways. Additionally, RGLS8429, an anti-miR targeting miR-17, is in Phase 1b clinical studies for the treatment of autosomal dominant polycystic kidney disease (ADPKD). ADPKD is a genetic disorder marked by the growth of numerous cysts in the kidneys, which can lead to kidney failure and other complications.
Beyond its clinical trials, Regulus Therapeutics is advancing a pipeline of preclinical drug candidates focused on target organ-selective delivery strategies. These innovative approaches are intended to enhance the precision and efficacy of treatment by ensuring that therapeutic agents are delivered specifically to affected organs or tissues. The company's research and development efforts reflect a commitment to exploring novel therapeutic avenues and advancing the field of microRNA-targeted therapies.
Incorporated in 2007, Regulus Therapeutics Inc. has established itself as a key player in the biopharmaceutical sector, with a dedicated focus on microRNA-based drug development. The company continues to expand its research initiatives and clinical programs, aiming to bring transformative treatments to patients with unmet medical needs and to drive forward the science of microRNA therapeutics.
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