Regulus Therapeutics is a clinical-stage biopharmaceutical company focused on developing drugs that target microRNAs — RNA molecules that regulate gene expression — to treat diseases driven by microRNA dysregulation. Regulus has no approved products or commercial revenue. Its sole clinical-stage asset is farabursen, an anti-miR oligonucleotide that inhibits miR-17 to treat autosomal dominant polycystic kidney disease (ADPKD), a genetic disorder in which fluid-filled cysts form in the kidneys, leading to end-stage renal disease in roughly half of patients by age 60. Farabursen is designed for every-other-week administration, preferentially targets the kidney, and works by restoring proteins that are deficient in ADPKD patients. Regulus funds itself through equity and debt issuances and collaboration payments, with $75.8M in cash at year-end 2024. The company employs only 34 people and contracts out manufacturing. Regulus' near-term strategy is to advance farabursen through a pivotal Phase 3 trial, with the FDA indicating an accelerated approval pathway may be viable. Farabursen holds orphan drug designation for ADPKD, which would provide seven years of market exclusivity upon first approval. If commercialized, Regulus would sell farabursen through specialty pharmacy channels and would owe royalties to founding companies Alnylam Pharmaceuticals and Ionis Pharmaceuticals. Beyond farabursen, Regulus conducts preclinical research in liver, kidney, and CNS diseases, with a strategy of either advancing programs independently or partnering them with larger companies.