Regulus Therapeutics
Regulus Therapeutics works on 1 rare disease tracked on Trial Friend, including Polycystic Kidney Disease.
Regulus Therapeutics was acquired by Novartis (completed June 2025). The company developed farabursen (formerly RGLS8429), a microRNA-targeting oligonucleotide that inhibits miR-17 for treating autosomal dominant polycystic kidney disease. Farabursen is now part of Novartis's renal disease pipeline.
Focus areas at Regulus Therapeutics
As a rare disease specialist, Regulus Therapeutics has active clinical trial programs and drug development efforts across 1 rare diseases, including Polycystic Kidney Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Regulus Therapeutics, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Regulus Therapeutics is headquartered in San Diego, United States, founded in 2007, a subsidiary of Novartis. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Active Clinical Trials
FDA-Approved Drugs
Rare Disease Focus Areas (1)
Diseases targeted by Regulus Therapeutics's clinical trial and drug development programs
Patient Resources
Organizations and resources related to Regulus Therapeutics's rare disease focus areas
Frequently Asked Questions About Regulus Therapeutics
Common questions about Regulus Therapeutics's rare disease programs, clinical trials, and treatments.