Regulus Therapeutics
Rare disease drugmaker · part of Novartis · based in San Diego, United States · founded 2007 · novartis.com
Regulus Therapeutics works on Polycystic Kidney Disease, one of the rare diseases we track.
Regulus Therapeutics was a San Diego biotech that Novartis acquired on June 25, 2025. Regulus developed farabursen (formerly RGLS8429), an oligonucleotide that blocks a small RNA called miR-17 to slow cyst growth in autosomal dominant polycystic kidney disease. Novartis now lists farabursen as CYX082 in Phase 1 on its pipeline page, with no filing date given.
Regulus Therapeutics clinical trials
We did not find active Regulus Therapeutics trials for the rare diseases we track on ClinicalTrials.gov right now.
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- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
More about Regulus Therapeutics
Regulus no longer runs trials under its own name.
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 active and recruiting rare disease 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.
Keep reading about Regulus Therapeutics
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.
Rare diseases Regulus Therapeutics works on
Each disease page lists every open trial, not only Regulus Therapeutics's, plus approved treatments.
Polycystic Kidney Disease is a genetic disorder characterized by the growth of thousands of cysts in the kidneys, gradually replacing normal kidney tissue and leading to kidney failure.
How common: Autosomal dominant PKD affects about 1 in 400 to 1,000 people worldwide and the recessive form about 1 in 20,000 children; about 500,000 people in the United States have PKD (NIDDK)
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Regulus Therapeutics's own site is novartis.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- PKD FoundationPolycystic Kidney Disease
- National Kidney FoundationPolycystic Kidney Disease