Regulus aims to develop important, novel oligonucleotide therapeutics that are designed to inhibit dysregulated microRNA targets for the benefit of patients with genetically based orphan diseases. We are focused on orphan kidney diseases where microRNA genetic drivers are implicated and there are clear unmet medical needs with an initial focus on orphan kidney diseases, including Autosomal Dominant Polycystic Kidney Disease (ADPKD).

Beyond genetic kidney disease, we are excited about the potential applications of oligonucleotide inhibitors of microRNA for additional genetically defined diseases.

Our technology is based on our position as exclusive licensee of IP provided by Alnylam and Ionis, two leaders in oligonucleotide-based therapeutics, along with our continued novel discoveries since inception. 

Home Page Ticker
Press Releases.
September 12, 2022

Phase 1b Multiple Ascending Dose (MAD) Study Initiated RGLS8429 in healthy volunteers was well-tolerated with dose-proportional PK Topline data from first cohort of patients with ADPKD anticipated...

September 02, 2022

Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the "Company" or "Regulus"), today...

August 11, 2022

IND accepted and first subject dosed in the Phase 1 Single-Ascending Dose (SAD) study of RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) RGLS8429 granted Orphan...

More press releases