INVESTORS.

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.