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. They 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, there are potential applications of oligonucleotide inhibitors of microRNA for additional genetically defined diseases.

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