Regulus and UCSD Partner to Develop miRNA-Targeting Anti-Angiogenesis Therapeutics

Collaboration will combine Regulus’ miRNA platform with UCSD’s expertise in angiogenesis models.

Regulus Therapeutics is teaming up with the University of California, San Diego (UCSD) to develop microRNA (miRNA)-based therapeutics against angiogenic diseases. Funded by a University of California Discovery Grant, the project will combine Regulus’ miRNA platform with UCSD’s expertise in animal models of angiogenesis, to discover anti-angiogenic miRNA-targeted therapeutics.

UCSD research published last year demonstrated that microRNA-132 (miR-132) functions as an angiogenic switch that turns on angiogenesis in quiescent endothelial cells, and targeting the molecule with an anti-miR-132 decreases blood vessel formation, explains David A Cheresh, Ph.D., who is principal investigator on the grant and professor of pathology at the UCSD School of Medicine and associate director for translational research at UCSD Moores Cancer Center. “The objective of our collaborative work with Regulus is to advance these initial discoveries and discover additional microRNAs involved in angiogenic diseases.”

Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals to develop microRNA therapeutics against multiple diseases. The firm’s preclinical pipeline includes both in house and partnered programs targeting fibrosis, HCV, immune-related diseases, metabolism and cardiovascular diseases, and cancer.

Lead fibrosis program, partnered with sanofi-aventis, targets microRNA-21 (miR-21). The firm claims studies in animal models have demonstrated treatment with anti-miR-21 can reverse fibrosis and significantly improve cardiac function in mice with failing hearts. Regulus and sanofi-aventis signed their fibrosis-focused miRNA therapeutics deal in June 2010. At the time Regulus claimed the deal represented the largest ever microRNA partnership, valued at potentially over $750 million.

Its lead HCV program, partnered with GlaxoSmithKline (GSK), targets microRNA-122 (miR-122). Regulus says preclinical studies suggest that anti-miR-122 can both reduce HCV infection and improve HCV-associated pathologies like steatosis. GSK and Regulus’ are also partnering on an immuno-inflammatory diseases project that targets microRNA-155 (miR-155). The firms inked their first, potentially $600 million immuno-inflammatory diseases miRNA therapeutics collaboration agreement in 2008. In February 2010 they announced a second collaboration to develop and commercialize microRNA therapeutics targeting microRNA-122 for the treatment of hepatitis C infection. This deal could be worth another $150 million to Regulus.

The firm’s in house projects include a cardiovascular/metabolic disease program targeting microRNA-33 (miR-33), and oncology programs focused on microRNA-21(miR-21) and microRNA-34 (miR-34).