miRNA therapeutic specialist already holds composition of matter patents to both cardiovascular disease targets.

Regulus Therapeutics negotiated exclusive rights to New York University (NYU)-owned IP covering the therapeutic applications of targeting miRNA-33a (miR-33a) and miR-33b against metabolic diseases including atherosclerosis and metabolic syndromes. Regulus is developing miRNA therapeutics that target both miR-33a and miR-33b. The firm says its collaborative research with NYU led to the first demonstration that anti-miR oligonucleotides can reduce atherosclerotic plaque and increase levels of HDL.

The licensed IP relates to the findings that miR-33a and miR-33b downregulate genes involved in cholesterol transport and genes impacting on metabolic syndrome, Regulus explains. Further collaborative work with NYU researchers has demonstrated that in mice, which carry a single copy of miR-33, treatment using an anti-miR-33 reduces arterial lesions in models of atherosclerosis with established atherosclerotic plaques. The firm points out it has developed technologies capable of inhibiting both miR-33a and miR-33b that are carried by nonhuman primates and humans.

Regulus already holds rights to composition of matter patents for miR-33a and miR-33b sequence and complement, and various chemically modified anti-miR compounds targeting both the miRNAs, which have been discovered by the firm.

Regulus was established in 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals to focus on the development of RNA-based therapeutics that target miRNAs. The firm’s lead preclinical fibrosis program, targeting miR-21, is partnered with sanofi-aventis. This collaboration, signed in June 2010, included a $25 million up-front fee paid to Regulus, a $10 million equity investment in the firm, and annual research support for three years initially. The partnership will cover up to four mRNA targets, including Regulus’s lead fibrosis program targeting miR-21.

Regulus also has a miRNA therapeutics collaboration in place with GlaxoSmithKline, signed back in 2008, which is focused on HCV and immune-related diseases. The firm’s in house research is targeting metabolism and cardiovascular disease, and hepatocellular carcinoma.

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