Regulus Therapeutics Inc., a biopharmaceutical company, focuses on discovering and developing drugs that target microRNAs to treat a range of diseases. The company is using its microRNA product platform to develop chemically modified, single-stranded oligonucleotides that it calls anti-miRs. The company uses these anti-miRs to modulate microRNAs and by doing so return diseased cells to their healthy state. The company is pursuing various microRNA targets and is focusing its proprietary efforts in oncology and orphan diseases. The company is optimizing anti-miRs in various programs, both independently and with its strategic alliance partners, AstraZeneca AB; GlaxoSmithKline plc; and Sanofi-Aventis. The company also has a collaboration agreement with Biogen Idec MA Inc. to evaluate the use of microRNA signatures as a biomarker for human patients with multiple sclerosis. The company is developing single-stranded oligonucleotides, which are chemically synthesized chains of nucleotides that are mirror images of specific target microRNAs. It incorporates proprietary chemical modifications to improve drug properties, such as potency, stability, and tissue distribution. The company refers to these chemically modified oligonucleotides as anti-miRs. Each anti-miR is designed to bind with and inhibit a specific microRNA target that is up-regulated in a cell and that is involved in the disease state. The company has demonstrated therapeutic benefits of its anti-miRs in approximately 20 different preclinical models of human diseases. The company has identified and validated various microRNA targets across various disease categories and is working independently and with its strategic alliance partners to optimize anti-miR development candidates. Regulus microMarkers In January 2014, the company established Regulus microMarkers, a research and development division focused on identifying microRNAs as biomarkers of human disease, which is designed to support its therapeutic pipeline, collaborators and strategic partners. Through its microRNA target identification and validation efforts, the company has developed proprietary technologies for microRNA profiling and analysis of human clinical samples, such as tissue. Strategy The major elements of the company’s strategy are to execute on its ‘Clinical Map Initiative’; rapidly advance its initial programs into clinical development; focus its resources on developing drugs for niche indications or orphan diseases; selectively form strategic alliances to augment its expertise and accelerate development and commercialization; selectively use its microRNA product platform to develop additional targets; develop microRNA biomarkers to support therapeutic product candidates; and maintain scientific and intellectual leadership in the microRNA field. Intellectual Property The company has intellectual property position and know-how relating to the development and commercialization of microRNA therapeutics, including approximately 200 patents or patent applications that it owns or has in-licensed from academic institutions and third parties, including its founding companies, Alnylam and Isis, related to microRNA and microRNA drug products; and approximately 850 patents or patent applications licensed from its founding companies, Alnylam and Isis, related to RNA technologies, including patent and patent applications relating to chemical modification of oligonucleotides that are useful for microRNA therapeutics. The company’s portfolio of exclusively and jointly owned patent and patent applications includes approximately 15 U.S. and foreign patents and approximately 80 U.S. and foreign applications with claims to methods of composition-of-matter related to its microRNA drug products and microRNA product platform. It jointly owns approximately 10 of the patents and pending applications, including those claiming methods for treating liver cancer, including hepatocellular carcinoma, using anti-miRs targeting miR-21. The patents have statutory expiration dates in 2024, 2025, 2026, or 2029. Any patents that might issue from the pending applications would have statutory expiration dates between 2024 and 2033. Research and Development Expenses In 2013, the company’s research and development expenses were $29.9 million. Government Regulation Any product candidate that the company develops must be approved by the FDA before it might be legally marketed in the United States and by the appropriate foreign regulatory agency before it might be legally marketed in foreign countries. Competition The company competes with Groove Biopharma, Inc.; InteRNA Technologies B.V.; miRagen Therapeutics, Inc.; MiReven Pty Ltd; Mirna Therapeutics, Inc.; Microlin Bio, Inc.; and Santaris Pharma A/S. History Regulus Therapeutics Inc. was founded in the state of Delaware in 2007.
regulus therapeutics inc
3545 John Hopkins Court
San Diego, CA 92121
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