miRagen Therapeutics, Inc. Exclusively Licenses Intellectual Property from the University of Glasgow for microRNA-145 Targeting in the Treatment of Pulmonary Arterial Hypertension

• IP generated as a result of collaboration between researchers from miRagen, the University of Glasgow and the University of Cambridge
• Preliminary results in pre-clinical models show significant promise in treatment of a disease with very high unmet medical need

BOULDER, Colo.--(BUSINESS WIRE)--miRagen Therapeutics, Inc., a biopharmaceutical company developing innovative microRNA (miRNA)-based therapeutics, and the University of Glasgow, a world-leader in cardiovascular research, announced today that they have signed an exclusive license agreement for intellectual property they developed as part of an ongoing collaboration for microRNA-145 in pulmonary arterial hypertension (PAH). This licensing agreement provides miRagen with exclusive rights to an important discovery made in collaboration with the University of Glasgow and the University of Cambridge, enabling miRagen to advance miR-145 inhibition as a potential therapeutic approach for PAH.

“Clearly there is an immediate need for new PAH therapies, particularly those that have the potential to change the underlying cause of this disease”

PAH is a devastating, progressive disease driven by abnormal cellular proliferation and obstruction of small blood vessels in the lung, resulting in elevated pulmonary arterial pressure and, ultimately, fatal right-sided heart failure. Existing vasodilator therapies do not adequately address the fundamental disease mechanisms, a fact highlighted by high mortality rates. The median period of survival after diagnosis, based on an early U.S. National Institutes of Health Registry with prospective follow-up, was less than 3 years with a mean age of 36 years. At present, average survival after diagnosis in adults is estimated at 5 years, with a similarly poor overall prognosis in children.¹

“Clearly there is an immediate need for new PAH therapies, particularly those that have the potential to change the underlying cause of this disease,” said Andrew Baker, British Heart Foundation Professor of Translational Cardiovascular Science at Institute of Cardiovascular and Medical Sciences at the University of Glasgow. “By targeting the underlying vessel remodeling process, a miR-145-based therapy holds the potential to be disease-modifying.” The research also involved Professor Mandy MacLean from the University of Glasgow and Professor Nicholas Morrell, based at the University of Cambridge, and was funded by the British Heart Foundation.

“The results we’ve generated during our collaboration suggest that microRNA-145 controls the expression of a complex biological network that is important in the development of this devastating disease,” said William S. Marshall, Ph.D., President and Chief Executive Officer of miRagen Therapeutics, Inc. “This agreement is one part of miRagen’s core strategy to leverage our comprehensive expertise in microRNA biology and combine optimal targets and chemistries for the development of groundbreaking therapeutics.”

The University of Glasgow was advised by Dundas & Wilson CS LLP in its negotiations with miRagen. Carina Healy, Partner, said: "It has been fantastic and extremely rewarding to work on a deal of this kind. The licensing of this intellectual property means that a potentially lifesaving technology will be developed to its full potential. This is also an excellent example of quality intellectual property being generated in the UK by leading Universities in the field, and we are delighted to have been able to assist the University of Glasgow in concluding the exclusive licence with miRagen."

About miRagen Therapeutics, Inc.

miRagen Therapeutics, Inc. is a biopharmaceutical company focused on the discovery and development of innovative microRNA-based therapeutics in disease areas with high unmet medical need. The company leverages insights from leading laboratories to evaluate and advance high-potential therapies for its own pipeline or in conjunction with strategic partners. For several cardiovascular disease programs, miRagen has partnered with Les Laboratoires Servier, a leading European pharmaceutical company in the area of cardiovascular disease. miRagen retains all rights for the Servier-partnered programs in the United States and Japan. With its commercial and academic alliances, miRagen strives to harness the power of microRNA biology and chemistry by translating discoveries into breakthrough therapies that improve human health. For more information, please visit www.miragenrx.com.

About the University of Glasgow

Founded in 1451, the University of Glasgow is a leading research-intensive institution ranked 54 in the 2012/13 QS World University Rankings. The University employs more than 2,000 active researchers with an impressive 75% of academic staff contributing to subjects where the majority of research is rated world-leading or internationally excellent. The University’s Institute of Cardiovascular and Medical Sciences is at the forefront of discovering new insights and treatments for cardiovascular disease.

About The University of Cambridge

The University of Cambridge's mission is to contribute to society through the pursuit of education, learning and research at the highest international levels of excellence. Cambridge's reputation for excellence is known internationally and reflects the scholastic achievements of its academics and students, as well as the world-class original research carried out by its staff. Some of the most significant scientific breakthroughs occurred at the University, including the splitting of the atom, invention of the jet engine and the discoveries of stem cells, plate tectonics, pulsars and the structure of DNA. From Isaac Newton to Stephen Hawking, the University has nurtured some of history's greatest minds and has produced more Nobel Prize winners than any other institution with 89 laureates.

About the British Heart Foundation

The British Heart Foundation (BHF) is the nation’s heart charity, dedicated to saving lives through pioneering research, patient care, campaigning for change and by providing vital information. But we urgently need help. We rely on donations of time and money to continue our life-saving work. Because together we can beat heart disease.

For more information visit bhf.org.uk/pressoffice.

¹ T. Thenappan et al. (2007). A USA-based registry for pulmonary arterial hypertension: 1982–2006. European Respiratory Journal, 30(6), 1103–1110.