BOULDER, Colo.—A unique industry/academia collaboration on the discovery and development of miRNA (microRNA) has been formed by the University of Colorado (CU) and biopharmaceutical firm miRagen Therapeutics Inc., due to a possible life-saving cardiovascular study conducted by the university.
The sponsored research arrangement, announced Oct. 26, gives miRagen access to the data—as well as the intellectual property rights—linked to the heart failure study conducted at CU's Cardiovascular Institute, titled, "Beta Blocker Effects on Remodeling and Gene Expression," or BORG.
The BORG study followed 63 chronic heart-failure/nonischemic dilated cardiomyopathy patients for an 18-month period. Measurements of chamber remodeling and messenger RNA (mRNA) as well as miRNA expression at baseline, three months and 12 months were made.
Additionally, a licensing deal will enable the company to commercialize intellectual property associated with discoveries made during the research project. Analysis of the completed study, funded by miRagen, will provide the company with data on miRNA changes in human heart failure patients followed over two years with associated disease outcomes.
Financial details of the agreements have not been disclosed. It is the hope of both parties that this arrangement will lead to more effective therapies to treat individuals suffering from cardiovascular disease, the leading cause of death globally and an enormous burden on global healthcare systems.
Dr. William S. Marshall, president and CEO of miRagen, says the collaboration is "an example of translating basic academic research and understanding of microRNA and gene expression changes associated with heart failure rapidly forward to help identify and validate completely novel approaches to treatment of disease."
"Our relationship with Mike (Dr. Michael R. Bristow, co-director of the Cardiovascular Institute, who also led the BORG study and co-founder of miRagen) gave us early access to the planned study," Marshall tells ddn. "The study itself is very powerful in that we are going to monitor microRNA levels in human heart tissue from heart failure patients followed over time with associated disease outcomes. The information will be very valuable in confirming the disease relevance of specific microRNAs in heart failure—and may point to important biomarker candidates as well."
Elaborating further, Marshall says the BORG study provides miRagen with access to unique data in human patients with heart failure; the ability to analyze miRNA levels, as well as gene expression changes, in a given patient at specific points in time in their disease progression, which may provide a very powerful tool in stratifying miRNA targets; and human data that contain novel information on miRNAs and their relationships to myocardial remodeling and mRNA behavior, which will be very useful in target selection for therapeutic miRNA approaches.
"The sponsored research agreement will support the analysis of miRNA and gene expression changes from the BORG study, it provides funding for the miRNA and gene expression profiling experiments performed on the study samples and then provides access to all discoveries generated from the studies," Marshall says.
The licensing agreement not only "enables the company to commercialize intellectual property associated with discoveries made during the research project, but it provides a piece of existing IP that may serve as the basis for expansion based on the follow-on studies." Marshall says. "Further analysis of the completed study, funded by miRagen, will provide the company with data on miRNA changes in human heart failure patients followed over two years with associated disease outcomes. The structure of the deal involves equity participation in miRagen, as well as downstream royalties for products developed from the research."
CU's Michael R. Bristow, who led the BORG study, says the data "will be very useful to miRagen in target selection for their therapeutic miRNA approaches. In drug development, animal models are of course very valuable, but for target validation as well as novel target discovery, human data is vitally important."
David Poticha, senior licensing manager at CU's Technology Transfer Office, says, "The team that has been assembled by miRagen has a strong history of successfully developing Colorado-based biotechnology companies, and we firmly believe miRagen is the right and best partner to help commercialize the microRNA technologies."
Additionally, the university "has a uniquely valuable asset in housing a very large heart tissue bank which has allowed for analysis of microRNA expression in healthy and diseased heart tissues from human patients," Poticha says.
"The team at the University of Colorado along with the team at the University of Texas, Southwestern (UTSW) saw the potential for creating a startup company around this technology," he says. "MiRagen was thus formed a couple of years ago and initially forged its first licensing relationship with UTSW. They obtained an option to the University of Colorado technology over a year and a half ago, which was finalized into the license."
What makes this collaboration so intriguing?
"It is the assembly of talent at both the University of Colorado and miRagen—in addition to miRagen's access to the university's clinical programs and specimens—which are unique and not accessible to other companies not engaged with the university's cardiovascular department," Poticha says.
