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Carrie Munk
The ALS Association

Christopher J. Morl
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miRagen Therapeutics Receives ALS Association Grant to Advance the Development of MRG-107

BOULDER, Colo. and WASHINGTON, D.C. (June 2, 2016)— Today, miRagen Therapeutics, Inc., a clinical-stage biopharmaceutical company developing innovative microRNA-based therapeutics and The ALS Association announced that miRagen has received the first installment of a Translational Research Advancing Therapy for ALS (TREAT ALS™) grant. This milestone-driven grant of up to $424,725 will be used to advance the development of MRG-107, a synthetic microRNA antagonist (LNA antimiR®) of microRNA-155 that is effective in pre-clinical models of amyotrophic lateral sclerosis (ALS).

“We are delighted to have received this grant from The ALS Association,” said William S. Marshall, President and Chief Executive Officer of miRagen. “MRG-107 is a mechanistically novel approach to the potential treatment of ALS patients and an example of miRagen’s focus on developing innovative product candidates for diseases where there is a significant unmet medical need.”

“The TREAT ALS™ portfolio is a diverse collection of ALS research aimed at finding treatments and a potential cure for ALS,” added Lucie Bruijn, Ph.D., M.B.A., Chief Scientist of The ALS Association. “We are pleased to support this research into MRG-107 being conducted by miRagen in collaboration with Howard Weiner, M.D., from the Ann Romney Center for Neurologic Diseases, Brigham and Women's Hospital, Boston.”

“Targeting mir-155 based on the work of Dr. Oleg Butovsky is a unique approach to modulate abnormal microglial responses in ALS,” added Dr. Weiner, and we are excited to collaborate with miRagen on the development of MRG-107.”

About microRNAs
MicroRNAs have emerged as an important class of small RNAs encoded in the genome, acting as master regulators of gene expression. Recent studies have indicated that microRNAs appear to be associated with many disease processes. Because they are thought to be single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

About MRG-107 and microRNA-155
MRG-107 is an antimiR (antagonist) of microRNA-155. In the spinal cords of patients with ALS miR-155 is inappropriately increased. miR-155 plays key roles in the inflammation and immune mechanisms that are implicated in ALS pathology. In preclinical models of ALS, inhibition of miR-155 alleviates symptoms and extends survival. ALS is a severe disease and the majority of patients ultimately succumb to breathing problems. Improvements in survival with existing drug treatments are modest and new therapeutic approaches are urgently needed.

About The ALS Association
The ALS Association is the only national non-profit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers and fostering government partnerships, The Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at

About miRagen Therapeutics, Inc.
miRagen Therapeutics, Inc., is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative microRNA (miRNA)-targeting therapies in disease areas of high unmet medical need. The Company seeks to leverage in-house expertise in miRNA biology, oligonucleotide chemistry, and drug development to evaluate and advance promising technologies and high-potential product candidates for its own pipeline and in conjunction with strategic collaborators. For certain cardiovascular disease programs, miRagen has a collaboration and license agreement with Servier, an independent French research-based pharmaceutical company. miRagen retains all rights for the Servier-partnered programs in the U.S and Japan.

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Safe Harbor Statement
This press release contains “forward-looking statements” for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements regarding the role of microRNAs in disease processes and as potential drug products, the potential for MRG-106, MRG-107 and MRG-201 to target diseases, the adequacy of the Company’s capital to support its future operations and the Company’s ability to successfully initiate and complete clinical trials. Such statements are based on management’s current expectations and involve risks and uncertainties. Actual results and performance could differ materially from those projected in the forward-looking statements as a result of many factors, including, without limitation, results of earlier studies and trials may not be predictive of future clinical trial results, the protection and market exclusivity provided by the Company’s intellectual property; risks related to the drug discovery and the regulatory approval process, the risks and uncertainties associated with: the Company’s financial resources and whether they will be sufficient to meet the Company’s business objectives and operational requirements; and, the impact of competitive products and technological changes. The Company disclaims any intent or obligation to update these forward-looking statements.

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