miRagen Therapeutics and Collaborators Identify Key Role for miR-133a as a Modulator of Skeletal Muscle Disorder

Data published in The Journal of Clinical Investigation

BOULDER, Colo.--(BUSINESS WIRE)--miRagen Therapeutics, Inc., a biopharmaceutical company focused on improving patients’ lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, announced today that new preclinical data reveal an essential role for miR-133a in the maintenance of adult skeletal muscle structure and function. The study suggests that miR-133a is a modulator of the development of human centronuclear myopathy (CNM), a rare condition affecting skeletal muscles. The research, licensed by miRagen Therapeutics, was conducted by researchers at the University of Texas Southwestern Medical Center and Virginia Polytechnic Institute and State University. Results of the study were published today in the August issue of The Journal of Clinical Investigation.

“The similarities in the skeletal muscle abnormalities found in the miR-133a deficient mice and human CNM patients suggest that miR-133a may play a role in the disease”

Human CNMs are a group of congenital diseases of muscle tissue characterized by muscle weakness and abnormal centralization of nuclei in muscle myofibers. In this study, adult mice lacking miR-133a developed progressive CNM, as well as mitochondrial dysfunction and fast-to-slow myofiber conversion. These findings demonstrate an essential role for miR-133a in the maintenance of adult skeletal muscle structure, function, bioenergetics, and myofiber identity; they also strongly suggest that miR-133a is a potential modulator of CNM.

“The similarities in the skeletal muscle abnormalities found in the miR-133a deficient mice and human CNM patients suggest that miR-133a may play a role in the disease,” said Eric N. Olson, Ph.D., Chief Scientific Advisor and Co-founder of miRagen Therapeutics, Inc. “The findings further underscore the potential of microRNA modulation as a novel therapeutic approach to treat skeletal muscle diseases.”

“We are very excited by the publication of these results that further demonstrate the important role of miR-133a in maintaining normal structure and function of adult skeletal muscle,” said William S. Marshall, Ph.D., President and Chief Executive Officer of miRagen Therapeutics, Inc. “These findings ultimately enhance our commitment to developing innovative microRNA-based therapeutics to treat patients with debilitating muscle diseases.”

About microRNAs: MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Recent studies have demonstrated that microRNAs are associated with many disease processes. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

About miRagen Therapeutics: miRagen Therapeutics, Inc., was founded in 2007 to develop innovative microRNA-based therapeutics for cardiovascular and muscle disease. Only recently discovered, microRNAs are short, single-stranded RNA molecules encoded in the genome that regulate gene expression and play a vital role in influencing cardiovascular and muscle disease. Cardiovascular disease is the leading cause of death globally and represents an enormous burden on global healthcare systems. Principally funded through venture capital investments, miRagen combines world recognized leadership in cardiovascular medicine with unprecedented in-house expertise in microRNA biology and chemistry. For more information, please visit www.miragentherapeutics.com.