Download RaNA Awarded First Patent for Transcriptional Activation Platform

 FOR IMMEDIATE RELEASE RaNA Awarded First Patent for Transcriptional Activation Platform -­‐-­‐ RaNA’s scientific founder Jeannie T. Lee, M.D., Ph.D., to receive Lurie Prize for foundational research behind PRC2 mechanism -­‐-­‐ Cambridge, Mass. – May 3, 2016 – RaNA Therapeutics, a leader in the discovery and development of a new class of medicines that target RNA, today announced that the United States Patent and Trademark office has issued the first patent covering RaNA’s transcriptional activation platform, U.S. Patent No. 9,328,346, titled “Polycomb-­‐Associated Non-­‐Coding RNAs.” This patent relates broadly to blocking the interaction of long non-­‐coding RNA (lncRNA) with recruiting complexes known as chromatin modifiers (PRC2), which can suppress the expression of certain genes. Separately, Jeannie Lee, M.D., Ph.D., a RaNA founder and scientific advisor, is being recognized for her foundational research behind this same mechanism with the Lurie Prize in Biomedical Sciences by the Foundation for the National Institutes of Health (FNIH). Dr. Lee’s work in uncovering the functions of long non-­‐coding RNA (lncRNA) and X-­‐inactivation has accelerated the understanding of mechanisms driving epigenetic regulation, which involves changes in gene function without changing the DNA sequence. By blocking lncRNA, RaNA’s transcriptional activation platform is capable of selectively upregulating individual genes to increase protein levels for therapeutic benefit. This technology is the basis of RaNA’s programs in spinal muscular atrophy, CNS and liver diseases, and the company has an exclusive license to this patent from Massachusetts General Hospital. “This patent is the first to issue of the more than 150 pending applications in our IP portfolio covering our novel transcriptional and post-­‐transcriptional activation platform,” says Ronald Renaud, CEO of RaNA Therapeutics. “We were also excited to learn Dr. Lee will be honored with a Lurie Prize for her important work, which undoubtedly will have a profound impact on the lives of patients as we leverage this platform to develop medicines that will treat a vast number of hard-­‐to-­‐treat diseases with significant need.” Dr. Lee is the fourth recipient of the Lurie Prize in Biomedical Sciences, which recognizes outstanding achievement by a promising young scientist in biomedical research. Previous recipients include Karl Deisseroth, M.D., Ph.D. (2015); Jennifer Doudna, Ph.D. (2014); and Ruslan M. Medzhitov, Ph.D. (2013). The award will be presented to Dr. Lee on May 18 in Washington D.C. “I am truly honored to receive the 2016 Lurie Prize in Biomedical Sciences, and I hope that we will soon be able to translate these discoveries into treatments for a wide range of diseases,” says Dr. Lee. “With the news of RaNA’s first patent and this award, I am thrilled that our work is continuing to progress in a meaningful way.” For more information about the Lurie Prize in Biomedical Sciences, please visit http://fnih.org/lurieprize. About RaNA Therapeutics RaNA Therapeutics is a leading biotechnology company committed to the development of next generation RNA-­‐targeted medicines that selectively upregulate gene expression to increase endogenous protein levels for therapeutic benefit. The company was founded by preeminent global leaders who pioneered the relationship between lncRNA and chromatic modifiers, as well as breakthrough oligonucleotide technologies. RaNA’s mission is to improve the lives of people suffering from serious life-­‐altering diseases by creating precision medicines that can change the course of their condition. RaNA’s technology has broad therapeutic potential to treat a wide range of diseases, including rare genetic disorders. The company has current lead programs in spinal muscular atrophy and Friedreich’s ataxia. For more information about the company and its platforms, please visit www.ranarx.com. Media Contact Rachel Hutman Pure Communications, Inc. (301) 801-­‐5540 [email protected]