Rumi Scientific Announces Data Demonstrating Disease Modifying Effect of BRD9 Inhibition in a Huntington’s Disease Animal Model

2023-04-26

临床研究

NEW YORK, April 26, 2023 (GLOBE NEWSWIRE) -- Rumi Scientific (“Rumi”), a developer of therapies for rare and neurodegenerative diseases discovered using its high throughput organoid phenotypic screening platform, announced data demonstrating in vivo proof of concept of a disease modifying effect of bromodomain protein 9 (BRD9) inhibition as a potential treatment for Huntington’s disease (HD). The research was presented at the CHDI Huntington’s Disease Therapeutics Conference, being held, April 24-27, in Dubrovnik, Croatia. “BRD9 inhibition is a novel target for the treatment of HD identified by our proprietary high throughput organoid phenotypic screening platform,” said Fred Etoc, Chief Scientific Officer of Rumi. “We have previously demonstrated, in vitro, that BRD9 inhibition rescues several of the biochemical dysfunctions known to occur in HD and thus could be disease modifying. The new in vivo data reported in this presentation provide both behavioral and molecular validation of this target. We are currently working on developing a brain penetrant BRD9 inhibitor that can ultimately enter IND enabling studies. Finally, these experiments demonstrate the usefulness of RUMI’s platform to discover potential new targets in a therapeutic disease area such as HD where new hypotheses are needed.” To demonstrate in vivo proof of concept, the researchers employed a long-lasting siRNA to block BRD9 in R6/2 mice, an animal model for HD, then evaluated the mice versus control animals on several behavioral and biochemical parameters. Compared to control R6/2 mice, treated mice showed a statistically significant reduction in mutant huntingtin (mHTT) protein aggregations in both the cerebral cortex and dorsal striatum. Aggregation of mHTT in neurons is a molecular hallmark of HD and a marker for disease progression. Additionally, two validated HD behavioral assays were evaluated: distance traveled and rearing behavior. Treated mice demonstrated a significantly increased total distance traveled at eight weeks post treatment compared to untreated mice. Rearing behavior also showed a statistically significant increase in total rearing at 10 and 12 weeks of age compared to control mice. Both measures are behavioral biomarkers for improved mobility, which is rapidly impaired in this mouse model. These data demonstrate the potential of a novel disease modifying therapeutic target to reverse pathologies in a HD animal model. Title: Deep learning analysis of micropattern-based organoid screens reveals BRD9 as a new potential target for Huntington’s disease Authors Shaun Peterson, Tomomi Haremaki, Yudelca Ogando, Carlota Pereda, Arjun Adhikari, Ali Brivanlou, Fred Etoc Time/date 1-4 p.m. CEST, April 26 About Rumi Scientific Rumi Scientific’s mission is to identify and develop novel therapeutics for rare and neurodegenerative diseases by employing its revolutionary synthetic human tissue platform to produce more predictive data leading to a safe and faster clinical trial process. The Company’s lead program is an orally available bromodomain-containing protein 9 (BRD9) inhibitor bromodomain-containing protein 9 (BRD9) inhibitor in lead optimization for the treatment of Huntington’s disease. Founded in 2016, Rumi licensed foundational technology from The Rockefeller University developed by co-founders Ali H. Brivanlou, Ph.D., and Eric D. Siggia, Ph.D. For more information on Rumi Scientific, please see or contact info@rumiscientific.com. Rumi Scientific: Allen Fienberg, Ph.D. Chairman and Chief Executive Officer allen@rumiscientific.com Fred Etoc, Ph.D. Chief Scientific Officer fred@rumiscientific.com Investors: Burns McClellan Lee Roth / Cameron Radinovic lroth@burnsmc.com / cradinovic@burnsmc.com

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