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事業概要 Dicerna Pharmaceuticals Inc. is a ribonucleic acid (RNA) interference-based biopharmaceutical company. The Company operates in the segment of discovery research and development of treatments based on its RNA interference (RNAi) technology platform. The Company is focused on the discovery and development of treatments for rare inherited diseases involving the liver and for cancers that are genetically defined. The Company uses its RNAi technology platform to build a pipeline in these therapeutic areas. The Company develops dacryocystorhinostomy (DCR)-Primary Hyperoxaluria Type 1 (PH1) for the treatment of PH1 by targeting the gene encoding the liver enzyme glycolate oxidase. The Company uses its dicer substrate RNA (DsiRNA)-EX Conjugate technology to develop a subcutaneously injected treatment for PH1. The Company develops DCR-MYC for the treatment of MYC-related cancers including hepatocellular carcinoma (HCC) and pancreatic neuroendocrine tumors (PNET). ディサ―ナ・ファ―マシュ―ティカルズは、米国のバイオ医薬品会社。肝臓の遺伝性疾患などの癌のためのRNA干渉技術プラットフォ―ムを使用した治療法の発見と開発に焦点を当てる。同社は、｢DCR-PH1｣と｢DCR-M1711｣製品を開発している。また癌遺伝子キルステンラット肉腫ウイルス癌遺伝子ホモログを対象に開発する。 Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using its proprietary GalXC™ and GalXC-Plus™ RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands on the functionality and application of its flagship liver-based GalXC technology, and has the potential to treat diseases across multiple therapeutic areas. In addition to its own pipeline of core discovery and clinical candidates, Dicerna has established collaborative relationships with some of the world's leading pharmaceutical companies, including Novo Nordisk A/S, Roche, Eli Lilly and Company, Alexion Pharmaceuticals, Inc., Boehringer Ingelheim International GmbH and Alnylam Pharmaceuticals, Inc. Between Dicerna and its collaborative partners, they currently have more than 20 active discovery, preclinical or clinical programs focused on rare, cardiometabolic, viral, chronic liver and complement-mediated diseases, as well as neurodegenerative diseases and pain. Dicerna's mission is to interfere - to silence genes, to fight disease, to restore health.
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DRNA - ディサ―ナ・ファ―マシュ―ティカルズ （Dicerna Pharmaceuticals Inc.） ディサ―ナ・ファ―マシュ―ティカルズ