NOVEL RNA THERAPEUTICS AND USES THEREOF

The present invention relates to novel therapeutic compounds, known as RNAi agents, that decrease expression of the ANGPTL8 receptor (expressed by the ANGPTL8 gene), thereby decreasing expression of mRNA and protein expression. Such RNAi agents are useful in the treatment of diseases involving the r...

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Hauptverfasser: BEYER, Thomas Patrick, CHENG, Christine Chih-Tao, EGGEN, Marijean, WILSON, Takako, ANTONELLIS, Patrick Joseph, MILES, Rebecca Ruth, BAWEL, Seth Andrew, WANG, Jibo, LACKNER, Gregory Lawrence
Format: Patent
Sprache:eng ; fre
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Zusammenfassung:The present invention relates to novel therapeutic compounds, known as RNAi agents, that decrease expression of the ANGPTL8 receptor (expressed by the ANGPTL8 gene), thereby decreasing expression of mRNA and protein expression. Such RNAi agents are useful in the treatment of diseases involving the regulation of ANGPTL8 expression and function, such as dyslipidemia, a cardiovascular disorder, or a cardiometabolic disorder. La présente invention concerne de nouveaux composés thérapeutiques, connus sous le nom d'agents ARNi, qui diminuent l'expression du récepteur ANGPTL8 (exprimé par le gène ANGPTL8), diminuant ainsi l'expression de l'ARNm et l'expression protéique. De tels agents ARNi sont utiles dans le traitement de maladies impliquant la régulation de l'expression et de la fonction d'ANGPTL8, telles que la dyslipidémie, le trouble cardiovasculaire ou le trouble cardiométabolique.