Pyrazolopyridine derivatives and uses thereof

The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing the expression levels of wide-spaced zinc finger motif (WIZ), or inducing the expression of fetal hemoglobin (HbF), and in the treatment of hereditary hematological diseases (e.g., he...

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Hauptverfasser: JAIN RAJU, BONAZZI SIMONE, MANNING JAMES R, DYLES NATHAN A, TING, PATRICK, Y, MALIK HASNAIN AHMED, COBB, JEFFREY, S, THOMSEN NOEL MARIE-FRANCE, DEWHURST JAMES, PATTERSON ANDREW W, KERRIGAN JOHN R, O 'BRIEN GARY, CERNIJENKO ARKADIY, HUSSEY MICHAEL J
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing the expression levels of wide-spaced zinc finger motif (WIZ), or inducing the expression of fetal hemoglobin (HbF), and in the treatment of hereditary hematological diseases (e.g., hemoglobinopathy, e.g., beta-hemoglobinopathy), e.g., sickle cell disease and beta-thalassemia. 本披露内容涉及具有式(I)的化合物和药物组合物以及它们在降低宽间隔的锌指基序(WIZ)表达水平、或诱导胎儿血红蛋白(HbF)表达、以及在治疗遗传性血液病(例如血红蛋白病,例如β-血红蛋白病),例如镰状细胞病和β-地中海贫血中的用途。