[EN] 3-(5-METHOXY-1-OXOISOINDOLIN-2-YL)PIPERIDINE-2,6-DIONE DERIVATIVES AND USES THEREOF<br/>[FR] DÉRIVÉS DE 3-(5-MÉTHOXY-1-OXOISOINDOLIN-2-YL)PIPÉRIDINE-2,6-DIONE ET LEURS UTILISATIONS
申请人:NOVARTIS AG
公开号:WO2021124172A1
公开(公告)日:2021-06-24
The present disclosure relates to compounds of formula (I') and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.
本公开涉及式(I')的化合物、药物组合物及其在降低广泛间隔锌指蛋白结构域(WIZ)表达水平、诱导胎儿血红蛋白(HbF)表达以及治疗遗传性血液疾病(例如,血红蛋白病,例如,β-血红蛋白病)方面的用途,如镰状细胞病和β-地中海贫血。