[EN] COMPOUNDS, COMPOSITIONS AND METHODS FOR INCREASING CFTR ACTIVITY<br/>[FR] COMPOSÉS, COMPOSITIONS ET MÉTHODES POUR AUGMENTER L'ACTIVITÉ DU CFTR
申请人:PROTEOSTASIS THERAPEUTICS INC
公开号:WO2016115090A1
公开(公告)日:2016-07-21
The present disclosure features compounds such as those having the Formulae (Ila), (lIb), (lIc), (Ild), (IlIa), and (Illb), which can increase cystic fibrosis transmembrane conductance regulator (CFTR) activity as measured in human bronchial epithelial (hBE) cells. The present disclosure also features methods of treating a condition associated with decreased CFTR activity or a condition associated with a dysfunction of proteostasis comprising administering to a subject an effective amount of a disclosed compound, such as a compound of Formula (Ila), (lIb), (lIc), (lId), (IlIa), or (Illb).
本公开涵盖了诸如具有以下式(Ila)、(lIb)、(lIc)、(Ild)、(IlIa)和(Illb)的化合物,这些化合物可以增加在人类支气管上皮细胞(hBE)中测量的囊性纤维化跨膜传导调节蛋白(CFTR)活性。本公开还涵盖了治疗与CFTR活性降低或蛋白质稳态功能障碍相关的疾病的方法,包括向受试者施用所述化合物的有效量,例如式(Ila)、(lIb)、(lIc)、(lId)、(IlIa)或(Illb)的化合物。