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    • 9. 发明申请
    • FATTY ACID CYSTEAMINE CONJUGATES AND THEIR USE AS ACTIVATORS OF AUTOPHAGY
    • 脂肪酸CYPTEAMINE CONJUGATES及其作为自动化的激活剂使用
    • WO2016086103A1
    • 2016-06-02
    • PCT/US2015/062620
    • 2015-11-25
    • CATABASIS PHARMACEUTICALS, INC.
    • VU, Chi, B.JIROUSEK, Michael, R.
    • A61K31/4425A61P29/00A61P11/00A61P21/00
    • A61K31/105A61K31/095A61K31/121A61K31/195A61K31/4406A61K45/06A61K47/48023C07C323/23C07D213/82A61K2300/00
    • The invention relates to (i) 6-membered heteroaryl substituted fatty acid cystamine conjugates, compositions thereof, methods of treating diseases involving dysregulation of autophagy, such as cystic fibrosis, idiopathic pulmonary fibrosis (IPF), a neurodegenerative disease, inflammatory disease, liver disease, muscle disease, infection and immune disease with this compound, or (ii) a method of treating idiopathic pulmonary fibrosis, mitochondrial diseases, Leigh Syndrome, Diabetes Mellitus and Deafness (DAD), Leber's hereditary optic neuropathy, Neuropathy-ataxia-retinis pigmentosa and ptosis (NARP), myoneurogenic gastrointestinal encephalopathy (MNG1E), myoclonic epilepsy with ragged red fibers (MERRF), or mitochondrial myopathy- encephalomyopathy-lactic acidosis stroke like symptoms (MELAS), comprising administering to a patient the fatty acid cysteamine conjugate, (4Z, 7Z. 10Z, 13Z, 16Z, 19Z)-N-(2-mercaptoethyl) docosa-4,7,10,13,16,19-hexaenamide or (5Z,8Z, 11Z, 14Z, 17Z)-N-(2-mercaptoethyl) icosa-5, 8,11,14, 17-pentaenamide.
    • 本发明涉及(i)6-元杂芳基取代的脂肪酸胱氨酸缀合物,其组合物,治疗涉及自噬失调的疾病如囊性纤维化,特发性肺纤维化(IPF),神经变性疾病,炎症性疾病,肝脏疾病的方法 ,(ii)治疗特发性肺纤维化,线粒体疾病,Leigh综合征,糖尿病和耳聋(DAD),Leber遗传性视神经病变,神经病共济失调 - 视网膜色素变性和视网膜色素变性的方法, 上睑下垂症(NARP),肌源性胃肠道脑病(MNG1E),具有粗糙红色纤维的肌阵挛性癫痫(MERRF)或线粒体肌病 - 脑病 - 乳酸性酸中毒症状(MELAS),包括向患者施用脂肪酸半胱胺缀合物(4Z ,7Z,10Z,13Z,16Z,19Z)-N-(2-巯基乙基)二十二碳-4,7,10,13,16,19-六烯酰胺或(5Z,8Z,11Z,14Z,17Z) 2- MERC 二甲苯基)二十碳-5,8,11,14,17-戊烯酰胺。