公开(公告)号：WO2015051135A2

公开(公告)日：2015-04-09

申请号：PCT/US2014/058851

申请日：2014-10-02

申请人： NOVARTIS AG ,  BARYZA, Jeremy Lee ,  BLOMMERS, Marcel ,  BORLAND, Todd ,  FERNANDEZ, Cesar ,  GEBUHR, Tom ,  GENO, Erin ,  GOSSERT, Alvar ,  GREENIDGE, Paulette ,  HUESKEN, Dieter ,  HUNZIKER, Juerg ,  NATT, Francois Jean-Charles ,  PATNAIK, Anup ,  PATTERSON, Andrew ,  RONDEAU, Jean-Michel Rene ,  SOLOMON, Jonathan ,  WEILER, Jan ,  ZHU, Meicheng

发明人： BARYZA, Jeremy Lee ,  BLOMMERS, Marcel ,  BORLAND, Todd ,  FERNANDEZ, Cesar ,  GEBUHR, Tom ,  GENO, Erin ,  GOSSERT, Alvar ,  GREENIDGE, Paulette ,  HUESKEN, Dieter ,  HUNZIKER, Juerg ,  NATT, Francois Jean-Charles ,  PATNAIK, Anup ,  PATTERSON, Andrew ,  RONDEAU, Jean-Michel Rene ,  SOLOMON, Jonathan ,  WEILER, Jan ,  ZHU, Meicheng

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2320/51

摘要： The present disclosure relates to methods of treating Hepcidin-related diseases such as anemia, iron-deficient erythropoiesis, hypoferremia, impaired dietary iron uptake, iron sequestration, anemia of inflammation (AI) (sometimes designated anemia of chronic disease or ACD), atherosclerosis, diabetes, and multiple neurodegenerative disorders such as Alzheimers disease, Parkinsons disease and Friedrichs ataxia, heart failure, chronic kidney disease, cardiorenal-anemia syndrome, infection, blood loss, hemolysis, vitamin B12 or folate deficiency, hyperparathyroidism, hemoglobinopathies and malignancies, cancer, AIDS, surgery, runted growth, and hair loss, using a therapeutically effective amount of a RNAi agent to Hepcidin.

摘要翻译： 本公开涉及治疗肝细胞因子相关疾病如贫血，缺铁性红细胞生成素，低铁血症，膳食铁吸收受损，铁螯合，炎症性贫血（AI）（有时称为慢性疾病或ACD的贫血），动脉粥样硬化， 糖尿病和多种神经变性疾病如阿尔茨海默病，帕金森病和弗里德里希共济失调，心力衰竭，慢性肾脏疾病，心肾贫血综合征，感染，失血，溶血，维生素B12或叶酸缺乏，甲状旁腺功能亢进，血红蛋白病和恶性肿瘤， 艾滋病，手术，发育不良和脱发，使用治疗有效量的RNAi剂对Hepcidin。

公开(公告)号：WO2011005765A1

公开(公告)日：2011-01-13

申请号：PCT/US2010/041072

申请日：2010-07-06

申请人： ALNYLAM PHARMACEUTICALS, INC. ,  ROSSOMANDO, Anthony ,  MARAGANORE, John ,  POLLARD, Stuart ,  KOCISKO, David ,  MANOHARAN, Muthiah ,  BORLAND, Todd ,  HOGAN, Shannon

发明人： ROSSOMANDO, Anthony ,  MARAGANORE, John ,  POLLARD, Stuart ,  KOCISKO, David ,  MANOHARAN, Muthiah ,  BORLAND, Todd ,  HOGAN, Shannon

IPC分类号： C12N5/00 ,  C12N15/11

CPC分类号： C12N7/00 ,  C12N15/113 ,  C12N15/1131 ,  C12N2310/14

摘要： The present invention relates to molecular biology, molecular genetics, and bioprocessing. The embodiments provide for compositions and methods for producing a biological product, such as an immunogenic agent, in an embryonated egg by introducing into the egg a RNA effector molecule capable of modulating expression of a target gene, wherein the modulation enhances production of the biological product in the egg. These methods provide for RNAi-based approaches to optimize the production of biologics from embryonated eggs, such as the production of viral vaccines including seasonal and pandemic flu vaccines. The invention also relates to molecules, reagents, cells, and kits useful for carrying out the methods, and biological products produced by the methods.

摘要翻译： 本发明涉及分子生物学，分子遗传学和生物处理。 这些实施方案提供了通过向蛋中引入能够调节靶基因表达的RNA效应分子来生产在胚胎卵中的生物产物如免疫原性试剂的组合物和方法，其中所述调节增强了生物产物的产生 在蛋里 这些方法提供了基于RNAi的方法来优化来自胚胎卵的生物制品的生产，例如生产包括季节性和大流行性流感疫苗的病毒疫苗。 本发明还涉及可用于实施方法的分子，试剂，细胞和试剂盒，以及通过该方法生产的生物制品。

公开(公告)号：WO2015051135A3

公开(公告)日：2015-09-03

申请号：PCT/US2014058851

申请日：2014-10-02

申请人： NOVARTIS AG ,  BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  BORLAND TODD ,  FERNANDEZ CESAR ,  GEBUHR TOM ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  SOLOMON JONATHAN ,  WEILER JAN ,  ZHU MEICHENG

发明人： BARYZA JEREMY LEE ,  BLOMMERS MARCEL ,  BORLAND TODD ,  FERNANDEZ CESAR ,  GEBUHR TOM ,  GENO ERIN ,  GOSSERT ALVAR ,  GREENIDGE PAULETTE ,  HUESKEN DIETER ,  HUNZIKER JUERG ,  NATT FRANCOIS JEAN-CHARLES ,  PATNAIK ANUP ,  PATTERSON ANDREW ,  RONDEAU JEAN-MICHEL RENE ,  SOLOMON JONATHAN ,  WEILER JAN ,  ZHU MEICHENG

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61P7/06

CPC分类号： C12N15/113 ,  C12N15/1136 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2320/51

摘要： The present disclosure relates to methods of treating Hepcidin-related diseases such as anemia, iron-deficient erythropoiesis, hypoferremia, impaired dietary iron uptake, iron sequestration, anemia of inflammation (AI) (sometimes designated anemia of chronic disease or ACD), atherosclerosis, diabetes, and multiple neurodegenerative disorders such as Alzheimers disease, Parkinsons disease and Friedrichs ataxia, heart failure, chronic kidney disease, cardiorenal-anemia syndrome, infection, blood loss, hemolysis, vitamin B12 or folate deficiency, hyperparathyroidism, hemoglobinopathies and malignancies, cancer, AIDS, surgery, runted growth, and hair loss, using a therapeutically effective amount of a RNAi agent to Hepcidin.

摘要翻译： 本发明涉及治疗铁调素相关疾病例如贫血，铁缺乏性红细胞生成，低铁血症，饮食铁摄取受损，铁螯合，炎症贫血（AI）（有时称为慢性疾病或ACD贫血），动脉粥样硬化， 糖尿病和多种神经退行性疾病如阿尔茨海默病，帕金森病和弗里德里希共济失调，心力衰竭，慢性肾病，心肾综合征，感染，失血，溶血，维生素B12或叶酸缺乏症，甲状旁腺功能亢进症，血红蛋白病和恶性肿瘤， 艾滋病，手术，运行的生长和脱发，使用治疗有效量的RNAi剂对Hepcidin。