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21. 发明申请

WO2017096395A1 METHODS OF TREATING BREAST CANCER 审中-公开
标题翻译：治疗乳腺癌的方法
公开(公告)号：WO2017096395A1
公开(公告)日：2017-06-08
申请号：PCT/US2016/064998
申请日：2016-12-05
申请人： IONIS PHARMACEUTICALS, INC. , COLD SPRING HARBOR LABORATORY
发明人： SPECTOR, David, L. , DIERMEIER, Sarah, Daniela , RIGO, Frank , BENNETT, C., Frank , FREIER, Susan, M. , ARUN, Gayatri , CHANG, Kung-chi
IPC分类号： A61K31/7088 , A61P35/00 , C12N15/11 , C12N15/113
CPC分类号： A61K31/713 , A61K45/06 , C12N15/113 , C12N2310/113 , C12N2310/315 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , A61K2300/00 , C12N2310/321 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a MaTAR in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate breast cancer in an individual in need.
摘要翻译：本文提供了用于降低动物中MaTAR表达的方法，化合物和组合物。此类方法，化合物和组合物可用于治疗，预防，延缓或改善有需要的个体中的乳腺癌。

22. 发明申请

WO2016040748A1 COMPOSITIONS AND METHODS FOR DETECTION OF SMN PROTEIN IN A SUBJECT AND TREATMENT OF A SUBJECT 审中-公开
标题翻译：用于检测受试者中的SMN蛋白质和治疗受试者的组合物和方法
公开(公告)号：WO2016040748A1
公开(公告)日：2016-03-17
申请号：PCT/US2015/049598
申请日：2015-09-11
申请人： IONIS PHARMACEUTICALS, INC.
发明人： RIGO, Frank , BISHOP, Katherine, M.
IPC分类号： G01N33/53
CPC分类号： G01N33/6896 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/3341 , C12N2320/32 , C12N2320/35 , G01N33/566 , G01N33/6854 , G01N33/6887 , G01N2333/47 , G01N2800/28 , G01N2800/2878 , G01N2800/52
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy. Also provided are kits for detecting the amount of SMN protein in a sample of cerebrospinal fluid.
摘要翻译：本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。还提供了用于检测脑脊液样品中SMN蛋白的量的试剂盒。

23. 发明申请

WO2022032060A2 COMPOUNDS AND METHODS FOR MODULATING SCN2A 审中-公开
公开(公告)号：WO2022032060A2
公开(公告)日：2022-02-10
申请号：PCT/US2021/044887
申请日：2021-08-06
申请人： IONIS PHARMACEUTICALS, INC.
发明人： JAFAR-NEJAD, Paymaan , BUI, Huynh-Hoa , FREIER, Susan M. , RIGO, Frank
IPC分类号： A61K31/7088 , A61K31/712 , A61K48/00 , A61P25/08 , C12N15/113 , C12N15/11
摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SCN2A RNA in a cell or subject, and in certain instances reducing the amount of SCN2A protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a disease or disorder associated with a voltage-gated sodium channel protein, such as, for example, a Developmental and Epileptic Encephalopathy, an intellectual disability, or an autism spectmm disorder. Such symptoms and hallmarks include, but are not limited to seizures, hypotonia, sensory integration disorders, motor development delays and dysfunctions, intellectual and cognitive dysfunctions, movement and balance dysfunctions, visual dysfunctions, delayed language and speech, gastrointestinal disorders, neurodevelopmental delays, sleep problems, and sudden unexpected death in epilepsy.

24. 发明申请

WO2021174019A1 COMPOUNDS AND METHODS FOR MODULATING SMN2 审中-公开
公开(公告)号：WO2021174019A1
公开(公告)日：2021-09-02
申请号：PCT/US2021/019934
申请日：2021-02-26
申请人： IONIS PHARMACEUTICALS, INC.
发明人： RIGO, Frank , PRAKASH, Thazha P. , LING, Kar Yun Karen , WAN, W. Brad , DRURY, William John, III.
IPC分类号： A61K31/712 , A61K31/7125 , C12N15/113
摘要： Provided are compounds, methods, and pharmaceutical compositions for modulating SMN2 RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disorder. Such symptoms include reduced muscle strength; inability or reduced ability to sit upright, to stand, and/or walk; reduced neuromuscular activity; reduced electrical activity in one or more muscles; reduced respiration; inability or reduced ability to eat, drink, and/or breathe without assistance; loss of weight or reduced weight gain; and/or decreased survival.

25. 发明申请

WO2019126641A2 MODULATION OF FRATAXIN EXPRESSION 审中-公开
公开(公告)号：WO2019126641A2
公开(公告)日：2019-06-27
申请号：PCT/US2018/067087
申请日：2018-12-21
申请人： IONIS PHARMACEUTICALS, INC. , BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
发明人： RIGO, Frank , PRAKASH, Thazha, P. , COREY, David
IPC分类号： A61K48/00
摘要： Certain embodiments are directed to methods and compounds for increasing FXN. Such methods and compounds are useful for increasing expression of FXN in cells and animals.

26. 发明申请

WO2018102745A1 MODULATION OF LNC05 EXPRESSION 审中-公开
公开(公告)号：WO2018102745A1
公开(公告)日：2018-06-07
申请号：PCT/US2017/064306
申请日：2017-12-01
申请人： COLD SPRING HARBOR LABORATORY , IONIS PHARMACEUTICALS, INC.
发明人： YU, Allen, Tingjin , SPECTOR, David, L. , RIGO, Frank , FREIER, Susan, M. , BERMANN, Jan , BERASAIN, Carmen
IPC分类号： C12N15/113 , C12N15/11
摘要： Provided herein are methods, compounds, and compositions for reducing expression of lnc05 in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a cancer in an individual.

27. 发明申请

WO2018057837A1 GENE THERAPY AND TARGETED DELIVERY OF CONJUGATED COMPOUNDS 审中-公开
标题翻译：共轭化合物的基因治疗和靶向输送
公开(公告)号：WO2018057837A1
公开(公告)日：2018-03-29
申请号：PCT/US2017/052868
申请日：2017-09-22
申请人： IONIS PHARMACEUTICALS, INC.
发明人： RIGO, Frank
IPC分类号： A61K35/30 , A61K47/64 , A61K47/62 , C07K14/00 , C12N5/0793
摘要： Provided herein are methods, compounds, and compositions useful for targeted delivery of compounds to non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions are useful, for example, in gene therapy mediated ectopic expression of cell surface receptors and targeted delivery of compounds, such as conjugated oligonucleotides, to the non-native cells ectopically expressing cell surface receptors. Such methods, compounds, and compositions can be useful, for example, to treat, prevent, delay or ameliorate disease in an individual by targeted reduction of a gene of interest in the non-native cell ectopically expressing cell surface receptors.
摘要翻译：本文提供了用于将化合物靶向递送至异位细胞的异位表达细胞表面受体的方法，化合物和组合物。这些方法，化合物和组合物可用于例如基因治疗介导的细胞表面受体的异位表达和化合物（例如偶联的寡核苷酸）向异位细胞异位表达细胞表面受体的靶向递送。这样的方法，化合物和组合物可用于例如通过靶向减少非天然细胞异位表达细胞表面受体中的感兴趣基因来治疗，预防，延缓或改善个体中的疾病。

28. 发明申请

WO2018014041A2 COMPOUNDS AND METHODS FOR MODULATION OF SMN2 审中-公开
标题翻译：用于调节SMN2的化合物和方法
公开(公告)号：WO2018014041A2
公开(公告)日：2018-01-18
申请号：PCT/US2017/042463
申请日：2017-07-17
申请人： IONIS PHARMACEUTICALS, INC.
发明人： RIGO, Frank , PRAKASH, Thazha, P. , SETH, Punit, P.
IPC分类号： A61K48/00 , C07H21/02
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.
摘要翻译：本文公开了用于调节SMN2的剪接的化合物，组合物和方法。还提供了公开的化合物和组合物在制造用于治疗脊髓性肌萎缩症的药物中的用途。

29. 发明申请

WO2017180835A1 METHODS FOR REDUCING C9ORF72 EXPRESSION 审中-公开
标题翻译：减少C9ORF72表达的方法
公开(公告)号：WO2017180835A1
公开(公告)日：2017-10-19
申请号：PCT/US2017/027355
申请日：2017-04-13
申请人： IONIS PHARMACEUTICALS, INC. , LUDWIG INSTITUTE FOR CANCER RESEARCH
发明人： BENNETT, C., Frank , RIGO, Frank , CLEVELAND, Don, W. , JIANG, Jie , ZHU, Qiang , LAGIER-TOURENE, Clotilde
IPC分类号： C12N15/113 , C12N15/11 , C12N15/09
摘要： Provided are methods for reducing the amount or activity of C90RF72 RNA, and in certain instances of reducing the amount of C90RF72 protein, in an animal. Such methods are useful to prevent or ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include anxiety, reduced spatial learning, and memory loss. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).
摘要翻译：提供了用于降低动物中C90RF72RNA的量或活性以及在某些情况下减少C90RF72蛋白质的量的方法。这样的方法可用于预防或改善神经退行性疾病的至少一种症状。这些症状包括焦虑，空间学习减少和记忆丧失。这类神经退行性疾病包括肌萎缩侧索硬化（ALS），额颞叶痴呆（FTD），皮质基底退化综合征（CBD），非典型帕金森氏综合征和橄榄体桥脑样变性（OPCD）。

30. 发明申请

WO2017053781A1 COMPOSITIONS AND METHODS FOR MODULATING ATAXIN 3 EXPRESSION 审中-公开
标题翻译：组合物和方法调节ATAX 3表达
公开(公告)号：WO2017053781A1
公开(公告)日：2017-03-30
申请号：PCT/US2016/053416
申请日：2016-09-23
申请人： IONIS PHARMACEUTICALS, INC. , ACADEMISCH ZIEKENHUIS LEIDEN H.O.D.N. LUMC
发明人： RIGO, Frank , PRAKASH, Thazha, P. , VAN ROON-MOM, Wilhelmina Maria, Clasina , TOONEN, Lodewijk Julius, Anton
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/3525
摘要： Disclosed are methods for modulating splicing of Ataxin 3 mRNA in an animal with modified oligonucleotides. Such compounds and methods are useful to treat, prevent, or ameliorate spinocerebellar ataxia type 3 (SCA3) in an individual in need thereof.
摘要翻译：公开了用修饰的寡核苷酸调节动物中Ataxin 3 mRNA的剪接的方法。这种化合物和方法可用于治疗，预防或改善有需要的个体的脊髓小脑性共济失调3型（SCA3）。

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