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1. 发明申请

WO2002094185A2 CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY 审中-公开
标题翻译：细胞传递的结合物和组合物
公开(公告)号：WO2002094185A2
公开(公告)日：2002-11-28
申请号：PCT/US2002/015876
申请日：2002-05-20
申请人： RIBOZYME PHARMACEUTICALS, INC , BEIGELMAN, Leonid , MATULIC-ADAMIC, Jasenka , VARGEESE, Chandra , KARPEISKY, Alexander , BLATT, Lawrence , SHAFFER, Christopher
发明人： BEIGELMAN, Leonid , MATULIC-ADAMIC, Jasenka , VARGEESE, Chandra , KARPEISKY, Alexander , BLATT, Lawrence , SHAFFER, Christopher
IPC分类号： A61K
CPC分类号： B82Y5/00 , A61K47/544 , A61K47/549 , A61K47/551 , A61K47/60 , A61K47/66
摘要： This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HAS) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers.
摘要翻译：本发明涉及生物活性化合物的缀合物，可降解接头，组合物，合成方法及其应用，包括叶酸，半乳糖，半乳糖胺，N-乙酰半乳糖胺，PEG，磷脂，肽和人血清白蛋白（HAS）衍生的缀合物，包括抗体，抗病毒剂，化学治疗剂，肽，蛋白质，激素核苷，核苷酸，非核苷和核酸，包括酶核酸，DNA酶，异构酶，反义，dsRNA，siRNA，三重寡核苷酸，2,5-A嵌合体，诱饵和适体。

2. 发明申请

WO2002094185A3 CONJUGATES AND COMPOSITIONS FOR CELLULAR DELIVERY 审中-公开
公开(公告)号：WO2002094185A3
公开(公告)日：2002-11-28
申请号：PCT/US2002/015876
申请日：2002-05-17
申请人： SIRNA THERAPEUTICS, INC. , BEIGELMAN, Leonid , MATULIC-ADAMIC, Jasenka , VARGEESE, Chandra , KARPEISKY, Alexander , BLATT, Lawrence , SHAFFER, Christopher
发明人： BEIGELMAN, Leonid , MATULIC-ADAMIC, Jasenka , VARGEESE, Chandra , KARPEISKY, Alexander , BLATT, Lawrence , SHAFFER, Christopher
IPC分类号： A61K9/14 , C08G63/08
摘要： This invention features conjugates, degradable linkers, compositions, methods of synthesis, and applications thereof, including folate, galactose, galactosamine, N-acetyl galactosamine, PEG, phospholipid, peptide and human serum albumin (HAS) derived conjugates of biologically active compounds, including antibodies, antivirals, chemotherapeutics, peptides, proteins, hormones nucleosides, nucleotides, non-nucleosides, and nucleic acids including enzymatic nucleic acids, DNAzymes, allozymes, antisense, dsRNA, siRNA, triplex oligonucleotides, 2,5-A chimeras, decoys and aptamers.

3. 发明申请

WO2006076674A2 DEPROTECTION AND PURIFICATION OF OLIGONUCLEOTIDES AND THEIR DERIVATIVES 审中-公开
标题翻译：寡核苷酸及其衍生物的去保护和纯化
公开(公告)号：WO2006076674A2
公开(公告)日：2006-07-20
申请号：PCT/US2006/001420
申请日：2006-01-12
申请人： SIRNA THERAPEUTICS, INC. , VARGEESE, Chandra , SHAFFER, Christopher
发明人： VARGEESE, Chandra , SHAFFER, Christopher
IPC分类号： C07H21/00 , C12N15/10
CPC分类号： C07H21/04 , C07H21/00 , C12N15/101
摘要： Method for synthesis, deprotection, and/or purification of nucleic acid molecules, such as oligonucleotides comprising one or more ribonucleotides. Such nucleic acid molecules include siRNA, dsRNA, ribozymes, antisense, and aptamers.
摘要翻译：核酸分子的合成，去保护和/或纯化的方法，例如包含一个或多个核糖核苷酸的寡核苷酸。这样的核酸分子包括siRNA，dsRNA，核酶，反义和适体。

4. 发明申请

WO2004007748A3 DEPROTECTION AND PURIFICATION OF OLIGONUCLEOTIDES AND THEIR DERIVATIVES 审中-公开
公开(公告)号：WO2004007748A3
公开(公告)日：2004-01-22
申请号：PCT/US2003/021775
申请日：2003-07-14
申请人： SIRNA THERAPEUTICS, INC. , VARGEESE, Chandra , SHAFFER, Christopher , BOWMAN, Keith
发明人： VARGEESE, Chandra , SHAFFER, Christopher , BOWMAN, Keith
IPC分类号： C07H1/02
摘要： Method for synthesis, deprotection, and/or purification of nucleic acid molecules, such as oligonucleotides comprising one or more ribonucleotides. Such nucleic acid molecules include siRNA, dsRNA, ribozymes, antisense, and aptamers.

5. 发明申请

WO2023076352A2 OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开
公开(公告)号：WO2023076352A2
公开(公告)日：2023-05-04
申请号：PCT/US2022/047846
申请日：2022-10-26
申请人： WAVE LIFE SCIENCES LTD. , BYRNE, Michael John , VATHIPADIEKAL, Vinod , IWAMOTO, Naoki , VARGEESE, Chandra , GUO, Lankai , HOSS, Andrew Guzior
发明人： BYRNE, Michael John , VATHIPADIEKAL, Vinod , IWAMOTO, Naoki , VARGEESE, Chandra , GUO, Lankai , HOSS, Andrew Guzior
IPC分类号： C12N15/85 , A61K31/712
摘要： Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and/or patterns thereof, and have improved properties, activities and/or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and/or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g., retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

6. 发明申请

WO2019075357A1 OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF 审中-公开
公开(公告)号：WO2019075357A1
公开(公告)日：2019-04-18
申请号：PCT/US2018/055653
申请日：2018-10-12
申请人： WAVE LIFE SCIENCES LTD. , ZHANG, Jason Jingxin , SHIVALILA, Chikdu Shakti , VARGEESE, Chandra , IWAMOTO, Naoki
发明人： ZHANG, Jason Jingxin , SHIVALILA, Chikdu Shakti , VARGEESE, Chandra , IWAMOTO, Naoki
IPC分类号： A61K31/711 , A61K31/712 , A61K31/7125 , C12N15/113 , C12Q1/68
摘要： Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).

7. 发明申请

WO2019055951A1 TECHNOLOGIES FOR OLIGONUCLEOTIDE PREPARATION 审中-公开
公开(公告)号：WO2019055951A1
公开(公告)日：2019-03-21
申请号：PCT/US2018/051398
申请日：2018-09-17
申请人： WAVE LIFE SCIENCES LTD. , BOWMAN, Keith Andrew , VARGEESE, Chandra , BUTLER, David Charles Donnell , KANDASAMY, Pachamuthu , ALAM, Mohammed Rowshon , SHIMIZU, Mamoru , STANDLEY, Stephany Michelle , ADUDA, Vincent , BOMMINENI, Gopal Reddy , TRIPATHI, Snehlata , KORBOUKH, Ilia
发明人： BOWMAN, Keith Andrew , VARGEESE, Chandra , BUTLER, David Charles Donnell , KANDASAMY, Pachamuthu , ALAM, Mohammed Rowshon , SHIMIZU, Mamoru , STANDLEY, Stephany Michelle , ADUDA, Vincent , BOMMINENI, Gopal Reddy , TRIPATHI, Snehlata , KORBOUKH, Ilia
IPC分类号： C07C317/28 , C07H21/02 , C12N15/11 , C12N15/113 , C12Q1/68
摘要： Among other things, the present disclosure provides technologies for oligonucleotide preparation, particularly chirally controlled oligonucleotide preparation, which technologies provide greatly improved crude purity and yield, and significantly reduce manufacturing costs.

8. 发明申请

WO2017192679A1 METHODS AND COMPOSITIONS OF BIOLOGICALLY ACTIVE AGENTS 审中-公开
标题翻译：生物活性剂的方法和组合物
公开(公告)号：WO2017192679A1
公开(公告)日：2017-11-09
申请号：PCT/US2017/030777
申请日：2017-05-03
申请人： WAVE LIFE SCIENCES LTD. , VARGEESE, Chandra , ZHANG, Jason Jingxin , DIVAKARAMENON, Sethumadhavan , BUTLER, David Charles Donnell , LU, Genliang , IWAMOTO, Naoki , YANG, Hailin , FRANK-KAMENETSKY, Maria David , MARAPPAN, Subramanian
发明人： VARGEESE, Chandra , ZHANG, Jason Jingxin , DIVAKARAMENON, Sethumadhavan , BUTLER, David Charles Donnell , LU, Genliang , IWAMOTO, Naoki , YANG, Hailin , FRANK-KAMENETSKY, Maria David , MARAPPAN, Subramanian
IPC分类号： C12N15/113 , C12Q1/68 , C12N15/11
摘要： In some embodiments, the present disclosure pertains to compositions and methods related to delivery of a biologically active agent, wherein the compositions comprise a biologically active agent and a lipid. In various embodiments, the lipid is selected from: lauric acid, myristic acid, palmitic acid, stearic acid, oleic acid, linoleic acid, alpha-linolenic acid, gamma-linolenic acid, docosahexaenoic acid (cis-DHA), turbinaric acid and dilinoleyl. In some embodiments, a composition and method are useful for delivery of a biologically active agent to a particular cell or tissue, e.g., a muscle cell or tissue.
摘要翻译：在一些实施方案中，本公开涉及与递送生物活性剂有关的组合物和方法，其中所述组合物包含生物活性剂和脂质。在各种实施方案中，脂质选自：月桂酸，肉豆蔻酸，棕榈酸，硬脂酸，油酸，亚油酸，α-亚麻酸，γ-亚麻酸，二十二碳六烯酸（顺式-DHA），二十二碳烯酸和二亚油基。在一些实施方案中，组合物和方法可用于将生物活性剂递送至特定细胞或组织，例如肌肉细胞或组织。

9. 发明申请

WO2008137761A3 RNA INTERFERENCE MEDIATED INHIBITION OF CYCLIC NUCLEOTIDE TYPE 4 PHOSPHODIESTERASE (PDE4B) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
公开(公告)号：WO2008137761A3
公开(公告)日：2008-11-13
申请号：PCT/US2008/062530
申请日：2008-05-02
申请人： SIRNA THERAPEUTICS, INC. , STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
发明人： STRAPPS, Walter , JADHAV, Vasant , VARGEESE, Chandra , RICHARDS, Ivan
IPC分类号： C12N15/11 , A61K31/713
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions, including but not limited to IL-6, IL-I, IL-8, IL- 15, TNF-alpha and matrix metalloproteinases (MMPs), such as MMP-I, MMP -2, MMP-3, MMP-9 and MMP- 12. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA), and multifunctional siNA molecules capable of mediating RNA interference (RNAi) against cyclic nucleotide type 4 phosphodiesterase (PDE4B) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, antisense and others that can inhibit the function of endogenous RNA molecules or RNAi pathway components (RNAi inhibitors), such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC) including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism.

10. 发明申请

WO2008011431A2 RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA) 审中-公开
标题翻译：干扰素介导的枯草杆菌蛋白酶KEXIN 9（PCSK9）基因表达的短干扰核酸（SINA）抑制作用
公开(公告)号：WO2008011431A2
公开(公告)日：2008-01-24
申请号：PCT/US2007/073723
申请日：2007-07-17
申请人： SIRNA THERAPEUTICS INC. , MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
发明人： MCSWIGGEN, James , JADHAV, Vasant , VAISH, Narendra, K. , GUERCIOLINI, Roberto , VARGEESE, Chandra
IPC分类号： C12N15/11
CPC分类号： C12N15/1137 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/3521
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression and/or activity. The present invention is also directed to compounds, compositions, and methods relating to traits, diseases and conditions that respond to the modulation of expression and/or activity of genes involved in Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases and conditions. Specifically, the invention relates to double stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro- RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. The present invention also relates to small nucleic acid molecules, such as siNA, siRNA, and others that can inhibit the function of endogenous RNA molecules, such as endogenous micro-RNA (miRNA) (e.g, miRNA inhibitors) or endogenous short interfering RNA (siRNA), (e.g., siRNA inhibitors) or that can inhibit the function of RISC (e.g., RISC inhibitors), to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs (e.g., RISC), including cocktails of such small nucleic acid molecules and lipid nanoparticle (LNP) formulations of such small nucleic acid molecules. Such small nucleic acid molecules and are useful, for example, in providing compositions to prevent, inhibit, or reduce metabolic diseases, traits and conditions, including but not limited to hyperlipidemia, hypercholesterolemia, cardiovascular disease, atherosclerosis, hypertension, diabetis (e.g., type I and/or type II diabetis), insulin resistance, obesity and/or other disease states, conditions, or traits that are associated with gene expression or activity in a subject or organism.
摘要翻译：本发明涉及用于研究，诊断和治疗对Prop蛋白转化酶枯草杆菌蛋白酶9（PCSK9）基因表达的调节作出响应的性状，疾病和病症的化合物，组合物和方法，和 /或活动。本发明还涉及涉及对Prop蛋白转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达途径或其他细胞过程中涉及的基因的表达和/或活性的调节作出响应的性状，疾病和病症的化合物，组合物和方法调解这些特征，疾病和状况的维持或发展。具体而言，本发明涉及双链核酸分子，包括小核酸分子，如短干扰核酸（siNA），短干扰RNA（siRNA），双链RNA（dsRNA），微RNA（miRNA）和能够介导针对Proprotein转化酶枯草杆菌蛋白酶Kexin 9（PCSK9）基因表达的RNA干扰（RNAi）的短发夹RNA（shRNA）分子，包括此类小核酸分子的混合物和此类小核酸分子的脂质纳米颗粒（LNP）制剂。本发明还涉及可以抑制内源RNA分子（例如内源性微RNA（miRNA）（例如，miRNA抑制剂）或内源性短干扰RNA（例如，miRNA））的功能的小核酸分子，例如siNA，siRNA， siRNA）（例如siRNA抑制剂）或可以抑制RISC功能（例如RISC抑制剂），通过干扰与内源RNA（例如RISC）相关的内源RNA或蛋白质的调节功能来调节基因表达，，包括这种小核酸分子的鸡尾酒和这种小核酸分子的脂质纳米颗粒（LNP）制剂。这样的小核酸分子可用于例如提供组合物以预防，抑制或减少代谢疾病，性状和病症，包括但不限于高脂血症，高胆固醇血症，心血管疾病，动脉粥样硬化，高血压，糖尿病（例如类型 I型和/或II型糖尿病），胰岛素抵抗，肥胖和/或与受试者或生物体中的基因表达或活性相关的其他疾病状态，病症或性状。

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