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51. 发明授权

US6153737A Derivatized oligonucleotides having improved uptake and other properties 失效
标题翻译：具有改善摄取和其他性质的衍生寡核苷酸
公开(公告)号：US6153737A
公开(公告)日：2000-11-28
申请号：US211882
申请日：1994-04-22
申请人： Muthiah Manoharan , Phillip Dan Cook , Clarence Frank Bennett
发明人： Muthiah Manoharan , Phillip Dan Cook , Clarence Frank Bennett
IPC分类号： A01N43/04 , C07H19/00 , C07H21/00 , C12Q1/68
CPC分类号： A01N37/46 , C07H19/00 , C07H21/00
摘要： Linked nucleosides having at least one functionalized nucleoside that bears a substituent such as a steroid molecule, a reporter molecule, a non-aromatic lipophilic molecule, a reporter enzyme, a peptide, a protein, a water soluble vitamin, a lipid soluble vitamin, an RNA cleaving complex, a metal chelator, a porphyrin, an alkylator, a pyrene, a hybrid photonuclease/intercalator, or an aryl azide photo-crosslinking agent exhibit increased cellular uptake and other properties. The substituent can be attached at the 2'-position of the functionalized nucleoside via a linking group. If at least a portion of the remaining liked nucleosides are 2'-deoxy-2'-fluoro, 2'-O-methoxy, 2'-O-ethoxy, 2'-O-propoxy, 2'-O-aminoalkoxy or 2'-O-allyloxy nucleosides, the substituent can be attached via a linking group at any of the 3' or the 5' positions of the nucleoside or on the heterocyclic base of the nucleoside or on the inter-nucleotide linkage linking the nucleoside to an adjacent nucleoside.
摘要翻译： PCT No.PCT / US92 / 09196 Sec。 371日期：1992年4月22日 102（e）日期1992年4月22日PCT 1992年10月23日提交了具有至少一个具有取代基的官能化核苷的连接核苷，例如类固醇分子，报道分子，非芳族亲油性分子，报道酶，肽，蛋白质，水溶性维生素，脂溶性维生素，RNA切割复合物，金属螯合剂，卟啉，烷基化剂，芘，混合光子核酸酶/嵌入剂或芳基叠氮化物光交联剂表现出增加的细胞吸收等性质。取代基可以通过连接基团连接在官能化核苷的2'-位上。如果至少一部分剩余的核苷类为2'-脱氧-2'-氟，则2'-O-甲氧基，2'-O-乙氧基，2'-O-丙氧基，2'-O-氨基烷氧基或2 '-O-烯丙氧基核苷，取代基可以通过连接基团在核苷的3'或5'位置或核苷的杂环碱基上或连接核苷与核苷酸之间的核苷酸间连接相邻核苷。

52. 发明授权

US5681941A Substituted purines and oligonucleotide cross-linking 失效
标题翻译：取代嘌呤和寡核苷酸交联
公开(公告)号：US5681941A
公开(公告)日：1997-10-28
申请号：US189792
申请日：1994-02-01
申请人： Phillip Dan Cook , Muthiah Manoharan , Kanda S. Ramasamy
发明人： Phillip Dan Cook , Muthiah Manoharan , Kanda S. Ramasamy
IPC分类号： A61K31/70 , A01N25/02 , A01N43/52 , A61K31/38 , A61K31/4178 , A61K31/7042 , A61K31/7052 , A61K31/7056 , A61K31/706 , A61K31/7064 , A61K31/7068 , A61K31/7072 , A61K31/7076 , A61K31/708 , A61K31/7088 , A61K38/00 , A61K48/00 , A61P31/04 , A61P31/12 , A61P43/00 , C07D409/12 , C07D473/04 , C07D473/16 , C07D473/40 , C07D487/04 , C07H19/00 , C07H19/04 , C07H19/052 , C07H19/056 , C07H19/06 , C07H19/10 , C07H19/12 , C07H19/14 , C07H19/16 , C07H19/167 , C07H19/173 , C07H19/19 , C07H19/20 , C07H19/23 , C07H21/00 , C07H21/02 , C07H21/04 , C07H23/00 , C07J43/00 , C07K14/16 , C12N9/02 , C12N15/09 , C12N15/113 , C12P21/00 , C12Q1/68 , C12Q1/70
CPC分类号： C07H19/16 , A61K48/00 , C07H19/04 , C07H19/06 , C07H19/10 , C07H19/20 , C07H21/00 , C07H23/00 , C07J43/003 , C07K14/005 , C12N15/113 , C12N15/1137 , C12N9/0069 , C12Q1/68 , C12Q1/6813 , C12Y113/11012 , A61K38/00 , C12N2310/3125 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/33 , C12N2310/333 , C12N2310/336 , C12N2310/3511 , C12N2310/3521 , C12N2310/3523 , C12N2310/3527 , C12N2310/3531 , C12N2310/3533 , C12N2740/16322
摘要： This invention is directed to novel purine-based compounds for inclusion into oligonucleotides. The compounds of the invention, when incorporated into oligonucleotides are especially useful as "antisense" agents--agents that are capable of specific hybridization with a nucleotide sequence of an RNA. The compounds of the invention may also be used for cross-linking oligonucleotides. Oligonucleotides are used for a variety of therapeutic and diagnostic purposes, such as treating diseases, regulating gene expression in experimental systems, assaying for RNA and for RNA products through the employment of antisense interactions with such RNA, diagnosing diseases, modulating the production of proteins, and cleaving RNA in site specific fashions. The compounds of the invention include novel heterocyclic bases, nucleosides, and nucleotides. When incorporated into oligonucleotides, the compounds of the invention can be useful for modulating the activity of RNA.
摘要翻译：本发明涉及用于包含在寡核苷酸中的新型基于嘌呤的化合物。当掺入寡核苷酸时，本发明的化合物特别可用作能够与RNA的核苷酸序列特异性杂交的“反义”试剂。本发明的化合物也可用于交联寡核苷酸。寡核苷酸用于各种治疗和诊断目的，例如通过使用与这种RNA的反义相互作用来治疗疾病，调节实验系统中的基因表达，测定RNA和RNA产物，诊断疾病，调节蛋白质的产生，并在现场具体时尚中切割RNA。本发明的化合物包括新的杂环碱基，核苷和核苷酸。当掺入寡核苷酸时，本发明的化合物可用于调节RNA的活性。

53. 发明授权

US09186325B2 Lipids and compositions for the delivery of therapeutics 有权
标题翻译：用于递送治疗剂的脂质和组合物
公开(公告)号：US09186325B2
公开(公告)日：2015-11-17
申请号：US13128283
申请日：2009-11-10
申请人： Muthiah Manoharan , Muthusamy Jayaraman , Kallanthottathil G. Rajeev , Laxman Eltepu , Steven Ansell , Jianxin Chen
发明人： Muthiah Manoharan , Muthusamy Jayaraman , Kallanthottathil G. Rajeev , Laxman Eltepu , Steven Ansell , Jianxin Chen
IPC分类号： A61K39/12 , A61K9/127 , A61K39/00 , C12N15/11
CPC分类号： A61K47/44 , A61K9/1272 , A61K31/7088 , A61K31/713 , A61K39/00 , A61K39/39 , A61K47/10 , A61K47/18 , A61K47/20 , A61K47/28 , A61K2039/55555 , A61K2039/55561 , C07C229/08 , C07C229/30 , C07C237/16 , C07C251/38 , C07C251/78 , C07C271/12 , C07C271/20 , C07C323/25 , C07D203/10 , C07D317/28 , C07D317/44 , C07D317/46 , C07D317/72 , C07D319/06 , C07D405/12 , C07D491/056 , C07D491/113 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/3515 , C12N2320/32 , Y02A50/464
摘要： The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention formula (I) provides lipids having the following structure XXXIII wherein: R1 and R2 are each independently for each occurrence optionally substituted C10-C30 alkyl, optionally substituted C10-C30 alkenyl, optionally substituted C10-C30 alkynyl, optionally substituted C10-C30 acyl, or -linker-ligand; R3 is H, optionally substituted C1-C10 alkyl, optionally substituted C2-C10 alkenyl, optionally substituted C2-C10 alkynyl, alky lhetro cycle, alkylphosphate, alkylphosphorothioate, alkylphosphorodithioate, alkylphosphonates, alkylamines, hydroxyalkyls, ω-aminoalkyls, ω-(substituted)aminoalkyls, ω-phosphoalkyls, ω-thiophosphoalkyls, optionally substituted polyethylene glycol (PEG, mw 100-40K), optionally substituted mPEG (mw 120-40K), heteroaryl, heterocycle, or linker-ligand; and E is C(O)O or OC(O).
摘要翻译：本发明提供有利地用于脂质颗粒以用于体内递送治疗剂至细胞的脂质。具体地，本发明式（I）提供具有以下结构XXXIII的脂质，其中：R1和R2各自独立地为任何取代的C 10 -C 30烷基，任选取代的C 10 -C 30烯基，任选取代的C 10 -C 30炔基，任选取代的 C10-C30酰基或 - 连接子 - 配体; R 3是H，任选取代的C 1 -C 10烷基，任选取代的C 2 -C 10烯基，任选取代的C 2 -C 10炔基，烷基杂芳基，烷基磷酸酯，烷基硫代磷酸酯，烷基磷酸二烷基酯，烷基膦酸酯，烷基胺，羟基烷基，ω-氨基烷基，ω-（取代的）氨基烷基，ω-磷酸烷基酯，ω-硫代磷酰基烷基，任选取代的聚乙二醇（PEG，mw 100-40K），任选取代的mPEG（mw 120-40K），杂芳基，杂环或连接体 - 配体; 并且E是C（O）O或OC（O）。

54. 发明授权

US08754201B2 Therapeutic compositions 有权
标题翻译：治疗组合物
公开(公告)号：US08754201B2
公开(公告)日：2014-06-17
申请号：US12721413
申请日：2010-03-10
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Bumcrot
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Bumcrot
IPC分类号： C07H21/04 , C12N15/113
CPC分类号： C12N15/113 , A01K67/0275 , A01K2217/05 , A01K2227/105 , A01K2267/0362 , A61K31/713 , A61K47/48123 , C07H21/02 , C07K14/775 , C12N15/111 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/344 , C12N2310/3515 , C12N2310/533 , C12N2320/32 , C12N2320/51 , C12N2320/53 , C12N2330/30
摘要： This application relates to therapeutic siRNA agents and methods of making and using the agents.
摘要翻译：本申请涉及治疗性siRNA剂及其制备和使用方法。

55. 发明授权

US08642076B2 Lipid containing formulations 有权
标题翻译：含脂质配方
公开(公告)号：US08642076B2
公开(公告)日：2014-02-04
申请号：US13211094
申请日：2011-08-16
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Martin Maier
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Akin Akinc , Narayanannair K. Jayaprakash , Muthusamy Jayaraman , Martin Maier
IPC分类号： A61K9/48
CPC分类号： C07C219/06 , A61K9/1272 , A61K9/1273 , A61K31/325 , A61K47/34 , C07C209/78 , C07C233/36 , C07C271/10 , C07C323/25
摘要： Compositions and methods useful in administering nucleic acid based therapies, for example association complexes such as liposomes and lipoplexes are described.
摘要翻译：描述了用于施用基于核酸的疗法，例如缔合复合物如脂质体和脂质复合物的组合物和方法。

56. 发明授权

US08604183B2 Compositions comprising alternating 2′-modified nucleosides for use in gene modulation 有权
公开(公告)号：US08604183B2
公开(公告)日：2013-12-10
申请号：US10701007
申请日：2003-11-04
申请人： Charles Allerson , Balkrishen Bhat , Anne B. Eldrup , Muthiah Manoharan , Richard H. Griffey , Brenda F. Baker , Eric E. Swayze
发明人： Charles Allerson , Balkrishen Bhat , Anne B. Eldrup , Muthiah Manoharan , Richard H. Griffey , Brenda F. Baker , Eric E. Swayze
IPC分类号： C07H21/02 , C07H21/04 , C12Q1/68 , C12P19/34
CPC分类号： C07H21/00 , C07H21/04 , C12N15/111 , C12N15/113 , C12N2310/14 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/343 , C12N2310/3521 , C12N2310/3533 , C12N2320/51
摘要： The present invention provides compositions comprising at least one oligomeric compound comprising an alternating motif and further include a region that is complementary to a nucleic acid target. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

57. 发明申请

US20130323269A1 METHODS AND COMPOSITIONS FOR DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：用于递送活性剂的方法和组合物
公开(公告)号：US20130323269A1
公开(公告)日：2013-12-05
申请号：US13813122
申请日：2011-07-29
申请人： Muthiah Manoharan , Kallanthotthathil G. Rajeev , Muthusamy Jayaraman , Michael E. Jung
发明人： Muthiah Manoharan , Kallanthotthathil G. Rajeev , Muthusamy Jayaraman , Michael E. Jung
IPC分类号： A61K47/14 , A61K31/7088 , A61K31/713
CPC分类号： A61K48/0033 , A61K31/7088 , A61K31/713 , A61K47/14 , A61K47/18 , C07C229/12 , C07C251/38 , C07C323/25 , C07C2601/02 , C07D491/113
摘要： A lipid particle can include a cationic lipid. The cationic lipid can include one or more hydrophobic tails, which can include one or more sites of unsaturation. The sites of unsaturation can include cycloalkyl groups, e.g., cyclopropyl, cyclobutyl, cyclopentyl, or cyclohexyl groups. The lipid particle is suitable for delivering an active agent.
摘要翻译：脂质颗粒可以包括阳离子脂质。阳离子脂质可以包括一个或多个疏水尾部，其可以包括一个或多个不饱和位点。不饱和位的可以包括环烷基，例如环丙基，环丁基，环戊基或环己基。脂质颗粒适用于递送活性剂。

58. 发明授权

US08575123B2 Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components 有权
标题翻译：通过将靶向配体与内体分解组分结合来进行核酸的位点特异性递送
公开(公告)号：US08575123B2
公开(公告)日：2013-11-05
申请号：US12936961
申请日：2009-04-10
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Butler , Muthusamy Jayaraman
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , David Butler , Muthusamy Jayaraman
IPC分类号： A61K48/00 , C12N15/11 , C12N15/00 , A01N63/00 , A01N65/00
CPC分类号： A61K47/554 , A61K47/545 , A61K47/549 , A61K47/551 , A61K48/0008 , C12N15/111 , C12N15/1137 , C12N15/87 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2310/3513 , C12N2320/32 , C12Y304/21021
摘要： The invention relates to compositions and methods for site-specific delivery of nucleic acids by combining them with targeting ligands and endosomolytic components.
摘要翻译：本发明涉及通过将它们与靶向配体和内体分解组分组合而用于位点特异性递送核酸的组合物和方法。

59. 发明授权

US08507455B2 Folate conjugates 有权
标题翻译：叶酸共轭物
公开(公告)号：US08507455B2
公开(公告)日：2013-08-13
申请号：US12328537
申请日：2008-12-04
申请人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Muthusamy Jayaraman , Jayaprakash K. Narayanannair
发明人： Muthiah Manoharan , Kallanthottathil G. Rajeev , Muthusamy Jayaraman , Jayaprakash K. Narayanannair
IPC分类号： C12N15/11 , A61K48/00 , C07H21/02 , C07H21/04
CPC分类号： C12N15/113 , A61K47/55
摘要： The present invention provides iRNA agent including at least one monomer having the structure shown in formula (I′) wherein: A and B are each independently for each occurrence O, N(RN) or S; X is H, a protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a polymer, —P(Z′)(Z″)O-L6-Q′-L7-OP(Z′″)(Z′″″)O-oligonucleotide, a nucleotide, or an oligonucleotide; Y is H, a protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, —P(Z′)(Z″)O-nucleoside, —P(Z′)(Z″)O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, —P(Z′)(Z″)O-L6-Q′-L7-OP(Z′″)(Z′″″)O-oligonucleotide, a nucleotide, or an oligonucleotide; R is folate, a folate analog a folate mimic or a folate receptor binding ligand; L6 and L7 are each independently for each occurrence —(CH2)n—, —C(R′)(R″)(CH2)n—, —(CH2)nC(R′)(R″)—, —(CH2CH2O)mCH2CH2—, or —(CH2CH2O)mCH2CH2NH—; Q′ is NH, O, S, CH2, C(O)O, C(O)NH, —NH—CH(Ra)—C(O)—, —C(O)—CH(Ra)—NH—, CO, where Ra is H or amino acid side chain; R′ and R″ are each independently H, CH3, OH, SH, NH2, NH(Alkyl=Me, Et, Pr, isoPr, Bu, Bn) or N(diAlkyl=Me2, Et2, Bn2); Z′, Z″, Z′″ and Z″″ are independently O or S; n represent independently for each occurrence 1-20; and m represent independently for each occurrence 0-50.
摘要翻译：本发明提供包含至少一种具有式（I'）所示结构的单体的iRNA试剂，其中：A和B各自独立地为O，N（RN）或S; X是H，保护基，磷酸基，磷酸二酯基，活化磷酸酯基，活化亚磷酸酯基，亚磷酰胺，固体载体，-P（Z'）（Z“）O-核苷，-P （Z'）（Z“）O-寡核苷酸，脂质，PEG，类固醇，聚合物，-P（Z'）（Z”）O-L6-Q'-L7-OP（Z“ '）（Z“”）O-寡核苷酸，核苷酸或寡核苷酸; Y是H，保护基，磷酸基，磷酸二酯基，活化磷酸酯基，活化亚磷酸酯基，亚磷酰胺，固体载体，-P（Z'）（Z“）O-核苷，-P （Z'）（Z“）（Z”）O-寡核苷酸，脂质，PEG，类固醇，亲脂性，聚合物，-P（Z'）（Z“）O-L6-Q'-L7-OP Z“'）（Z”“）O-寡核苷酸，核苷酸或寡核苷酸; R是叶酸，叶酸类似物叶酸类似物或叶酸受体结合配体; L 6和L 7各自独立地为 - （CH 2）n - ， - C（R'）（R“）（CH 2）n - ， - （CH 2）n C（R'）（R” （CH 2 CH 2 O）mCH 2 CH 2 - 或 - （CH 2 CH 2 O）m CH 2 CH 2 NH-; Q'是NH，O，S，CH2，C（O）O，C（O）NH，-NH-CH（Ra）-C（O） - ， - （O）-CH（Ra），CO，其中Ra为H或氨基酸侧链; R'和R“各自独立地为H，CH 3，OH，SH，NH 2，NH（烷基= Me，Et，Pr，isoPr，Bu，Bn）或N（二烷基= Me 2，Et 2，Bn 2）; Z'，Z“，Z”“和”Z“独立地为O或S; n每次出现1-20，独立地表示1-20; 而m代表每次出现的0-50。

60. 发明申请

US20130165499A1 Methods And Compositions For Prevention Or Treatment Of RSV Infection 有权
标题翻译：方法和组合物预防或治疗RSV感染
公开(公告)号：US20130165499A1
公开(公告)日：2013-06-27
申请号：US13778614
申请日：2013-02-27
申请人： Akshay Vaishnaw , Saraswathy V. Nochur , Sayda Elbashir , Muthiah Manoharan , Rachel Meyers
发明人： Akshay Vaishnaw , Saraswathy V. Nochur , Sayda Elbashir , Muthiah Manoharan , Rachel Meyers
IPC分类号： A61K31/713 , A61M11/00 , A61K31/708
CPC分类号： C12N15/1131 , A61K31/708 , A61K31/713 , A61M11/00 , C12N2310/14
摘要： Methods and compositions are provided for the prevention or treatment of RSV infection in a human. The methods include administering one or more doses of a composition comprising an siRNA. The dose can be formulated for topical or parenteral administration. Topical administration includes administration as a nasal spray, or by inhalation of respirable particles or droplets.
摘要翻译：提供了用于预防或治疗人体RSV感染的方法和组合物。所述方法包括施用一种或多种剂量的包含siRNA的组合物。剂量可以配制用于局部或肠胃外给药。局部给药包括作为鼻喷雾给药，或通过吸入可吸入颗粒或液滴。

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