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1. 发明申请

WO2012016188A2 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：WO2012016188A2
公开(公告)日：2012-02-02
申请号：PCT/US2011/045972
申请日：2011-07-29
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYRAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYRAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
IPC分类号： A61K47/48
CPC分类号： A61K47/44 , A61K47/183 , A61K47/42 , A61K47/54 , A61K47/542 , A61K47/549 , A61K47/60 , A61K47/61 , A61K47/62 , A61K47/6911 , A61K47/6919
摘要： A targeting lipid can have the formula (I) in which T4 is a targeting moiety. The targeting moiety can be chosen to favor the lipid being localized with a desired organ, tissue, cell, cell type or subtype, or organelle. The targeting moiety can include, for example, transferrin, anisamide, an RGD peptide, prostate specific membrane antigen (PSMA), fucose, an antibody, or an aptamer.
摘要翻译：靶向脂质可以具有式（I），其中T4是靶向部分。可以选择靶向部分以有利于所需的器官，组织，细胞，细胞类型或亚型或细胞器定位脂质。靶向部分可以包括例如转铁蛋白，茴香酰胺，RGD肽，前列腺特异性膜抗原（PSMA），岩藻糖，抗体或适体。

2. 发明申请

WO2010054406A1 NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS 审中-公开
标题翻译：用于输送治疗药物的新型脂肪和组合物
公开(公告)号：WO2010054406A1
公开(公告)日：2010-05-14
申请号：PCT/US2009/063933
申请日：2009-11-10
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy , ELTEPU, Laxman
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy , ELTEPU, Laxman
IPC分类号： A01N43/04
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 provides lipids having the following structures: (Formula (I) or (XXXV))
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。特别地，本发明提供具有以下结构的脂质：（式（I）或（XXXV））

3. 发明申请

WO2010054405A1 NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS 审中-公开
标题翻译：用于输送治疗药物的新型脂肪和组合物
公开(公告)号：WO2010054405A1
公开(公告)日：2010-05-14
申请号：PCT/US2009/063931
申请日：2009-11-10
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , MAIER, Martin , ELTEPU, Laxman
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , MAIER, Martin , ELTEPU, Laxman
IPC分类号： A61K47/00 , A61K31/01
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 provides lipids having the following structure (I) wherein R 1 and R 2 are each independently for each occurrence optionally substituted C 10 -C 30 alkyl, optionally substituted C 10 -C 30 alkenyl, optionally substituted C 10 -C 30 alkynyl, optionally substituted C 10 -C 30 acyl, or -linker-ligand; R 3 is H, optionally substituted C 1 -C 10 alkyl, optionally substituted C 2 -C 10 alkenyl, optionally substituted C 2 -C 10 alkynyl, alkylhetrocycle, 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; E is O, S, N(Q), C(O), N(Q)C(O), C(O)N(Q), (Q)N(CO)O, O(CO)N(Q), S(O), NS(O)2N(Q), S(O)2, N(Q)S(O)2, SS, O=N, aryl, heteroaryl, cyclic or heterocycle; and, Q is H, alkyl, ω-aminoalkyl, ω-(substituted)aminoalky, ω-phosphoalkyl or ω-thiophosphoalkyl.
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。特别地，本发明提供具有以下结构（I）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C 10 -C 30 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链烯基，任选取代的C 2 -C 10炔基，烷基化合物，烷基磷酸酯，烷基硫代磷酸酯，烷基二硫代磷酸酯，烷基膦酸酯，烷基胺，羟基烷基， - （取代的）氨基烷基，α-磷酸烷基， - 硫代磷酸烷基，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; E是O，S，N（Q），C（O），N（Q）C（O），C（O）N（Q），（Q）），S（O），NS（O）2N（Q），S（O）2，N（Q）S（O）2，SS，O = N，芳基，杂芳基，环状或杂环; Q为H，烷基，α-氨基烷基，β-（取代）氨基烷基，α-磷酸烷基或α-硫代磷酰烷基。

4. 发明申请

WO2009082607A2 TARGETING LIPIDS 审中-公开
标题翻译：目标人物
公开(公告)号：WO2009082607A2
公开(公告)日：2009-07-02
申请号：PCT/US2008/085582
申请日：2008-12-04
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy
IPC分类号： A61K47/48
CPC分类号： A61K47/28 , A61K31/70 , A61K31/7004 , A61K31/7052 , A61K31/7088 , A61K31/713 , A61K47/16 , A61K47/22 , A61K47/543 , A61K47/549 , A61K47/60 , A61K48/00 , C07H21/02 , Y02A50/385 , Y02A50/411 , Y02A50/467 , Y02P20/55
摘要： The present invention provides targeting lipids of structure (Cl) L 100 - linker - L 101 , where L 100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L 101 is a ligand or - CH 2 CH 2 (OCH 2 CH 2 ) p O(CH 2 ) q CH 2 -ligand, p is 1-1000, and q is 1-20. In addition, the invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic aicd to cells in vivo .
摘要翻译：本发明提供结构（C1）L100-接头-L101的靶向脂质，其中L100是脂质，亲脂性，烷基，烯基或炔基，L101是配体或-CH 2 CH 2（OCH 2 CH 2）p O（CH 2）q CH 2 - 配体，p 为1-1000，q为1-20。此外，本发明提供了将治疗剂递送至细胞的组合物和方法。特别地，这些包括提供核酸的有效包封的新型脂质和核酸 - 脂质颗粒，并将包封的核酸在体内有效递送到细胞。

5. 发明申请

WO2010144740A1 IMPROVED LIPID FORMULATION 审中-公开
标题翻译：改进的脂质配方
公开(公告)号：WO2010144740A1
公开(公告)日：2010-12-16
申请号：PCT/US2010/038224
申请日：2010-06-10
申请人： ALNYLAM PHARMACEUTICALS, INC. , CHEN, Jianxin , ANSELL, Steven , AKINC, Akin , DORKIN, Joseph, R. , QIN, Xiaojun , CANTLEY, William , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy
发明人： CHEN, Jianxin , ANSELL, Steven , AKINC, Akin , DORKIN, Joseph, R. , QIN, Xiaojun , CANTLEY, William , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , NARAYANANNAIR, Jayaprakash, K. , JAYARAMAN, Muthusamy
IPC分类号： A61K9/127 , C07C211/00 , A61K31/20
CPC分类号： A61K9/1272 , A61K9/1278 , A61K31/22 , A61K31/7088 , A61K31/712 , A61K31/713 , A61K38/1709 , A61K38/4846 , A61K47/14 , A61K47/549 , A61K47/551 , A61K47/6911 , A61K48/0033 , C07C29/58 , C07C227/18 , C07C227/40 , C07C229/12 , C07D475/04 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , Y02A50/463 , A61K2300/00 , C12N2310/3521 , C12N2310/3533
摘要： The invention features a cationic lipid of formula (I), an improved lipid formulation comprising a cationic lipid of formula I and corresponding methods of use. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译：本发明的特征在于式（I）的阳离子脂质，包含式I的阳离子脂质和相应的使用方法的改进的脂质制剂。还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。

6. 发明申请

WO2010054384A1 LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS 审中-公开
标题翻译：用于输送治疗药物的脂肪和组合物
公开(公告)号：WO2010054384A1
公开(公告)日：2010-05-14
申请号：PCT/US2009/063897
申请日：2009-11-10
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , CIUFOLINI, Marco, A.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , BUTLER, David , NARAYANANNAIR, Jayaprakash, K. , CIUFOLINI, Marco, A.
IPC分类号： A61K47/00 , A61K31/01
CPC分类号： A61K31/7088 , A61K9/1272 , A61K31/7105 , A61K39/00 , A61K2039/55561 , A61K2039/6018 , C12N15/111 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2320/32 , C12N2310/3521 , C12N2310/3533
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 provides lipids having the following structures XIV or XVII.
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。特别地，本发明提供具有以下结构XIV或XVII的脂质。

7. 发明申请

WO2009082606A2 FOLATE CONJUGATES 审中-公开
标题翻译：聚光灯
公开(公告)号：WO2009082606A2
公开(公告)日：2009-07-02
申请号：PCT/US2008/085577
申请日：2008-12-04
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
IPC分类号： A61K47/48
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(R ) 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-L 6 -Q'-L 7 -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-L 6 -Q'-L 7 -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)I1-, -C(R')(R")(CH2)n-, - (CH 2 ) n C(R')(R")-, -(CH 2 CH 2 O) m CH 2 CH 2 -, or -(CH 2 CH 2 O) m CH 2 CH 2 NH-; Q' is NH, O, S, CH 2 , C(O)O, C(O)NH, -NH-CH(R a )-C(0)-, -C(0)-CH(R a )-NH-, CO, formula (A) where R a is H or amino acid side;;chain. R' and R" are each independently H, CH 3 , OH, SH, NH 2 , NH(Alkyl = Me, Et, Pr, isoPr, Bu, Bn) or N(diAlkyl = Me 2 , Et 2 , Bn 2 ); Z', Z", Z' " and Z"" are independently 0 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（R）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' “”）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“）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“”）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; （CH 2）n - （CH 2）n C（R'）（R“） - ， - （CH 2 CH 2 O））mCH2CH2-或 - （CH2CH2O）mCH2CH2NH-; Q'是NH，O，S，CH 2，C（O）O，C（O）NH，-NH-CH（R a）-C（O） - ， - C（O），CO，式（A）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= Me2，Et2，Bn2）; Z'，Z “，Z”“和”Z“”独立地为0或S; n代表每次出现1-20个独立地表示; m代表每次出现的0-50。

8. 发明申请

WO2009126933A2 SITE-SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS 审中-公开
标题翻译：通过将目标配体与内分泌组分组合来实现特定地提供核酸
公开(公告)号：WO2009126933A2
公开(公告)日：2009-10-15
申请号：PCT/US2009/040274
申请日：2009-04-10
申请人： ALNYLAM PHARMACEUTICALS, INC. , MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , BUTLER, David , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K. , MATSUDA, Shigeo
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , BUTLER, David , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K. , MATSUDA, Shigeo
IPC分类号： A61K48/00
CPC分类号： A61K47/545 , A61K47/549 , A61K47/551 , A61K47/554 , 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。

9. 发明授权

EP2231194B1 FOLATE-IRNA CONJUGATES 有权
标题翻译： FOLAT-IRNA-KONJUGATE
公开(公告)号：EP2231194B1
公开(公告)日：2017-02-22
申请号：EP08864322.6
申请日：2008-12-04
申请人： Alnylam Pharmaceuticals Inc.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
IPC分类号： A61K47/54
CPC分类号： C12N15/113 , A61K47/55

10. 发明公开

EP2231194A2 FOLATE-IRNA CONJUGATES 有权
标题翻译：叶酸IRNA结合物
公开(公告)号：EP2231194A2
公开(公告)日：2010-09-29
申请号：EP08864322.6
申请日：2008-12-04
申请人： Alnylam Pharmaceuticals Inc.
发明人： MANOHARAN, Muthiah , RAJEEV, Kallanthottathil, G. , JAYARAMAN, Muthusamy , NARAYANANNAIR, Jayaprakash, K.
IPC分类号： A61K47/48
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(R ) 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-L
6 -Q'-L
7 -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-L
6 -Q'-L
7 -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)I1-, -C(R')(R")(CH2)n-, - (CH
2 )
n C(R')(R")-, -(CH
2 CH
2 O)
m CH
2 CH
2 -, or -(CH
2 CH
2 O)
m CH
2 CH
2 NH-; Q' is NH, O, S, CH
2 , C(O)O, C(O)NH, -NH-CH(R
a )-C(0)-, -C(0)-CH(R
a )-NH-, CO, formula (A) where R
a is H or amino acid side;;chain. R' and R" are each independently H, CH
3 , OH, SH, NH
2 , NH(Alkyl = Me, Et, Pr, isoPr, Bu, Bn) or N(diAlkyl = Me
2 , Et
2 , Bn
2 ); Z', Z", Z' " and Z"" are independently 0 or S; n represent independently for each occurrence 1-20; and m represent independently for each occurrence 0-50.

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