公开(公告)号：EP2663548B1

公开(公告)日：2017-04-05

申请号：EP12700912.4

申请日：2012-01-11

申请人： Alnylam Pharmaceuticals, Inc.

发明人： RAJEEV, Kallanthottathil, G. ,  MANOHARAN, Muthiah ,  JAYARAMAN, Muthusamy ,  MAIER, Martin ,  JAYAPRAKASH, Narayanannair, K. ,  BUTLER, David

IPC分类号： C07D207/14 ,  C07D307/12 ,  C08L71/02 ,  C12N15/88 ,  A61K9/127

CPC分类号： A61K47/18 ,  A61K47/6911 ,  C07C235/06 ,  C07C271/16 ,  C07D207/12 ,  C07D207/14 ,  C07D307/12 ,  C07D307/20 ,  C08G65/329 ,  C08L2203/02

摘要： The invention provides poly(ethylene glycol)-lipid conjugates for use in drug delivery.

公开(公告)号：EP2663548A1

公开(公告)日：2013-11-20

申请号：EP12700912.4

申请日：2012-01-11

申请人： Alnylam Pharmaceuticals, Inc.

发明人： RAJEEV, Kallanthottathil, G. ,  MANOHARAN, Muthiah ,  JAYARAMAN, Muthusamy ,  MAIER, Martin ,  JAYAPRAKASH, Narayanannair, K. ,  BUTLER, David

IPC分类号： C07D207/14 ,  C07D307/12 ,  C08L71/02 ,  C12N15/88 ,  A61K9/127

CPC分类号： A61K47/18 ,  A61K47/6911 ,  C07C235/06 ,  C07C271/16 ,  C07D207/12 ,  C07D207/14 ,  C07D307/12 ,  C07D307/20 ,  C08G65/329 ,  C08L2203/02

摘要： The invention provides poly(ethylene glycol)-lipid conjugates for use in drug delivery.

摘要翻译： 本发明提供的聚（乙二醇），用于药物递送 - 脂质缀合物。

公开(公告)号：EP2509636A2

公开(公告)日：2012-10-17

申请号：EP10836504.0

申请日：2010-12-07

申请人： Alnylam Pharmaceuticals, Inc.

发明人： MANOHARAN, Muthiah ,  RAJEEV, Kallanthottahil, G. ,  JAYARAMAN, Muthusamy ,  BUTLER, David ,  KAPOOR, Mamta ,  KAINTHAN, Rajesh, Kumar

IPC分类号： A61K48/00 ,  C07C269/02

CPC分类号： A61K47/18 ,  A61K31/7105 ,  A61K31/713 ,  A61K47/186 ,  A61K47/22 ,  C07C217/08 ,  C07C219/08 ,  C07C229/12 ,  C07C251/40 ,  C07C271/12 ,  C07C271/20 ,  C07C323/25 ,  C07C2601/02 ,  C07D317/28 ,  C07D317/44 ,  C07D317/72 ,  C07D491/10 ,  C12N15/88

摘要： A method for delivering a nucleic acid to a cell can include exposing sample cells to a composition which includes charged lipids.

公开(公告)号：EP3587434A1

公开(公告)日：2020-01-01

申请号：EP19173718.8

申请日：2009-09-23

申请人： Alnylam Pharmaceuticals Inc.

发明人： MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil G. ,  YAMADA, Takeshi ,  BUTLER, David ,  JAYAPRAKASH, K. Narayanannair ,  JAYRAMAN, Muthusamy ,  MATSUDA, Shigeo ,  PANDEY, Rajendra K. ,  PENG, Chang Geng

IPC分类号： C07H21/00 ,  A61K31/7088 ,  A61P31/12

摘要： The present invention relates to specific modified oligonucleotide compounds, pharmaceutical compositions comprising the same, and uses thereof.

公开(公告)号：EP2416760A1

公开(公告)日：2012-02-15

申请号：EP10772790.1

申请日：2010-05-05

申请人： Alnylam Pharmaceuticals, Inc. ,  Manoharan, Muthiah ,  Rajeev, Kallanthottathil G. ,  Jayaraman, Muthusamy ,  Butler, David ,  Jung, Michael, E.

发明人： MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil, G. ,  JAYARAMAN, Muthusamy ,  BUTLER, David ,  JUNG, Michael, E.

IPC分类号： A61K9/127 ,  A61K31/50

CPC分类号： A61K47/22 ,  A61K9/1272 ,  C07D295/13 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/14 ,  Y02A50/463

摘要： Disclosed herein are lipid compositions comprising a cationic lipid formula (I), a neutral lipid, a sterol and a PEG or PEG-modified lipid, wherein formula (I) is (F). Also disclosed are methods of producing the cationic lipid of formula (I).

公开(公告)号：EP3202760B1

公开(公告)日：2019-08-21

申请号：EP17000424.6

申请日：2012-01-11

申请人： Alnylam Pharmaceuticals, Inc.

发明人： RAJEEV, Kallanthottathil G. ,  MANOHARAN, Muthiah ,  JAYARAMAN, Muthusamy ,  MAIER, Martin ,  JAYAPRAKASH, Narayanannair K. ,  BUTLER, David

IPC分类号： C07D207/14 ,  C07D307/12 ,  C08L71/02 ,  C12N15/88 ,  A61K9/127

公开(公告)号：EP2367571A1

公开(公告)日：2011-09-28

申请号：EP09825613.4

申请日：2009-11-10

申请人： Alnylam Pharmaceuticals Inc.

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, É-aminoalkyls, É-(substituted)aminoalkyls, É-phosphoalkyls, É-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, É-aminoalkyl, É-(substituted)aminoalky, É-phosphoalkyl or É-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的脂质：（式（I）或（XXXV））。

公开(公告)号：EP2355658A1

公开(公告)日：2011-08-17

申请号：EP09825614.2

申请日：2009-11-10

申请人： Alnylam Pharmaceuticals Inc.

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 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, É-aminoalkyls, É-(substituted)aminoalkyls, É-phosphoalkyls, É-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, É-aminoalkyl, É-(substituted)aminoalky, É-phosphoalkyl or É-thiophosphoalkyl.

公开(公告)号：EP2342616A2

公开(公告)日：2011-07-13

申请号：EP09737234.6

申请日：2009-09-23

申请人： Alnylam Pharmaceuticals Inc.

发明人： MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil, G. ,  YAMADA, Takeshi ,  BUTLER, David ,  JAYAPRAKASH, K., Narayanannair ,  JAYRAMAN, Muthusamy ,  MATSUDA, Shigeo ,  PANDEY, Rajendra, K. ,  PENG, Chang, Geng

IPC分类号： G06F3/01 ,  G06F15/16

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/544 ,  A61K47/64 ,  C07H21/00 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/3513 ,  C12N2310/3515 ,  H04L65/602 ,  H04L67/36 ,  Y02A50/465

摘要： The invention features compounds of formula I or II: In one embodiment, the invention relates compounds and processes for conjugating ligand to oligonucleotide. The invention further relates to methods for treating various disorders and diseases such as viral infections, bacterial infections, parasitic infections, cancers, allergies, autoimmune diseases, immunodeficiencies and immunosuppression.

摘要翻译： 本发明的特征在于式I或II化合物：在一个实施方案中，本发明涉及将配体与寡核苷酸缀合的化合物和方法。 本发明还涉及治疗各种病症和疾病如病毒感染，细菌感染，寄生虫感染，癌症，过敏症，自身免疫疾病，免疫缺陷和免疫抑制的方法。

公开(公告)号：EP2274425A2

公开(公告)日：2011-01-19

申请号：EP09730178.2

申请日：2009-04-10

申请人： Alnylam Pharmaceuticals Inc.

发明人： MANOHARAN, Muthiah ,  RAJEEV, Kallanthottathil, G. ,  BUTLER, David ,  JAYARAMAN, Muthusamy ,  NARAYANANNAIR, Jayaprakash, K. ,  MATSUDA, Shigeo

IPC分类号： C12N15/11

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： 本发明通过将它们与靶向配体和内涵体溶解组分合并涉及用于核酸的位点特异性递送的组合物和方法。