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1. 发明申请

WO2009082607A8 TARGETING LIPIDS 审中-公开
标题翻译：目标人物
公开(公告)号：WO2009082607A8
公开(公告)日：2010-01-28
申请号：PCT/US2008085582
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 , Y02P20/55
摘要： The present invention provides targeting lipids of structure (Cl) L100 - linker - L101, where L100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L101 is a ligand or - CH2CH2(OCH2CH2)pO(CH2)qCH2-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。

2. 发明申请

WO2009082607A3 TARGETING LIPIDS 审中-公开
标题翻译：目标人物
公开(公告)号：WO2009082607A3
公开(公告)日：2009-12-17
申请号：PCT/US2008085582
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 , Y02P20/55
摘要： The present invention provides targeting lipids of structure (Cl) L100 - linker - L101, where L100 is a lipid, lipophile, alkyl, alkenyl or alkynyl, L101 is a ligand or - CH2CH2(OCH2CH2)pO(CH2)qCH2-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。

3. 发明申请

WO2009126933A3 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：WO2009126933A3
公开(公告)日：2010-01-28
申请号：PCT/US2009040274
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： A61K31/713 , A61K47/48 , C07H21/00 , C12N15/11 , C12N15/87
CPC分类号： A61K47/48092 , 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。

4. 发明申请

WO2009082606A3 FOLATE-IRNA CONJUGATES 审中-公开
标题翻译： FOLATE-IRNA结合
公开(公告)号：WO2009082606A3
公开(公告)日：2009-12-17
申请号：PCT/US2008085577
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-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)I1-, -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(0)-, -C(0)-CH(Ra)-NH-, CO, formula (A) 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 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。

5. 发明申请

WO2010048536A2 PROCESSES FOR PREPARING LIPIDS 审中-公开
标题翻译：制备脂肪的方法
公开(公告)号：WO2010048536A2
公开(公告)日：2010-04-29
申请号：PCT/US2009061897
申请日：2009-10-23
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , JAYARAMAN MUTHUSAMY
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTATHIL G , JAYARAMAN MUTHUSAMY
IPC分类号： A61K48/00
CPC分类号： C07D317/28 , C12N15/88
摘要： The present 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. The compositions of the present invention are highly potent, thereby allowing effective knock-down of specific tagrget protein at relatively low doses. The compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art.
摘要翻译：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。本发明的组合物是非常有效的，从而允许在相对低的剂量下有效地敲除特定的标记蛋白。与本领域先前已知的组合物和方法相比，本发明的组合物和方法毒性低，并提供更大的治疗指数。

6. 发明申请

WO2011153493A3 BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：用于交付活性剂的生物可降解脂肪
公开(公告)号：WO2011153493A3
公开(公告)日：2012-01-19
申请号：PCT/US2011039164
申请日：2011-06-03
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , MAIER MARTIN , JAYARAMAN MUTHUSAMY , MATSUDA SHIGEO , JAYAPRAKASH NARAYANANNAIR K , RAJEEV KALLANTHOTTATHIL G
发明人： MANOHARAN MUTHIAH , MAIER MARTIN , JAYARAMAN MUTHUSAMY , MATSUDA SHIGEO , JAYAPRAKASH NARAYANANNAIR K , RAJEEV KALLANTHOTTATHIL G
IPC分类号： A61K9/10 , A61K39/00 , A61K47/48 , A61K48/00 , C07C69/02 , C07C211/62 , C07C211/63 , C12N15/87
CPC分类号： C07C229/12 , A61K31/713 , A61K39/0005 , A61K47/44 , C12N15/113 , C12N15/88 , C12N2310/14 , C12N2320/30
摘要： The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.
摘要翻译：本发明涉及一种阳离子脂质，其具有位于阳离子脂质的脂质部分（例如，疏水链）的中间或远端部分中的一个或多个可生物降解基团。这些阳离子脂质可以掺入用于递送活性剂（例如核酸）的脂质颗粒中。本发明还涉及包含中性脂质，能够减少聚集的脂质，本发明的阳离子脂质和任选的固醇的脂质颗粒。脂质颗粒可以进一步包括治疗剂如核酸。

7. 发明申请

WO2010088537A3 IMPROVED LIPID FORMULATION FOR THE DELIVERY OF NUCLEIC ACIDS 审中-公开
标题翻译：改进的提供核酸的脂质配方
公开(公告)号：WO2010088537A3
公开(公告)日：2010-09-23
申请号：PCT/US2010022614
申请日：2010-01-29
申请人： ALNYLAM PHARMACEUTICALS INC , AKINC AKIN , QUERBES WILLIAM , WONG FRANCES , DORKIN JOSEPH ROBERT , QIN XIAOJUN , CANTLEY WILLIAM , BORODOVSKY ANNA , DE SOMA , MANOHARAN MUTHIAH , JAYARAMAN MUTHUSAMY , RAJEEV KALLANTHOTTATHIL G
发明人： AKINC AKIN , QUERBES WILLIAM , WONG FRANCES , DORKIN JOSEPH ROBERT , QIN XIAOJUN , CANTLEY WILLIAM , BORODOVSKY ANNA , DE SOMA , MANOHARAN MUTHIAH , JAYARAMAN MUTHUSAMY , RAJEEV KALLANTHOTTATHIL G
IPC分类号： A61K9/127 , A61K48/00
CPC分类号： A61K48/0008 , A61K9/1272
摘要： The invention features an improved lipid formulation comprising a cationic lipid of formula (A), a neutral lipid, a sterol and a PEG or PEG-modified lipid, where R1 and R2 are independently alkyl, alkenyl or alkynyl, each can be optionally substituted, and R3 and R4 are independently lower alkyl or R3 and R4 can be taken together to form an optionally subsituted heterocyclic ring. In one embodiment, R1 and R2 are independently selected from oleoyl, pamitoyl, steroyl, linoleyl and R3 and R4 are methyl. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids.
摘要翻译：本发明的特征在于改进的脂质制剂，其包含式（A）的阳离子脂质，中性脂质，固醇和PEG或PEG修饰的脂质，其中R 1和R 2独立地为烷基，烯基或炔基，各自可任选被取代，并且R 3和R 4独立地为低级烷基或R 3和R 4可以一起形成任选取代的杂环。在一个实施方案中，R 1和R 2独立地选自油酰基，苯甲酰基，甾烷基，亚油酰基，并且R 3和R 4是甲基。还公开了靶向脂质和包含这种靶向脂质的特定脂质制剂。

8. 发明申请

WO2012016184A2 METHODS AND COMPOSITIONS FOR DELIVERY OF ACTIVE AGENTS 审中-公开
标题翻译：递送活性剂的方法和组合物
公开(公告)号：WO2012016184A2
公开(公告)日：2012-02-02
申请号：PCT/US2011045963
申请日：2011-07-29
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTHATHIL G , JAYARAMAN MUTHUSAMY , JUNG MICHAEL E
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTHATHIL G , JAYARAMAN MUTHUSAMY , JUNG MICHAEL E
IPC分类号： C07C229/28
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 unstaturation 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：脂质颗粒可以包含阳离子脂质。阳离子脂质可以包括一个或多个疏水性尾部，其可以包括一个或多个不饱和位点。不饱和位点可以包括环烷基，例如环丙基，环丁基，环戊基或环己基。脂质颗粒适合于递送活性剂。

9. 发明申请

WO2011094580A3 CHELATED COPPER FOR USE IN THE PREPARATION OF CONJUGATED OLIGONUCLEOTIDES 审中-公开
标题翻译：用于制备结合的寡核苷酸的螯合铜
公开(公告)号：WO2011094580A3
公开(公告)日：2012-06-28
申请号：PCT/US2011022977
申请日：2011-01-28
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , ELTEPU LAXMAN , RAJEEV KALLANTHOTTAHIL G , JAYARAMAN MUTHUSAMY
发明人： MANOHARAN MUTHIAH , ELTEPU LAXMAN , RAJEEV KALLANTHOTTAHIL G , JAYARAMAN MUTHUSAMY
IPC分类号： B01J31/18 , B01J31/16 , B01J31/22 , C07D213/22 , C07D249/04 , C12Q1/68
CPC分类号： B01J31/1658 , B01J31/1683 , B01J31/1805 , B01J31/1815 , B01J2231/327 , B01J2531/0244 , B01J2531/0258 , B01J2531/16 , C07D249/04 , C07H21/00
摘要： In one embodiment, the invention features a process for preparing ligand conjugated oligonucleotides comprising a step of contacting a nucleoside or oligonucleotide containing an alkyne moiety with ligand containing an azide moiety in the presence of a solid supported copper catalyst of formula A, B, C or D.
摘要翻译：在一个实施方案中，本发明的特征在于制备配体缀合的寡核苷酸的方法，其包括在式A，B，C的固体负载的铜催化剂存在下使含有炔部分的核苷或寡核苷酸与含有叠氮化物部分的配体接触的步骤，或 D.

10. 发明申请

WO2011071860A3 COMPOSITIONS FOR NUCLEIC ACID DELIVERY 审中-公开
标题翻译：用于核酸递送的组合物
公开(公告)号：WO2011071860A3
公开(公告)日：2011-09-09
申请号：PCT/US2010059206
申请日：2010-12-07
申请人： ALNYLAM PHARMACEUTICALS INC , MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTAHIL G , JAYARAMAN MUTHUSAMY , BUTLER DAVID , KAPOOR MAMTA , KAINTHAN RAJESH KUMAR
发明人： MANOHARAN MUTHIAH , RAJEEV KALLANTHOTTAHIL G , JAYARAMAN MUTHUSAMY , BUTLER DAVID , KAPOOR MAMTA , KAINTHAN RAJESH KUMAR
IPC分类号： C12N5/02 , C07C229/00
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.
摘要翻译：将核酸递送至细胞的方法可包括将样品细胞暴露于包含带电脂质的组合物中。

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