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    Folate Conjugates
    34.
    发明申请
    Folate Conjugates 有权
    叶酸缀合物

    公开(公告)号：US20090247614A1

    公开(公告)日：2009-10-01

    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： A61K31/7088 ,  C07H21/02 ,  C12N5/02

    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 L1 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; （CH 2）n - ， - （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。

    Targeting Lipids
    36.
    发明申请
    Targeting Lipids 审中-公开
    靶向脂质

    公开(公告)号：US20090247608A1

    公开(公告)日：2009-10-01

    申请号：US12328669

    申请日：2008-12-04

    申请人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Gayaprakash K. Narayananovair ,  Muthusamy Jayaraman

    发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Gayaprakash K. Narayananovair ,  Muthusamy Jayaraman

    IPC分类号： A61K31/713 ,  C07K14/00 ,  A61K47/42 ,  A61K31/7088 ,  C07K7/04 ,  C07K5/04 ,  A61K47/12

    摘要： The present invention provides targeting lipids of structure L100—linker—L101   (CI), 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 acid to cells in vivo.

    摘要翻译： 本发明提供了结构<？in-line-formula description =“In-line Formulas”end =“lead”？> L100-linker-L101（CI），<？in-line-formula description =“In 其中L100是脂质，亲脂性，烷基，烯基或炔基，L101是配体或-CH2CH2（OCH2CH2）pO（CH2）qCH2-配体，p是1-1000，和 q是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，并将包封的核酸有效地递送到细胞体内。

    Carbohydrate conjugates as delivery agents for oligonucleotides
    40.
    发明授权
    Carbohydrate conjugates as delivery agents for oligonucleotides 有权
    碳水化合物缀合物作为寡核苷酸的递送剂

    公开(公告)号：US08106022B2

    公开(公告)日：2012-01-31

    申请号：US12328528

    申请日：2008-12-04

    申请人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Jayaprakash K. Narayanannair ,  Martin Maier

    发明人： Muthiah Manoharan ,  Kallanthottathil G. Rajeev ,  Jayaprakash K. Narayanannair ,  Martin Maier

    IPC分类号： A01N43/04 ,  A61K31/70

    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 iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(RN) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl 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-Linker-OP(Z′″)(Z″″)O-oligonucleotide, a nucleotide, an oligonucleotide, —P(Z′)(Z″)-formula (I), —P(Z′)(Z″)— or -Linker-R; R is LG, -Linker-LG, or has the structure shown below: LG is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; RN is independently for each occurrence H, methyl, ethyl, propyl, isopropyl, butyl, or benzyl; and Z′, Z″, Z′″ and Z″″ are each independently for each occurrence O or S.

    摘要翻译： 本发明提供包含至少一个式（I）亚基的iRNA试剂：其中：A和B各自独立地为O，N（RN）或S; X和Y各自独立地为H，OH，羟基保护基，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-Linker- OP（Z'“）（Z”“） - 寡核苷酸，寡核苷酸，-P（Z'）（Z”） - 式（I），-P（Z'）（Z“） - 链接器R R是LG，-Linker-LG，或具有如下结构：每次出现LG独立为碳水化合物，例如单糖，二糖，三糖，四糖，寡糖，多糖; 乙烯基，丙基，异丙基，丁基或苄基各自独立地为N， 和Z'，Z“，Z”“和Z”“各自独立地为O或S.