公开(公告)号：WO2021183563A1

公开(公告)日：2021-09-16

申请号：PCT/US2021/021572

申请日：2021-03-09

Applicant: ARCTURUS THERAPEUTICS, INC. ,  SULLIVAN, Sean Michael ,  MATSUDA, Daiki ,  TACHIKAWA, Kiyoshi ,  CHIVUKULA, Padmanabh ,  KARMALI, Priya Prakash ,  DAVIS, Jared Henry ,  BAO, Yanjie ,  SAGI, Amit

Inventor： SULLIVAN, Sean Michael ,  MATSUDA, Daiki ,  TACHIKAWA, Kiyoshi ,  CHIVUKULA, Padmanabh ,  KARMALI, Priya Prakash ,  DAVIS, Jared Henry ,  BAO, Yanjie ,  SAGI, Amit

IPC: A61K39/215 ,  A61P31/14 ,  C07K14/18 ,  C12N7/01 ,  C12N15/86

Abstract: Provided herein are nucleic acid molecules encoding viral replication proteins and antigenic coronavirus proteins or fragments thereof. Also provided herein are compositions that include nucleic acid molecules encoding viral replication and antigenic proteins, and lipids. Nucleic acid molecules provided herein are useful for inducing immune responses.

公开(公告)号：WO2012170952A9

公开(公告)日：2013-02-21

申请号：PCT/US2012041753

申请日：2012-06-08

Applicant: NITTO DENKO CORP ,  NIITSU YOSHIRO ,  PAYNE JOSEPH E ,  HOU ZHENG ,  GAUDETTE JOHN A ,  NAGARAJAN SRIDHAR C ,  KNOPOV VICTOR ,  WITTE RICHARD P ,  AHMADIAN MOHAMMAD ,  PERELMAN LOREN A ,  TANAKA YASUNOBU ,  AKOPIAN VIOLETTA ,  KARMALI PRIYA

Inventor： NIITSU YOSHIRO ,  PAYNE JOSEPH E ,  HOU ZHENG ,  GAUDETTE JOHN A ,  NAGARAJAN SRIDHAR C ,  KNOPOV VICTOR ,  WITTE RICHARD P ,  AHMADIAN MOHAMMAD ,  PERELMAN LOREN A ,  TANAKA YASUNOBU ,  AKOPIAN VIOLETTA ,  KARMALI PRIYA

IPC: C07C237/08 ,  A61K31/16 ,  A61P1/16 ,  C07C237/22 ,  C07C323/60 ,  C07C333/04

CPC classification number: C07C237/08 ,  A61K9/1271 ,  C07C237/22 ,  C07C323/60 ,  C07C333/04

Abstract: Here described are compounds of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleyl group; wherein R3 and R4 are independently selected from a group consisting of C1 to C6 alkyl, and C2 to C6 alkanol; wherein X is selected from a group consisting of -CH2-, -S-, and -O- or absent; wherein Y is selected from -(CH2)n, -S(CH2)n, -O(CH2)n-, thiophene, -SO2(CH2)n-, and ester, wherein n = 1 -4; wherein a = 1 -4; wherein b=l -4; wherein c=l-4; and wherein Z is a counterion; and compounds consisting of the structure (targeting molecule)m-linker-(targeting molecule)n, wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including one or both of these compounds which are useful for the delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

公开(公告)号：WO2018119163A1

公开(公告)日：2018-06-28

申请号：PCT/US2017/067756

申请日：2017-12-20

Applicant: PAYNE, Joseph, E. ,  CHIVUKULA, Padmanabh ,  TANIS, Steven, P. ,  KARMALI, Priya

Inventor： PAYNE, Joseph, E. ,  CHIVUKULA, Padmanabh ,  TANIS, Steven, P. ,  KARMALI, Priya

IPC: C07C333/04

Abstract: What is described is a compound of formula (I) consisting of a compound in which R1 is a branched chain alkyl consisting of 10 to 31 carbons; R2 is a linear alkyl, alkenyl, or alkynyl consisting of 2 to 20 carbons, or a branched chain alkyl consisting of 10 to 31 carbons; L1 and L2 are the same or different, each a linear alkane of 1 to 20 carbons or a linear alkene of 2 to 20 carbons; X1 is S or O; R3 is a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each a hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons; or a pharmaceutically acceptable salt thereof.

公开(公告)号：WO2012170952A2

公开(公告)日：2012-12-13

申请号：PCT/US2012/041753

申请日：2012-06-08

Applicant: NITTO DENKO CORPORATION ,  NIITSU, Yoshiro ,  PAYNE, Joseph E. ,  HOU, Zheng ,  GAUDETTE, John A. ,  NAGARAJAN, Sridhar C. ,  KNOPOV, Victor ,  WITTE, Richard P. ,  AHMADIAN, Mohammad ,  PERELMAN, Loren A. ,  TANAKA, Yasunobu ,  AKOPIAN, Violetta ,  KARMALI, Priya

Inventor： NIITSU, Yoshiro ,  PAYNE, Joseph E. ,  HOU, Zheng ,  GAUDETTE, John A. ,  NAGARAJAN, Sridhar C. ,  KNOPOV, Victor ,  WITTE, Richard P. ,  AHMADIAN, Mohammad ,  PERELMAN, Loren A. ,  TANAKA, Yasunobu ,  AKOPIAN, Violetta ,  KARMALI, Priya

IPC: C07C237/08 ,  A61K31/16 ,  A61P1/16 ,  C07C237/22 ,  C07C323/60 ,  C07C333/04

CPC classification number: C07C237/08 ,  A61K9/1271 ,  C07C237/22 ,  C07C323/60 ,  C07C333/04

Abstract: Here described are compounds of formula I: wherein R 1 and R 2 is independently selected from a group consisting of C 10 to C 18 alkyl, C 12 to C 18 alkenyl, and oleyl group; wherein R 3 and R 4 are independently selected from a group consisting of C 1 to C 6 alkyl, and C 2 to C 6 alkanol; wherein X is selected from a group consisting of -CH 2 -, -S-, and -O- or absent; wherein Y is selected from -(CH 2 )n, -S(CH 2 ) n , -O(CH 2 ) n -, thiophene, -SO 2 (CH 2 ) n -, and ester, wherein n = 1 -4; wherein a = 1 -4; wherein b=l -4; wherein c=l-4; and wherein Z is a counterion; and compounds consisting of the structure (targeting molecule) m -linker-(targeting molecule) n , wherein the targeting molecule is a retinoid or a fat soluble vitamin having a specific receptor on the target cell; wherein m and n are independently 0, 1, 2 or 3; and wherein the linker comprises a polyethylene glycol (PEG) or PEG-like molecule, as well as compositions and pharmaceutical formulations including one or both of these compounds which are useful for the delivery of therapeutic agents; and methods of using these compositions and pharmaceutical formulations.

Abstract translation: 这里描述的是式I化合物：其中R 1和R 2独立地选自C 10至C 18烷基，C 12至C 18烯基和油基; 其中R3和R4独立地选自C1-C6烷基和C2-C6链烷醇; 其中X选自-CH 2 - ， - S-和-O-或不存在; 其中Y选自 - （CH 2）n，-S（CH 2）n，-O（CH 2）n - ，噻吩，-SO 2（CH 2）n - 和酯，其中n = 1-4; 其中a = 1-4; 其中b = 1-4; 其中c = 1-4; 并且其中Z是抗衡离子; 以及由结构（靶向分子）m-接头（靶向分子）n组成的化合物，其中靶向分子是靶细胞上具有特异性受体的类视黄醇或脂溶性维生素; 其中m和n独立地为0,1,2或3; 并且其中所述接头包含聚乙二醇（PEG）或PEG样分子，以及包含这些化合物中的一种或两种可用于递送治疗剂的组合物和药物制剂; 以及使用这些组合物和药物制剂的方法。

公开(公告)号：WO2007116276A3

公开(公告)日：2007-12-21

申请号：PCT/IB2007000826

申请日：2007-03-30

Applicant: COUNCIL SCIENT IND RES ,  MAJETI BHARAT KUMAR ,  KARMALI PRIYA PRAKASH ,  DIPANKAR PRAMANIK ,  CHAUDHURI ARABINDA

Inventor： MAJETI BHARAT KUMAR ,  KARMALI PRIYA PRAKASH ,  DIPANKAR PRAMANIK ,  CHAUDHURI ARABINDA

IPC: A61K48/00 ,  A61K38/07 ,  C07K5/10

CPC classification number: C07K5/1019 ,  A61K38/00 ,  A61K48/00 ,  A61K48/0008

Abstract: The present invention provides synthesis of a novel series of cationic lipopeptides with integrin-binding RGD functionalities. The invention also provides high L27 (transformed Sl 80, mouse sarcoma cells) cell tropic gene transfer properties of these novel RGD-lipopeptides. Since L27 cell surface contains over expressed integrins, the present class of lipopeptides with integrin-binding RGD ligands are likely to find future applications in targeting anti-cancer genes/drugs to the endothelial cells of tumor vasculatures (possessing over expressed integrins).

Abstract translation: 本发明提供具有整联蛋白结合RGD功能的新一系列阳离子脂肽的合成。 本发明还提供了这些新型RGD-脂肽的高L27（转化的S180，小鼠肉瘤细胞）细胞嗜性基因转移特性。 由于L27细胞表面含有过度表达的整联蛋白，所以目前类型的具有整合素结合RGD配体的脂肽很可能会发现将抗癌基因/药物靶向肿瘤血管系统内皮细胞（拥有超表达的整联蛋白）的未来应用。

公开(公告)号：WO2007116276A2

公开(公告)日：2007-10-18

申请号：PCT/IB2007/000826

申请日：2007-03-30

Applicant: COUNCIL OF SCIENTIFIC AND INDUSTRIAL RESEARCH ,  MAJETI, Bharat, Kumar ,  KARMALI, Priya, Prakash ,  DIPANKAR, Pramanik ,  CHAUDHURI, Arabinda

Inventor： MAJETI, Bharat, Kumar ,  KARMALI, Priya, Prakash ,  DIPANKAR, Pramanik ,  CHAUDHURI, Arabinda

IPC: A61K48/00 ,  C07K5/10 ,  A61K38/07

CPC classification number: C07K5/1019 ,  A61K38/00 ,  A61K48/00 ,  A61K48/0008

Abstract: The present invention provides synthesis of a novel series of cationic lipopeptides with integrin-binding RGD functionalities. The invention also provides phenomenally high L27 (transformed Sl 80, mouse sarcoma cells) cell tropic gene transfer properties of these novel RGD-lipopeptides. Since L27 cell surface contains over expressed integrins, the present class of lipopeptides with integrin-binding RGD ligands are likely to find future applications in targeting anti-cancer genes/drugs to the endothelial cells of tumor vasculatures (possessing over expressed integrins).

Abstract translation: 本发明提供具有整联蛋白结合RGD功能的新一系列阳离子脂肽的合成。 本发明还提供了这些新型RGD-脂肽的非常高的L27（转化的S180，小鼠肉瘤细胞）细胞嗜性基因转移特性。 由于L27细胞表面含有过度表达的整联蛋白，所以目前类型的具有整合素结合RGD配体的脂肽很可能会发现将抗癌基因/药物靶向肿瘤血管系统内皮细胞（拥有超表达的整联蛋白）的未来应用。

公开(公告)号：WO2010075540A1

公开(公告)日：2010-07-01

申请号：PCT/US2009/069460

申请日：2009-12-23

Applicant: BURNHAM INSTITUTE FOR MEDICAL RESEARCH ,  RUOSLAHTI, Erkki ,  KOTAMRAJU, Venkata, Ramana ,  KARMALI, Priya

Inventor： RUOSLAHTI, Erkki ,  KOTAMRAJU, Venkata, Ramana ,  KARMALI, Priya

IPC: A61K9/14

CPC classification number: A61K45/06 ,  A61K47/62 ,  A61K47/6907 ,  A61K47/6923 ,  A61K49/0043 ,  A61K49/0082 ,  A61K49/0093 ,  A61K49/1866 ,  B82Y5/00

Abstract: Disclosed are compositions and methods useful for targeting therapeutics to cancerous cells and tumors. The disclosed targeting is useful for delivering therapeutic and detectable agents to cancerous cells and tumors.

Abstract translation: 公开了用于将治疗剂靶向癌细胞和肿瘤的组合物和方法。 所公开的靶向对于将治疗和可检测的药物递送至癌细胞和肿瘤是有用的。

公开(公告)号：WO2013185116A1

公开(公告)日：2013-12-12

申请号：PCT/US2013/044849

申请日：2013-06-07

Applicant: PAYNE, Joseph, E. ,  GAUDETTE, John, A. ,  HOU, Zheng ,  AHMADIAN, Mohammad ,  YU, Lei ,  KNOPOV, Victor ,  AKOPIAN, Violetta ,  KARMALI, Priya ,  WITTE, Richard, P. ,  SAFARZADEH, Neda ,  YING, Wenbin ,  ZHANG, Jun

Inventor： PAYNE, Joseph, E. ,  GAUDETTE, John, A. ,  HOU, Zheng ,  AHMADIAN, Mohammad ,  YU, Lei ,  KNOPOV, Victor ,  AKOPIAN, Violetta ,  KARMALI, Priya ,  WITTE, Richard, P. ,  SAFARZADEH, Neda ,  YING, Wenbin ,  ZHANG, Jun

IPC: C07C271/16 ,  C07D333/40 ,  C07C317/44 ,  C07C321/14 ,  C07C333/04 ,  C07C235/08 ,  C07C237/08 ,  C07D207/16 ,  A61K47/16 ,  A61K47/18 ,  A61K47/22

CPC classification number: A61K47/20 ,  A61K9/1272 ,  A61K47/14 ,  A61K47/18 ,  A61K47/22 ,  C07C235/08 ,  C07C237/08 ,  C07C237/16 ,  C07C271/16 ,  C07C317/44 ,  C07C323/60 ,  C07C333/04 ,  C07D207/16 ,  C07D333/38 ,  C07D333/40 ,  Y10T428/2998

Abstract: The description is directed to ionizable lipids useful for enhancing the delivery of therapeutic agents in liposomes.

Abstract translation: 该描述针对可用于增强脂质体中治疗剂递送的可离子化脂质。

公开(公告)号：WO2009109996A2

公开(公告)日：2009-09-11

申请号：PCT/IN2009000119

申请日：2009-02-24

Applicant: COUNCIL SCIENT IND RES ,  KARMALI PRIYA PRAKASH ,  PRAMANIK DIPANKAR ,  MAJETI BHARAT KUMAR ,  CHAUDHURI ARABINDA ,  SISTLA RAMAKRISHNA

Inventor： KARMALI PRIYA PRAKASH ,  PRAMANIK DIPANKAR ,  MAJETI BHARAT KUMAR ,  CHAUDHURI ARABINDA ,  SISTLA RAMAKRISHNA

IPC: C07C235/40 ,  A61K31/16 ,  A61K48/00

CPC classification number: A61K48/0008 ,  A61K2039/53 ,  C07C235/40 ,  C12N15/88

Abstract: The present invention provides a novel series of glycomimicking cationic amphiphiles containing quinic acid head-groups and methods for preparing the said cationic amphiphiles. The invention provides novel compositions containing the said amphiphiles with remarkable gene transfer properties. Furthermore, the present invention provides methods for producing immune response in mice using the above-mentioned formulations containing the said cationic amphiphiles and genetic materials encoding immunogenic antigens. The area of medical science that is likely to benefit most from the present invention is genetic immunization.

Abstract translation: 本发明提供了含有奎尼酸头基的新型一系列含有羟基乙二醇的阳离子两亲物和制备所述阳离子两亲物的方法。 本发明提供含有具有显着基因转移性质的所述两亲物的新型组合物。 此外，本发明提供使用上述含有所述阳离子两亲物和编码免疫原性抗原的遗传物质的制剂在小鼠中产生免疫应答的方法。 可能受益于本发明的医学领域是遗传免疫。

公开(公告)号：WO2021183564A1

公开(公告)日：2021-09-16

申请号：PCT/US2021/021573

申请日：2021-03-09

Applicant: ARCTURUS THERAPEUTICS, INC. ,  SULLIVAN, Sean Michael ,  MATSUDA, Daiki ,  TACHIKAWA, Kiyoshi ,  CHIVUKULA, Padmanabh ,  KARMALI, Priya Prakash ,  DAVIS, Jared Henry ,  BAO, Yanjie

Inventor： SULLIVAN, Sean Michael ,  MATSUDA, Daiki ,  TACHIKAWA, Kiyoshi ,  CHIVUKULA, Padmanabh ,  KARMALI, Priya Prakash ,  DAVIS, Jared Henry ,  BAO, Yanjie

IPC: A61K39/00 ,  C07K14/18 ,  C12N15/86

Abstract: The present disclosure provides a nucleic acid molecule comprising a first polynucleotide encoding one or more viral replication proteins, wherein the first polynucleotide is codon-optimized as compared to a wild-type polynucleotide encoding the one or more viral replication proteins; and a second polynucleotide comprising a first transgene encoding a first antigenic protein or a fragment thereof. In some embodiments, the one or more viral replication proteins may be alphavirus proteins or rubivirus proteins.