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1. 发明申请

WO2023283403A2 BIS-RNAI COMPOUNDS FOR CNS DELIVERY 审中-公开
公开(公告)号：WO2023283403A2
公开(公告)日：2023-01-12
申请号：PCT/US2022/036455
申请日：2022-07-08
申请人： ALNYLAM PHARMACEUTICALS, INC.
发明人： ZLATEV, Ivan , THEILE, Christopher, S. , MANOHARAN, Muthiah , LENTINI, Scott, P. , MATSUDA, Shigeo , NAIR, Jayaprakash, K. , PENG, Haiyan
IPC分类号： C12N15/11 , A61K31/713 , A61K47/549 , C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3515 , C12N2310/3519 , C12N2310/352 , C12N2310/51 , C12N2320/31 , C12N2320/32
摘要： One aspect of the invention relates to molecules that target more than one target nucleic acid sequence and that exhibit efficacy in the tissues of the CNS of a subject upon contact. Another aspect of the present invention relates to a small circular interfering RNA (sciRNA) for modulating one or more target mRNAs in the central nervous system (CNS) of a subject. Other aspects of the invention relate a pharmaceutical composition, a method for inhibiting the expression of one or more target mRNAs in the CNS of a subject, and a method of treating or preventing a CNS disease or disorder in a subject using the molecules and sciRNA.

2. 发明申请

WO2021261995A1 COMBINATION OF AN ANTIBODY-DRUG CONJUGATE AND AN ANTIBODY-SAPONIN CONJUGATE 审中-公开
公开(公告)号：WO2021261995A1
公开(公告)日：2021-12-30
申请号：PCT/NL2021/050391
申请日：2021-06-22
申请人： SAPREME TECHNOLOGIES B.V.
发明人： POSTEL, Ruben , HERMANS, Guy
IPC分类号： A61K47/54 , A61K47/55 , A61P35/00 , A61K45/06 , A61K47/68 , A61K47/549 , A61K47/554 , A61K47/6845 , A61K47/6855
摘要： The invention relates to a therapeutic combination comprising: (a) a first pharmaceutical composition comprising a conjugate comprising a first binding molecule for binding to a first binding site of a cell- surface molecule and the conjugate comprising a saponin bound to said first binding molecule, wherein the saponin is a triterpene glycoside; and (b) a second pharmaceutical composition comprising a conjugate comprising a second binding molecule different from the first binding molecule, the second binding molecule comprising a second binding region different from the first binding region, for binding to a second binding site of said cell-surface molecule different from the first binding site of said cell- surface molecule, and the conjugate comprising an effector molecule covalently bound to said second binding molecule. The invention also relates to a pharmaceutical composition comprising said two conjugates. In addition, the invention relates to the pharmaceutical combination or the pharmaceutical composition of the invention for use as a medicament. Furthermore, the invention relates to the pharmaceutical combination or the pharmaceutical composition of the invention, for use in the treatment or prevention of a cancer, an autoimmune disease, a disease relating to (over)expression of a protein, a disease relating to an aberrant cell such as a tumor cell or a diseased liver cell, a disease relating to a mutant gene, a disease relating to a gene defect, a disease relating to a mutant protein, a disease relating to absence of a (functional) protein, a disease relating to a (functional) protein deficiency.

3. 发明申请

WO2021195467A2 RNAi AGENTS FOR INHIBITING EXPRESSION OF PNPLA3, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS OF USE 审中-公开
公开(公告)号：WO2021195467A2
公开(公告)日：2021-09-30
申请号：PCT/US2021/024299
申请日：2021-03-26
申请人： ARROWHEAD PHARMACEUTICALS, INC.
发明人： NICHOLAS, Anthony , PEI, Tao , XU, Zhao , SCHIENEBECK, Casi , DING, Zhi-Ming
IPC分类号： A61K31/713 , C12N15/113 , A61P3/06 , A61K47/549 , A61P1/16 , C12N15/1137 , C12N15/1138 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2310/351 , C12N2320/35
摘要： The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Also disclosed are pharmaceutical compositions that include PNPLA3 RNAi agents and methods of use thereof. The PNPLA3 RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the PNPLA3 RNAi agents in vivo provides for inhibition of PNPLA3 gene expression. The RNAi agents can be used in methods of treatment of PNPLA3 -related diseases and disorders, including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), hepatic fibrosis, and alcoholic or non-alcoholic liver diseases, including cirrhosis.

4. 发明申请

WO2021191686A1 TARGETED POLY(BETA-AMINO ESTER)S 审中-公开
公开(公告)号：WO2021191686A1
公开(公告)日：2021-09-30
申请号：PCT/IB2021/000181
申请日：2021-03-29
申请人： ARATINGA.BIO TNP
发明人： TEZGEL, Özgül , MOURLANE, Frederic , VAILLANT, Renaud , BAUCHE, Cécile
IPC分类号： A61K47/54 , A61K47/59 , A61K47/62 , A61K47/69 , A61K47/549 , A61K47/593 , A61K47/6935
摘要： Nontoxic, targeted poly beta- amino esters (PBAEs) are synthesized by using click chemistry to attach a targeting moiety. The click chemistry uses a dienophile, such as a strained alkene ring, and a diene, such as tetrazine, to provide rapid attachment of targeting moieties to PBAE polymers and nanoparticle surfaces containing them. Targeting moieties such as aptamers, antibodies or antibody-like proteins can be quickly and safely coupled to PBAEs to provide highly specific localization of nanoparticles for gene therapy or targeted delivery of therapeutics.

5. 发明申请

WO2021130266A1 PHARMACEUTICAL COMBINATION OF A THERAPEUTIC OLIGONUCLEOTIDE TARGETING HBV AND A TLR7 AGONIST FOR TREATMENT OF HBV 审中-公开
公开(公告)号：WO2021130266A1
公开(公告)日：2021-07-01
申请号：PCT/EP2020/087697
申请日：2020-12-22
申请人： F. HOFFMANN-LA ROCHE AG , HOFFMANN-LA ROCHE INC.
发明人： GAO, Lu , ZHU, Yonghong , OTTOSEN, Søren , MUELLER, Henrik , ZHOU, Xue , BLAISING, Julie Elisabeth Françoise , JIN, Yuyan , BO, Qingyan , TYAGI, Gaurav
IPC分类号： C12N15/113 , A61K31/4375 , A61K31/506 , A61K31/519 , A61K31/5377 , A61K31/554 , A61P31/12 , A61K31/7064 , A61K31/712 , A61P31/20 , A61K31/522 , A61K31/675 , A61K31/683 , A61K47/54 , A61K31/713 , A61K47/549 , C12N15/1131 , C12N2310/11 , C12N2310/14 , C12N2310/341 , C12N2320/31
摘要： The present invention is directed to compositions and methods for treating hepatitis B virus infection. In particular, the present invention is directed to a combination therapy comprising administration of a therapeutic oligonucleotide targeting HBV and a TLR7 agonist for use in the treatment of a chronic hepatitis B patient.

6. 发明申请

WO2021014026A2 BIOACTIVE SAPONIN LINKED TO A FUNCTIONAL MOIETY 审中-公开
公开(公告)号：WO2021014026A2
公开(公告)日：2021-01-28
申请号：PCT/EP2020/071145
申请日：2020-07-27
申请人： SAPREME TECHNOLOGIES B.V. , CHARITÉ - UNIVERSITÄTSMEDIZIN BERLIN
发明人： FUCHS, Hendrik , POSTEL, Ruben
IPC分类号： A61K47/54 , A61K47/55 , A61K47/59 , A61K47/64 , A61K47/549 , A61K47/593 , A61K47/595 , A61K47/60 , A61K47/6415 , A61K47/642 , A61K47/643 , A61K47/6807 , A61K47/6825 , A61K47/6849 , A61K47/6855 , A61K47/6885 , A61K47/6889
摘要： The invention relates to an endosomal and/or lysosomal escape enhancing conjugate comprising a saponin optionally linked to a targeting molecule such as an antibody and optionally linked to an effector molecule such as a toxinor an oligonucleotide. The invention also relates to a therapeutic combination of such an endosomal and/or lysosomal escape enhancing conjugate of the invention and a functionalized binding molecule comprising an effector molecule, wherein the endosomal and/or lysosomal escape enhancing conjugate comprises an enhancer of said effector molecule, i.e. a saponin. In particular the invention relates to such a therapeutic combination for use as a medicament, in particular for use in the treatment of a tumour. The invention further relates to a method of treating cancer or an autoimmune disease by administering an effective dose of the therapeutic combination to a patient in need thereof or by administering an effective dose of the endosomal and/or lysosomal escape enhancing conjugate comprising a saponin complexed with a targeting molecule such as an immunoglobulin specific for a tumor-cell surface molecule and complexed with an effector molecule, to a patient in need thereof. The invention also relates to a functionalized saponin with endosomal/lysosomal escape enhancing activity.

7. 发明申请

WO2022271806A1 NOVEL THERAPEUTIC DELIVERY MOIETIES AND USES THEREOF 审中-公开
公开(公告)号：WO2022271806A1
公开(公告)日：2022-12-29
申请号：PCT/US2022/034500
申请日：2022-06-22
申请人： ELI LILLY AND COMPANY
发明人： ANTONELLIS, Patrick Joseph , LACKNER, Gregory Lawrence , WILSON, Takako
IPC分类号： C12N15/113 , A61K47/54 , C12N15/87 , A61K47/549 , C12N15/111 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/351 , C12N2320/32 , C12N2330/30
摘要： The present invention relates to novel compounds comprising novel delivery moieties for delivery of oligonucleotides, which are useful in the treatment of disease, suitably diseases of the liver.

8. 发明申请

WO2022268864A1 GRIFFITHSIN FOR USE IN A METHOD OF PREVENTING OR TREATING INFECTIONS WITH RESPIRATORY VIRUSES 审中-公开
公开(公告)号：WO2022268864A1
公开(公告)日：2022-12-29
申请号：PCT/EP2022/066974
申请日：2022-06-22
申请人： SOLMIC BIOTECH GMBH
发明人： FURCH, Marcus , BESECKE, Karen F. W. , PANNEM, Rajeswara Rao
IPC分类号： A61K47/69 , A61K47/54 , A61K38/16 , A61K9/127 , A61K9/107 , A61K9/51 , A61P31/14 , A61P31/16 , A61K38/168 , A61K47/549 , A61K47/6911 , A61K9/0019 , A61K9/0021 , A61K9/0043 , A61K9/0048 , A61K9/0078 , A61K9/08 , A61K9/5123 , A61K9/513
摘要： The present invention relates to Griffithsin (GRFT) for use in a method of preventing or treating infections with respiratory viruses, wherein the GRFT is encapsulated. A preferred embodiment is a combination of targeted nanocarrier-mediated delivery of GRFT and non-encapsulated GRFT to GLR-positive cells infected with, or susceptible to infection with, a respiratory virus.

9. 发明申请

WO2022000884A1 基于甘露糖的mRNA靶向递送系统及其应用审中-公开
公开(公告)号：WO2022000884A1
公开(公告)日：2022-01-06
申请号：PCT/CN2020/123993
申请日：2020-10-27
申请人：深圳市瑞吉生物科技有限公司
发明人：胡勇 , 张苗苗
IPC分类号： A61K31/7115 , A61K31/7105 , A61K31/712 , C12N15/11 , A61K48/00 , A61P35/00 , A61K2039/53 , A61K39/0011 , A61K47/549 , A61K47/65 , A61P43/00
摘要：提供了一种基于甘露糖的mRNA靶向递送系统及其应用，其中mRNA可以编码一种或多种目标多肽并包含至少一个甘露糖修饰。通过对mRNA分子进行甘露糖修饰，从而使得mRNA可以与甘露糖直接偶联，在不需要载体的情况下实现mRNA的靶向递送。

10. 发明申请

WO2021260061A2 SAPONIN DERIVATIVES FOR USE IN MEDICINE 审中-公开
公开(公告)号：WO2021260061A2
公开(公告)日：2021-12-30
申请号：PCT/EP2021/067239
申请日：2021-06-23
申请人： SAPREME TECHNOLOGIES B.V. , CHARITÉ - UNIVERSITÄTSMEDIZIN BERLIN
发明人： POSTEL, Ruben , HERMANS, Guy , FUCHS, Hendrik
IPC分类号： A61K47/54 , A61P43/00 , A61K47/68 , A61K47/549 , A61K47/6807
摘要： The invention relates to a Quillaja saponaria saponin derivative based on a saponin comprising a triterpene aglycone and a first saccharide chain and/or a second saccharide chain, and comprising: an aglycone core structure comprising an aldehyde group which has been derivatised; and/or the first saccharide chain wherein the first saccharide chain comprises a carboxyl group, which has been derivatised; and/or the second saccharide chain wherein the second saccharide chain comprises at least one acetoxy group which has been derivatised. The invention also relates to a first pharmaceutical composition comprising the saponin derivative of the invention. In addition, the invention relates to a pharmaceutical combination comprising the first pharmaceutical composition of the invention and a second pharmaceutical composition comprising any one or more of an antibody-toxin conjugate, a receptor-ligand – toxin conjugate, an antibody-drug conjugate, a receptor-ligand – drug conjugate, an antibody-oligonucleotide conjugate or a receptor-ligand – oligonucleotide conjugate. The invention also relates to the first pharmaceutical composition or the pharmaceutical combination of the invention, for use as a medicament, or use in the treatment or prophylaxis of a cancer, an infectious disease, viral infection, hypercholesterolemia, primary hyperoxaluria, haemophilia A, haemophilia B, alpha-1 antitrypsin related liver disease, acute hepatic porphyria, transthyretin-mediated amyloidosis, or an autoimmune disease. Furthermore, the invention relates to an in vitro or ex vivo method for transferring a molecule from outside a cell to inside said cell, comprising contacting said cell with the molecule and with a saponin derivative of the invention.

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