公开(公告)号：WO2022239001A1

公开(公告)日：2022-11-17

申请号：PCT/IL2022/050486

申请日：2022-05-10

申请人： YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD.

发明人： AMAL, Haitham ,  DOMB, Abraham Jacob

IPC分类号： A61K31/416 ,  A61K9/00 ,  A61K31/713 ,  A61K38/00 ,  A61K39/00 ,  A61K45/06 ,  A61K49/00 ,  A61P25/00 ,  A61P25/06 ,  A61P25/08 ,  A61P25/16 ,  A61P25/18 ,  A61P25/28 ,  A61P25/30 ,  A61P35/00 ,  A61K47/32 ,  A61K49/0008 ,  A61K9/0019 ,  A61K9/0043 ,  A61K9/0048 ,  A61K9/0085 ,  A61K9/06 ,  A61K9/1647 ,  A61K9/2013 ,  A61K9/4858 ,  A61K9/5123 ,  A61P43/00 ,  C12N15/1137 ,  C12N2310/14 ,  C12N2320/30

摘要： Method and compositions that comprise an agent that reduces nNOS activity and uses thereof in the treatment of a disease or condition in which a beneficial clinical effect is achieved by reduction in neuronal nitric oxide synthase (nNOS) activity are provided.

公开(公告)号：WO2021255492A1

公开(公告)日：2021-12-23

申请号：PCT/IB2020/001053

申请日：2020-12-07

申请人： YILDIZ TEKNIK ÜNIVERSITESI

发明人： DERMAN, Serap ,  MANSUROGLU, Banu ,  BOZKURT, Yagmur ,  ACAR, Tayfun ,  SAYAN POYRAZ, Fatma

IPC分类号： A61K9/00 ,  A61P35/00 ,  A61K9/51 ,  A61K45/06 ,  A61K35/644 ,  A61K31/216 ,  A61K9/0019 ,  A61K9/0085 ,  A61K9/5153

摘要： The present invention provides a cancer drug for use in the treatment of cancer in which caffeic acid phenethyl ester (CAPE) is encapsulated in poly [lactic-co-glycolic acid] (PLGA) to increase its solubility in aqueous media. Angiopep-2 is used as a specific ligand to increase biological activity. In an embodiment of the invention, PLGA is preferably in the form of nanoparticles, thereby ensuring better solubility.

公开(公告)号：WO2021113596A2

公开(公告)日：2021-06-10

申请号：PCT/US2020/063243

申请日：2020-12-04

申请人： SORRENTO THERAPEUTICS, INC.

发明人： BRESSON, Damien ,  ZHOU, Heyue ,  PEDROS, Christophe

IPC分类号： A61K39/00 ,  A61P35/02 ,  C07K16/28 ,  A61K2039/505 ,  A61K2039/507 ,  A61K2039/54 ,  A61K2039/545 ,  A61K9/0019 ,  A61K9/0085 ,  C07K16/2803 ,  C07K16/2887 ,  C07K16/2896 ,  C07K2317/21 ,  C07K2317/24 ,  C07K2317/33 ,  C07K2317/52 ,  C07K2317/54 ,  C07K2317/55 ,  C07K2317/622 ,  C07K2317/73 ,  C07K2317/732 ,  C07K2317/76 ,  C07K2317/92

摘要： The present disclosure provides compositions and methods comprising a first antibody comprising a fully human anti-CD47 antibody and a second antibody comprising an Fc portion that binds an Fcγ receptor on an effector cell. In various embodiments the anti-CD47 antibody used in the methods and compositions exhibits a low level of binding to red blood cells and does not induce hemagglutination even at high concentrations of antibody. In some embodiments, the second antibody comprises a tumor-targeting antibody including an antibody that binds CD20, PD-L1, CD38 or SLAMF7 antigens. The combination of the fully human anti-CD47 antibody and the second antibody can decrease cancer burden in a subject.

公开(公告)号：WO2022212648A1

公开(公告)日：2022-10-06

申请号：PCT/US2022/022751

申请日：2022-03-31

申请人： BIOGEN MA INC.

发明人： YILDIZ, Hasan, M. ,  BORNAPOUR, Mandana ,  PENG, Paul ,  PATIL, Vishwesh, A. ,  SIMLER, Brian, R. ,  KIESMAN, William, F.

IPC分类号： A61K9/00 ,  A61K9/51 ,  A61K47/50 ,  C12N15/00 ,  A61K47/59 ,  A61K9/0085 ,  A61K9/5153 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/345

摘要： Featured are polymeric nanocarriers (e.g., PLGA nanoparticles) with encapsulated nucleic acid (e.g., an antisense oligonucleotide) for delivery (e.g., intrathecally) to the central nervous system. These polymeric nanocarriers are useful in the treatment of central nervous system disorders. They are capable of delivering their cargo (e.g., an antisense oligonucleotide) in higher amounts, for a longer period of time, and into deeper regions of the brain than a free or unformulated antisense oligonucleotide. The efficient delivery and distribution of antisense oligonucleotides results in reducing the number of administrations and patient compliance and improves patient experience.

公开(公告)号：WO2021202621A2

公开(公告)日：2021-10-07

申请号：PCT/US2021/025019

申请日：2021-03-30

申请人： NEUBASE THERAPEUTICS, INC.

发明人： LY, Danith H. ,  THADKE, Shivaji A. ,  BATWAL, Ramesh U. ,  DI CARO, Valentina ,  STEPHAN, Dietrich A. ,  SOOTER, Letha J. ,  BACKENROTH, Samuel I.

IPC分类号： C12Q1/6848 ,  C12Q1/6853 ,  A61K38/00 ,  A61K47/02 ,  A61K47/12 ,  A61K47/542 ,  A61K47/645 ,  A61K51/0491 ,  A61K9/0019 ,  A61K9/0053 ,  A61K9/0085 ,  A61K9/0095 ,  A61K9/06 ,  A61K9/08 ,  A61K9/10 ,  A61K9/107 ,  A61K9/127 ,  A61K9/2004 ,  A61K9/4841 ,  A61P25/28 ,  C07K14/003 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/3181 ,  C12N2320/34 ,  G01N30/7233 ,  G01N33/6896

摘要： The present disclosure relates to compounds useful for the detection or modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of trinucleotide repeat disorders, which can include administration of oligonucleotide analogues that can bind pathogenic nucleotide repeats in DNA or RNA.

公开(公告)号：WO2020263639A1

公开(公告)日：2020-12-30

申请号：PCT/US2020/038050

申请日：2020-06-17

申请人： THE PENN STATE RESEARCH FOUNDATION

发明人： CHEN, Gong ,  WU, Zheng ,  GUO, Ziyuan ,  CHEN, Yuchen ,  PEI, Zifei

IPC分类号： A61P25/28 ,  C07K14/475 ,  C12N5/00 ,  C12N9/22 ,  A01K2227/105 ,  A01K2267/0318 ,  A61K38/02 ,  A61K38/1709 ,  A61K48/00 ,  A61K48/005 ,  A61K9/0085 ,  A61P43/00 ,  C12N15/86 ,  C12N15/907 ,  C12N2501/195 ,  C12N2506/08 ,  C12N2510/00 ,  C12N2750/14143 ,  C12N5/0619 ,  C12N5/0622

摘要： This document provides methods and materials for treating a mammal having Huntington's disease. For example, methods and materials for forming GABAergic neurons that are functionally integrated into the brain of a living mammal (e.g., a human) and/or for modifying one or both huntingtin (Htt) genes (or HTT RNAs or HTT polypeptides) present in a mammal with Huntington's disease are provided.