公开(公告)号：WO2023077012A1

公开(公告)日：2023-05-04

申请号：PCT/US2022/078798

申请日：2022-10-27

申请人： REGENERON PHARMACEUTICALS, INC.

发明人： SABIN, Leah ,  KYRATSOUS, Christos ,  PEFANIS, Evangelos ,  MOLLER-TANK, Sven ,  KATAKOWSKI, Joseph ,  BAIK, Andrew ,  CYGNAR, Katherine ,  SAMAI, Poulami ,  CALAFATI, Philip

IPC分类号： C07K14/745 ,  A61K38/36

摘要： Nucleic acid constructs and compositions that allow insertion of a FIX coding sequence into a target genomic locus such as an endogenous ALB locus and/or expression of the FIX coding sequence are provided. The nucleic acid constructs and compositions can be used in methods of introducing a F9 nucleic acid into a cell, methods of integration of a F9 nucleic acid into a target genomic locus, methods of expression of FIX in a cell, and in methods of treating hemophilia B or FIX deficiency in a subject.

公开(公告)号：WO2023044100A1

公开(公告)日：2023-03-23

申请号：PCT/US2022/043967

申请日：2022-09-19

申请人： REVIVICOR, INC.

发明人： KOKKINAKI, Maria ,  KURAVI, Kasinath V.

IPC分类号： A01K67/027 ,  C12N15/877 ,  C07K14/705 ,  C07K14/745 ,  C12N15/85 ,  C12N15/90

摘要： The present invention provides transgenic animals (e.g., transgenic porcine animals), organs, tissues, and cells derived from the transgenic animals that are particularly useful for xenotransplantation therapies. In particular, the present invention provides transgenic porcine animals, as well as organs, tissues and cells derived from the transgenic porcine animals, which lack any expression of a functional alpha 1,3 galactosyltransferase (GTKO) gene and comprise at least six transgenes under the control of at least three promoters within a single multi-gene expression vector, and further comprise at least four additional genetic modifications. Also provided are methods of making the transgenic animals (e.g., transgenic porcine animals), and methods of using the transgenic animals, organs, tissues, and cells derived from the transgenic animals for xenotransplantation therapies and treating a disease or condition.

公开(公告)号：WO2022123827A1

公开(公告)日：2022-06-16

申请号：PCT/JP2021/030430

申请日：2021-08-19

申请人： 株式会社血栓トランスレーショナルリサーチラボ THROMBO TRANSLATIONAL RESEARCH LAB INC. [JP]/[JP]

发明人： 神窪　勇一 KAMIKUBO, Yuichi

IPC分类号： C07K14/745 ,  G01N33/86

摘要： 出血性疾患における直接経口抗凝固薬（Ｄｉｒｅｃｔ　Ｏｒａｌ　Ａｎｔｉｃｏａｇｕｌａｎｔｓ：ＤＯＡＣｓ）などの出血のリスクを正確に判定、評価できる高感度の血液凝固検査試薬等を提供する。　活性型の血液凝固第ＸＩ因子（ＦＸＩａ）と、組織因子（ｔｉｓｓｕｅ　ｆａｃｔｏｒ，　ＴＦ）と、を含む血液凝固検査試薬。被験検体である生体試料と、０．５ｐＭ～１，０００ｐＭの活性型の血液凝固第ＸＩ因子と、１ｆＭ～１，０００ｆＭの組織因子とを含む検査試薬と、を反応させることで形成するトロンビンの量を測定する工程を有する、血液凝固検査方法。

公开(公告)号：WO2022023431A1

公开(公告)日：2022-02-03

申请号：PCT/EP2021/071181

申请日：2021-07-28

申请人： BIA SEPARATIONS D.O.O.

发明人： JOSIC, Djuro ,  BEGIC, Marija ,  ANDJELKOVIC, Uros

IPC分类号： C07K14/745 ,  C12N9/64 ,  B01D15/08 ,  B01D15/36 ,  B01D15/42 ,  A61K38/48 ,  A61K38/36

摘要： A process for the purification of Prothrombin Complex Concentrate (PCC) from complete plasma or cryo-poor plasma, in particular undiluted cryo-poor plasma, by means of chromatography using a monolithic stationary phase, the method comprising performing an initial sample displacement chromatography step of complete plasma or cryo-poor plasma on an anion exchanger to obtain a first fraction enriched in PCC.

公开(公告)号：WO2021163298A1

公开(公告)日：2021-08-19

申请号：PCT/US2021/017620

申请日：2021-02-11

申请人： HCW BIOLOGICS, INC.

发明人： WONG, Hing C. ,  SHRESHA, Niraj ,  GEORGE, Varghese ,  DEE, Michael

IPC分类号： C07K14/745 ,  C12N5/0783

摘要： Provided herein are methods of stimulating or increasing the proliferation of a Treg cell, compositions including a population of Treg cells generated by these methods, and methods of treating a subject using these compositions.

公开(公告)号：WO2020089619A1

公开(公告)日：2020-05-07

申请号：PCT/GB2019/053071

申请日：2019-10-30

申请人： UCL BUSINESS LTD

发明人： NATHWANI, Amit ,  PATEL, Nishil ,  GOMEZ, Keith

IPC分类号： A61P7/04 ,  A61K38/36 ,  A61K38/48 ,  C07K14/745 ,  C12N9/64

摘要： The present invention relates to modified Factor IX polypeptides comprising a mutation at a position corresponding to position 347 of wild type immature (precursor) Factor IX, polynucleotides encoding the polypeptides, and treatments utilising the polypeptides or polynucleotides.

公开(公告)号：WO2019246094A1

公开(公告)日：2019-12-26

申请号：PCT/US2019/037726

申请日：2019-06-18

申请人： PORTOLA PHARMACEUTICALS, INC.

发明人： CONLEY, Pamela B. ,  LU, Genmin ,  PRESTA, Leonard G. ,  CURNUTTE, John T.

IPC分类号： A61K38/36 ,  A61K38/48 ,  A61P7/02 ,  A61P39/02 ,  C07K14/745 ,  C12N9/64

摘要： The present disclosure relates to antidotes to anticoagulants targeting factor Xa. The antidotes are factor Xa protein derivatives that bind to the factor Xa inhibitors thereby substantially neutralizing them but do not assemble into the prothrombinase complex. In one embodiment, the derivatives described herein lack or have reduced intrinsic coagulant activity.

公开(公告)号：WO2019201868A1

公开(公告)日：2019-10-24

申请号：PCT/EP2019/059695

申请日：2019-04-15

申请人： SWEDISH ORPHAN BIOVITRUM AB (PUBL)

发明人： CORNVIK, Tobias ,  NILSSON, Joakim ,  NORDLING, Erik ,  SVENSSON GELIUS, Stefan

IPC分类号： A61K38/36 ,  C12N9/64 ,  C07K14/745 ,  C07K14/47 ,  A61K38/00

摘要： A fusion protein is provided, comprising i) a coagulation factor protein selected from coagulation factor X (FX), coagulation factor IX (FIX) and variants thereof; and ii) a half-life extending polypeptide moiety comprising 2-80 units independently selected the amino acid sequences according to SEQ ID NO: 1: in which, independently: X1 is P or absent; X2 is V or absent; X3 is P or T; X4 is P or T; X5 is T or V; X6 is D, G or T; X8 is A, Q or S; X9 is E, G or K; X10 is A, E P or T; and X11 is A, P or T. The half-life extending polypeptide moiety has a generally unfolded conformation and provides a fusion protein with a large hydrodynamic radius that may avoid renal clearance. As a result, the biological half-life of the fusion protein is increased and the biological effect of the FX or FIX may thus be prolonged.

公开(公告)号：WO2019150050A1

公开(公告)日：2019-08-08

申请号：PCT/FR2019/050223

申请日：2019-01-31

申请人： LABORATOIRE FRANCAIS DU FRACTIONNEMENT ET DES BIOTECHNOLOGIES

发明人： PLANTIER, Jean-Luc ,  ABACHE, Toufik

IPC分类号： C07K14/745 ,  A61P7/04

CPC分类号： C07K14/745 ,  A61P7/04 ,  C07K2319/30

摘要： La présente invention concerne des dimères de variants du facteur X, et leur utilisation pour le traitement des troubles de la coagulation sanguine.

公开(公告)号：WO2019084691A1

公开(公告)日：2019-05-09

申请号：PCT/CA2018/051389

申请日：2018-11-01

申请人： QUEEN'S UNIVERSITY AT KINGSTON ,  KINGSTON HEALTH SCIENCES CENTRE ,  EMORY UNIVERSITY ,  GEORGIA TECH RESEARCH CORPORATION

发明人： ZAKAS, Philip ,  LILLICRAP, David ,  DOERING, Christopher ,  GAUCHER, Eric ,  RADFORD, Caelan ,  SPENCER, Harold Trent

IPC分类号： C12N15/85 ,  A61K38/36 ,  A61P7/04 ,  C07K14/745 ,  C07K14/755 ,  C12N15/12 ,  C12N15/867

CPC分类号： C07K14/755 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0381 ,  A61K38/00 ,  A61K48/005 ,  A61P7/04

摘要： A recombinant von Willebrand Factor (VWF) protein comprising one or more mutations and uses thereof are described. A recombinant protein complex comprising a von Willebrand factor sequence and one artificial Factor VIII sequence is described. A recombinant protein complex comprising a Factor VIII sequence and one artificial von Willebrand sequence is described. A recombinant protein complex is described that includes one artificial von Willebrand factor sequence and one artificial Factor VIII sequence. Also described are nucleic acid sequences and a vector encoding a VWF sequence and a pharmaceutical composition for inducing blood clotting that includes a VWF protein.