公开(公告)号：US20240360433A1

公开(公告)日：2024-10-31

申请号：US18661004

申请日：2024-05-10

申请人： Beam Therapeutics Inc.

发明人： Tanggis BOHNUUD ,  Genesis LUNG

IPC分类号： C12N9/78 ,  A61K48/00 ,  C12N15/11

CPC分类号： C12N9/78 ,  A61K48/005 ,  C12N15/111 ,  C12Y305/04004 ,  C12Y305/04005

摘要： The invention of the disclosure features compositions and methods for treating hereditary angioedema by introducing one or more alterations to a kallikrein B1 (KLKB1) polynucleotide in a cell. In particular embodiments, the invention provides a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying a KLKB1 polynucleotide, where the modification is associated with reduced expression and/or reduced activity of the KLKB1 polypeptide encoded by the polynucleotide.

公开(公告)号：US20240358859A1

公开(公告)日：2024-10-31

申请号：US18766963

申请日：2024-07-09

申请人： 4D MOLECULAR THERAPEUTICS INC.

发明人： David H. Kirn ,  Melissa A. Kotterman ,  Peter Francis

IPC分类号： A61K48/00 ,  A61P27/02 ,  C12N9/10 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61P27/02 ,  C12N9/1085 ,  C12N15/86 ,  C12Y205/01059 ,  C12N2750/14143

摘要： The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.

公开(公告)号：US20240358853A1

公开(公告)日：2024-10-31

申请号：US18598746

申请日：2024-03-07

申请人： KOREA INSTITUTE OF SCIENCE AND TECHNOLOGY

发明人： Heh-In IM ,  Jin Hee BAE ,  Nazarii FRANKIV

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K38/17 ,  A61P25/24 ,  C12N15/86 ,  G01N33/68

CPC分类号： A61K48/005 ,  A61K9/0019 ,  A61K38/1709 ,  A61P25/24 ,  C12N15/86 ,  G01N33/6854 ,  G01N33/6893 ,  C12N2750/14143

摘要： According to the present disclosure, the relation between the MeCP2 expression level and depression in D2R neurons of the ventral striatum was first found, and it was confirmed that MeCP2 in D2R neurons of the ventral striatum was significantly decreased due to depressive symptoms, and depressive symptoms were improved when MeCP2 expression in the neurons was up-regulated. Accordingly, the expression level of MeCP2 in D2R neurons of the ventral striatum is provided as a biomarker for diagnosing depression and the up-regulation of the MeCP2 expression is provided as a treatment strategy for depression, and thus, it is expected to be useful as a development platform for drugs for preventing or treating depression in the future.

公开(公告)号：US20240358737A1

公开(公告)日：2024-10-31

申请号：US18771994

申请日：2024-07-12

申请人： Emory University ,  Kodikaz Therapeutic Solutions, Inc.

发明人： Leon BERNAL-MIZRACHI ,  Matthew Charles TINDALL

IPC分类号： A61K31/711 ,  A61K45/06 ,  A61K48/00 ,  A61K51/02

CPC分类号： A61K31/711 ,  A61K45/06 ,  A61K48/0058 ,  A61K51/02

摘要： The present disclosure provides nucleic acid sequences and nucleic acid delivery constructs comprising “zip code” sequence(s) that home, target, cross a cytoplasm, and/or cross a nuclear membrane of a target cell (e.g., a diseased cell such as a cancer cell) or cell population (e.g., tissue), and integration sequence(s) that allow for integration of at least a portion of such nucleic acid or nucleic acid delivery system into a genome of such target cell. The present disclosure also provides non-naturally occurring nucleic acid constructs and delivery systems comprising such Zip Code and integration sequences as well as one or more cargo molecules that may be coupled covalently or non-covalently to such nucleic acid constructs and systems. Further provided herein are methods of diagnosing and treating diseases such as cancer using the target cell specific nucleic acid constructs and systems described herein.

公开(公告)号：US12129485B2

公开(公告)日：2024-10-29

申请号：US17057973

申请日：2019-05-23

申请人： National University of Singapore

发明人： Dario Campana ,  Natasha Vinanica ,  Yi Tian Png ,  Takahiro Kamiya

IPC分类号： A61K48/00 ,  A61K35/17 ,  C07H21/04 ,  C07K14/705 ,  C07K14/725 ,  C07K16/28 ,  C12N5/0783 ,  C12N15/63 ,  A01K67/00

CPC分类号： C12N5/0636 ,  A61K35/17 ,  C07K14/70507 ,  C07K14/7051 ,  C07K14/70578 ,  C07K16/2806 ,  C12N5/0646 ,  C07K2317/622 ,  C07K2319/03 ,  C07K2319/04 ,  C12N2510/00

摘要： The present invention provides engineered immune cells comprising an anti-CD2 protein expression blocker (PEBL) and an anti-CD2 chimeric antigen receptor (CAR). In some embodiments, such engineered immune cells lack surface expression CD2. Also, provided herein are methods of using such cells in cancer therapies.

公开(公告)号：US12129471B2

公开(公告)日：2024-10-29

申请号：US15550954

申请日：2016-02-23

申请人： Vertex Pharmaceuticals Incorporated

发明人： Matthew Hebden Porteus

IPC分类号： C12N15/63 ,  A61K48/00 ,  A61P7/06 ,  C12N15/09 ,  C12N15/113

CPC分类号： C12N15/63 ,  A61K48/005 ,  A61P7/06 ,  C12N15/09 ,  C12N15/113 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2320/11

摘要： The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.

公开(公告)号：US20240352090A1

公开(公告)日：2024-10-24

申请号：US18650037

申请日：2024-04-29

申请人： NANJING UNIVERSITY ,  CENTER FOR EXCELLENCE IN MOLECULAR CELL SCIENCE ,  HUAZHONG AGRICULTURAL UNIVERSITY

发明人： Xianchi DONG ,  Wen LIU ,  Jianping DING ,  Yu SHI ,  Shutong XU ,  Jianbo XU

IPC分类号： C07K14/75 ,  A61K38/00 ,  A61K45/06 ,  A61K48/00 ,  A61P7/02 ,  C07K1/22 ,  C12N15/85

CPC分类号： C07K14/75 ,  A61K45/06 ,  A61K48/005 ,  A61P7/02 ,  C07K1/22 ,  C12N15/85 ,  A61K38/00 ,  C07K2319/00

摘要： Provided are a protein containing an amino acid sequence capable of binding to a peptide fragment of a substrate GPRP, a fibrinogen-like protein 1 (FGL1) containing the amino acid sequence, and a fibrinogen domain (FD) of the FGL1, wherein a single FD protein also has a function the same as or similar to that of the FGL1. The FGL1 and FD, which have different action mechanisms from existing drugs, can inhibit fibrin assembly in the process of thrombogenesis by means of competing for fibrin substrate binding pockets. The FGL1 and FD can have a stronger affinity to the substrate by means of mutation, thereby effectively enhancing a usage effect of drugs for treating thrombus.

公开(公告)号：US20240350674A1

公开(公告)日：2024-10-24

申请号：US18682359

申请日：2022-08-11

申请人： Solid Biosciences Inc.

发明人： Kristy Jean Brown ,  Jennifer Green

IPC分类号： A61K48/00 ,  C07K14/47 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K48/0033 ,  A61K48/0066 ,  C07K14/4707 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2750/14145

摘要： The invention described herein provides a microdystrophin-encoding, codon optimized polynucleotide with reduced CPG island, and use thereof in the treatment of muscular dystrophy such as DMD/BMD.

公开(公告)号：US20240350664A1

公开(公告)日：2024-10-24

申请号：US18252153

申请日：2021-11-19

申请人： INTAS PHARMACEUTICALS LTD.

发明人： Ajit GUPTA ,  Lakshmikanth GANDIKOTA

IPC分类号： A61K48/00 ,  A61K38/42 ,  A61P7/06 ,  C12N15/86

CPC分类号： A61K48/005 ,  A61K38/42 ,  A61P7/06 ,  C12N15/86 ,  C12N2740/15043 ,  C12N2740/15071 ,  C12N2830/001 ,  C12N2830/48 ,  C12N2830/50

摘要： The invention provides persistent expression of β-globin gene by using cell and/or gene therapy based administration of nucleotide sequence encoding β-globin gene to treat thalassemia and sickle cell anemia. Lentivirus (LV) based viral vector system containing an expression cassette of β-globin gene. Whereas, the lentivirus particle is packed with genes expressing functional β-globin gene in erythroid cell lineage specifically.

公开(公告)号：US20240350663A1

公开(公告)日：2024-10-24

申请号：US18684886

申请日：2022-08-21

申请人： JOINT STOCK COMPANY "BIOCAD"

发明人： Anna Nikolaevna STRELKOVA ,  Tatiana Evgenievna SHUGAEVA ,  Pavel Mikhailovich GERSHOVICH ,  Pavel Andreevich IAKOVLEV ,  Dmitry Valentinovich MOROZOV

IPC分类号： A61K48/00 ,  C07K14/005 ,  C12N15/86

CPC分类号： A61K48/0041 ,  C07K14/005 ,  C12N15/86 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14151

摘要： The present application relates to the fields of gene therapy and molecular biology. More specifically, the present invention relates to an isolated modified capsid protein VP1 from adeno-associated virus serotype 9 (AAV9) comprising one or more amino acid substitutions compared to the wild-type AAV9 capsid protein VP1, which substitutions increase the efficiency of production (assembly) of the vector based on recombinant adeno-associated virus serotype 9 (rAAV9), to a capsid and a vector based on the above VP1, as well as to uses thereof.