公开(公告)号：US20240325493A1

公开(公告)日：2024-10-03

申请号：US18623995

申请日：2024-04-01

申请人： Washington University

发明人： Kyunghee Choi ,  Karen Krchma ,  Ashraf Ul Kabir

IPC分类号： A61K38/18 ,  A61K39/395 ,  A61K48/00 ,  A61P35/00 ,  C12Q1/6886

CPC分类号： A61K38/18 ,  A61K39/39558 ,  A61P35/00 ,  C12Q1/6886 ,  A61K48/00 ,  C12Q2600/106 ,  C12Q2600/158

摘要： Among the various aspects of the present disclosure is the provision of a regulator of tumor cell functions and uses thereof. Methods to treat a cancer patient with immunotherapy and a Zbtb46 modulation agent are disclosed. A method to identify a cancer patient in need of an adjuvant therapy to an immunotherapy is also disclosed.

公开(公告)号：US20240316216A1

公开(公告)日：2024-09-26

申请号：US18698888

申请日：2022-10-06

申请人： Massachusetts Institute of Technology

发明人： Robert S. Langer ,  Li-Huei Tsai ,  Owen Shea Fenton ,  Joel Blanchard ,  Jason Andresen ,  Julia Bonner ,  William Ralvenius

IPC分类号： A61K48/00 ,  A61K47/14 ,  A61K47/28

CPC分类号： A61K48/0033 ,  A61K47/14 ,  A61K47/28 ,  A61K48/0058

摘要： Provided herein are methods of selectively delivering an agent to a cell, comprising contacting the cell with a composition comprising an agent and lipids selected from: (a) an ionizable amino lipid, (b) a sterol, (c) a phospholipid, and (d) a PEG-lipid. The methods are selective for delivery to microglia over other neuroglia, such as astrocytes.

公开(公告)号：US20240316200A1

公开(公告)日：2024-09-26

申请号：US18611477

申请日：2024-03-20

申请人： Lung Biotechnology PBC

发明人： Colin EXLINE ,  Jintang DU ,  Sean STEVENS

IPC分类号： A61K39/00 ,  A61K48/00 ,  A61P35/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC分类号： A61K39/464471 ,  A61K39/4611 ,  A61K39/4613 ,  A61K39/4614 ,  A61K39/4615 ,  A61K39/4631 ,  A61K48/0033 ,  A61P35/00 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  A61K2039/575 ,  C12N2840/203

摘要： The present disclosure relates to bicistronic polypeptide constructs for use in allogeneic gene therapy, for example, CAR-T cell therapy. The bicistronic constructs comprise first polynucleotide encoding a therapeutic molecule (e.g., a CAR-T or an antibody) and a second polynucleotide encoding an Immune Surveillance Masking Molecule (ISMM). The ISMM comprises a human leukocyte antigen-E genetically fused to a non-functional version, e.g., a fragment, of the protein knock-out by the insertion of the bicistronic construct, e.g., beta-2 microglobulin or B2M. Also provided are vectors comprising the bicistronic constructs, cells (e.g., CAR-T cells), and methods of use. Also provided are kits and articles of manufacture. The present disclosure also provides four novel insertion sites that can be used to insert an expression construct in the B2M gene.

公开(公告)号：US20240309382A1

公开(公告)日：2024-09-19

申请号：US18660147

申请日：2024-05-09

申请人： Avidity Biosciences, Inc.

发明人： Barbora MALECOVA ,  Rob BURKE ,  David SALA CANO ,  Beatrice Diana DARIMONT

IPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/68 ,  A61K48/00 ,  A61P21/00 ,  A61P21/06 ,  C07K16/28 ,  C07K19/00

CPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/6807 ,  A61K47/6849 ,  A61K47/6889 ,  A61K48/005 ,  A61P21/00 ,  A61P21/06 ,  C07K16/2881 ,  C07K19/00 ,  C07K2317/55 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3513 ,  C12N2320/32

摘要： Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

公开(公告)号：US20240309349A1

公开(公告)日：2024-09-19

申请号：US18577942

申请日：2022-07-08

申请人： ACRIGEN BIOSCIENCES

发明人： David Rabuka ,  Allison Sharrar ,  Michael Schelle ,  Luisa Mayumi Arake De Tacca ,  Mia Christina Pulos-Holmes

IPC分类号： C12N9/22 ,  A61K48/00 ,  C07K16/28 ,  C12N5/0783 ,  C12N15/11 ,  C12N15/82 ,  C12N15/90

CPC分类号： C12N9/22 ,  A61K48/005 ,  C07K16/2803 ,  C12N5/0636 ,  C12N15/11 ,  C12N15/8213 ,  C12N15/907 ,  C07K2317/53 ,  C07K2319/03 ,  C07K2319/09 ,  C12N2310/20 ,  C12N2510/00 ,  C12N2800/80

摘要： The present disclosure provides nucleases and compositions, methods, and systems thereof for nucleic acid modification. More particularly, the present disclosure provides compositions and system comprising a nuclease comprising an amino acid sequence having at least 70% identity, at least 80% identity, at least 90% identity, at least 95% identity or at least 99% identity to any of SEQ ID NOs: 1-23 or 32-35 and at least one gRNA.

公开(公告)号：US20240307559A1

公开(公告)日：2024-09-19

申请号：US18578623

申请日：2022-07-13

申请人： MeiraGTx UK II Limited

发明人： Anastasios Georgiadis

IPC分类号： A61K48/00 ,  A61K38/17 ,  C07K14/705 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K38/177 ,  C07K14/705 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008

摘要： Provided herein are expression constructs, viral genomes, and vectors for the expression of Kv8.2, as well as pharmaceutical compositions comprising the vectors disclosed herein. Also provided are methods of using the expression constructs and vectors disclosed herein, including methods of treating a retinal disease in a subject in need thereof, wherein the retinal disease is associated with one or more mutations in the KCNV2 gene, the method comprising administering to the subject a vector disclosed herein.

公开(公告)号：US20240307558A1

公开(公告)日：2024-09-19

申请号：US18269582

申请日：2019-12-20

申请人： CELL AND GENE THERAPY LTD ,  OBSCHESTVO S OGRANICHENNOI OTVETSTVENNOSTJU "PRORYVNYE INNOVATSIONNYE TEKHNOLOGII"

发明人： Natalia SAVELIEVA

IPC分类号： A61K48/00 ,  C07K14/47 ,  C07K14/78 ,  C12N15/85

CPC分类号： A61K48/0058 ,  C07K14/4741 ,  C07K14/78 ,  C12N15/85 ,  C12N2800/107

摘要： The invention refers to genetic engineering and can be used in biotechnology, medicine, and agriculture for the manufacture of gene therapy products. Gene therapy DNA vector based on the gene therapy DNA vector VTvaf1V carrying the therapeutic gene selected from the group of KRT5, KRT14, LAMB 3, and COL7A1 genes was constructed in order to increase the expression level of this therapeutic gene in humans and animals, while gene therapy DNA vector VTvaf17-KRT5, or VTvaf17-KRT14, or VTvaf17-LAMB3, or VTvaf17-COL7A1 has the nucleotide sequence SEQ ID No. 1, or SEQ ID No. 2, or SEQ ID No. 3, or SEQ ID No. 4, respectively. The gene therapy DNA vector contains no nucleotide sequences of viral origin and no antibiotic resistance genes, which ensures its safe use for gene therapy in humans and animals. A method of obtaining the specified vector, the use of the vector, a strain of Escherichia coli carrying the specified vector, and a method of industrial production of the specified vector are also provided.

公开(公告)号：US20240307556A1

公开(公告)日：2024-09-19

申请号：US18002567

申请日：2021-07-02

申请人： Maritime Therapeutics, Inc.

发明人： Michael Heartlein

IPC分类号： A61K48/00 ,  C12N15/90

CPC分类号： A61K48/005 ,  C12N15/907 ,  C12N2800/22

摘要： The present invention provides, among other things, mRNA constructs and compositions and methods for reverse gene therapy, including administering to a subject in need of treatment a mRNA gene transfer construct comprising a reverse complement sequence encoding a protein of interest and a sequence encoding a human L1 retro-element.

公开(公告)号：US20240307514A1

公开(公告)日：2024-09-19

申请号：US18576078

申请日：2022-07-01

申请人： Dignity Health ,  Board of Trustees of Michigan State University ,  Van Andel Research Institute

发明人： Fredric Manfredsson ,  Lena Brundin

IPC分类号： A61K38/51 ,  A61K48/00 ,  A61P25/28 ,  C12N9/88 ,  C12N15/86

CPC分类号： A61K38/51 ,  A61K48/0058 ,  A61P25/28 ,  C12N9/88 ,  C12N15/86 ,  C12Y401/01045 ,  C12N2750/14143

摘要： Compositions for increasing the expression of ACMSD in a target cell and methods of using the compositions to provide a general protective effect in a cell or tissue in a subject, or protect a subject from or treating a disease condition associated with inflammation, oxidative stress, protein aggregation, energy failure, toxic exposure such as exposure to pollutants, or any combination thereof.

公开(公告)号：US12091675B2

公开(公告)日：2024-09-17

申请号：US17296944

申请日：2019-07-15

申请人： Takeda Pharmaceutical Company Limited

发明人： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC分类号： C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

CPC分类号： C12N15/861 ,  A61K38/37 ,  A61K48/0083 ,  C07K14/755 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0381

摘要： The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.