公开(公告)号：US20240002863A1

公开(公告)日：2024-01-04

申请号：US18338966

申请日：2023-06-21

申请人： ENZERNA BIOSCIENCES, INC.

发明人： Joseph RUIZ ,  Zefeng WANG

IPC分类号： C12N15/117 ,  C12N15/86 ,  A61P25/00

CPC分类号： C12N15/117 ,  C12N15/86 ,  A61P25/00

摘要： The present disclosure provides compositions comprising synthetic site-specific RNA editing entities engineered to target pathogenic RNA comprising a CAG repeat associated with a CAG repeat disorder. Also disclosed herein are methods of treating the CAG repeat disorders of the present disclosure, such as Huntington's disease with the compositions and pharmaceutical formulations comprising the compositions disclosed herein.

公开(公告)号：US11851660B2

公开(公告)日：2023-12-26

申请号：US18065267

申请日：2022-12-13

申请人： GLAXOSMITHKLINE BIOLOGICALS SA

发明人： Andrew Geall

IPC分类号： C12N15/117 ,  C12N15/86 ,  A61K39/155 ,  A61K39/39 ,  A61K39/12 ,  A61P31/12 ,  A61P37/04 ,  A61K48/00 ,  A61K39/00

CPC分类号： C12N15/117 ,  A61K39/12 ,  A61K39/155 ,  A61K39/39 ,  A61K48/0041 ,  A61K48/0083 ,  A61P31/12 ,  A61P37/04 ,  C12N15/86 ,  A61K2039/53 ,  A61K2039/552 ,  A61K2039/55555 ,  A61K2039/55566 ,  C12N2760/18534 ,  C12N2770/36143 ,  C12N2820/60

摘要： RNA encoding an immunogen is delivered to a large mammal at a dose of between 2 μg and 100 μg. Thus the invention provides a method of raising an immune response in a large mammal, comprising administering to the mammal a dose of between 2 μg and 100 μg of immunogen-encoding RNA. Similarly, RNA encoding an immunogen can be delivered to a large mammal at a dose of 3 ng/kg to 150 ng/kg. The delivered RNA can elicit an immune response in the large mammal.

公开(公告)号：US20230374523A1

公开(公告)日：2023-11-23

申请号：US18027895

申请日：2021-09-21

申请人： TriSalus Life Sciences, Inc.

发明人： Steven C. Katz ,  Bryan F. Cox ,  David Benjamin Jaroch

IPC分类号： C12N15/117 ,  A61K39/395 ,  A61K39/39 ,  A61P35/00

CPC分类号： C12N15/117 ,  A61K39/39558 ,  A61K39/39 ,  A61P35/00 ,  A61K2039/54

摘要： Embodiments of the present invention provide for methods of treating cancer and methods of delivering toll-like receptor (TLR) agonists to solid tumors in the pancreas using a locoregional therapy through the vasculature. In one aspect, the present invention relates to a method of treating pancreatic cancer comprising administering TLR agonists to the pancreas.

公开(公告)号：US11801314B2

公开(公告)日：2023-10-31

申请号：US17840967

申请日：2022-06-15

申请人： The Trustees of the University of Pennsylvania

发明人： Katalin Kariko ,  Drew Weissman

IPC分类号： C07H21/04 ,  A61K48/00 ,  C07K14/47 ,  C07K14/505 ,  C12N15/117 ,  C12N15/67 ,  C12N5/071 ,  C12N5/074 ,  C07H21/02 ,  C12Q1/68 ,  C12N15/85 ,  A61K38/17 ,  A61K38/18 ,  A61K38/50

CPC分类号： A61K48/0066 ,  A61K38/177 ,  A61K38/1816 ,  A61K38/50 ,  A61K48/005 ,  A61K48/0041 ,  A61K48/0075 ,  C07H21/02 ,  C07K14/47 ,  C07K14/4712 ,  C07K14/505 ,  C12N5/0602 ,  C12N5/0696 ,  C12N15/117 ,  C12N15/67 ,  C12N15/85 ,  C12Q1/68 ,  C12Y305/04004 ,  A61K48/00 ,  C12N2310/17 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2501/40 ,  C12N2800/95

摘要： This invention provides RNA, oligoribonucleotide, and polyribonucleotide molecules comprising pseudouridine or a modified nucleoside, gene therapy vectors comprising same, methods of synthesizing same, and methods for gene replacement, gene therapy, gene transcription silencing, and the delivery of therapeutic proteins to tissue in vivo, comprising the molecules. The present invention also provides methods of reducing the immunogenicity of RNA, oligoribonucleotide, and polyribonucleotide molecules.

公开(公告)号：US20230265214A1

公开(公告)日：2023-08-24

申请号：US18043534

申请日：2021-08-31

申请人： YALE UNIVERSITY ,  GENNAO BIO, INC.

发明人： Elias Quijano ,  Peter Glazer ,  Bruce C. Turner ,  Audrey Turchick ,  W. Mark Saltzman

IPC分类号： C07K16/40 ,  C12N15/117 ,  A61P35/00

CPC分类号： C07K16/40 ,  C12N15/117 ,  A61P35/00 ,  C07K2317/565 ,  C07K2317/622 ,  C07K2317/24

摘要： Compositions and methods of use thereof for delivering nucleic acid cargo into cells are provided. The compositions typically include (a) a 3E10 monoclonal antibody or an antigen binding, cell-penetrating fragment thereof; a monovalent, divalent, or multivalent single chain variable fragment (scFv); or a diabody; or humanized form or variant thereof, and (b) a nucleic acid cargo including, for example, a nucleic acid encoding a polypeptide, a functional nucleic acid, a nucleic acid encoding a functional nucleic acid, or a combination thereof. Elements (a) and (b) are typically non-covalently linked to form a complex.

公开(公告)号：US20230111669A1

公开(公告)日：2023-04-13

申请号：US18080164

申请日：2022-12-13

申请人： GLAXOSMITHKLINE BIOLOGICALS SA

发明人： Andrew GEALL

IPC分类号： C12N15/117 ,  C12N15/86 ,  A61K39/155 ,  A61K39/39 ,  A61K39/12 ,  A61P31/12 ,  A61P37/04 ,  A61K48/00

摘要： RNA encoding an immunogen is delivered to a large mammal at a dose of between 2 μg and 100 μg. Thus the invention provides a method of raising an immune response in a large mammal, comprising administering to the mammal a dose of between 2 μg and 100 μg of immunogen-encoding RNA. Similarly, RNA encoding an immunogen can be delivered to a large mammal at a dose of 3 μg/kg to 150 μg/kg. The delivered RNA can elicit an immune response in the large mammal.

公开(公告)号：US20230094580A1

公开(公告)日：2023-03-30

申请号：US17930042

申请日：2022-09-06

申请人： Wayne State University

发明人： Haipeng Liu ,  Chunsong Yu

IPC分类号： A61K39/39 ,  A61K47/54 ,  C12N15/117

摘要： Amphiphilic oligonucleotide conjugates that enhance adjuvant function are disclosed. The conjugates typically include: a lipophilic component, and conjugated thereto (directly or indirectly) an immunomodulating oligonucleotide that, if it were not conjugated to the lipophilic component, would suppress TLR7 and/or TLR8 stimulation. In the presence of albumin, these conjugates significantly enhance adjuvant function, in particular the function of TLR7/8-mediated adjuvants such as an imidazoquinolinamine. The conjugates can be administered, along with an adjuvant compound, to a subject in order to cause and/or enhance an immune response (for instance, to an infectious agent or a cancer antigen) in the subject.

公开(公告)号：US11583581B2

公开(公告)日：2023-02-21

申请号：US15760424

申请日：2016-09-21

申请人： GILEAD SCIENCES, INC.

发明人： Matthias Schroff ,  Manuel Schmidt ,  Kerstin Kapp ,  Alfredo Zurlo ,  Ole Schmeltz Sogaard ,  Martin Tolstrup ,  Rasmus Offersen

IPC分类号： A61K39/39 ,  A61K31/711 ,  C12N15/113 ,  C12N15/117 ,  A61K39/00

摘要： The invention relates to a non-coding sequence of deoxyribonucleic acids comprising at least one sequence motif N1N2CGN3N4, wherein N is a nucleotide comprising A, C, T, or G, and C is deoxycytidine, G is deoxyguanosine, A is deoxyadenosine and T is deoxy-thymidine for the treatment of viral infections. In particular, the non-coding sequence of deoxyribonucleic acids is used in combination with antiretroviral therapy and/or histone de-acetylase inhibitors.

公开(公告)号：US20220401467A1

公开(公告)日：2022-12-22

申请号：US17177111

申请日：2021-02-16

申请人： WAVE LIFE SCIENCES LTD.

发明人： Jason Jingxin Zhang ,  Naoki Iwamoto ,  Christopher J. Francis ,  Chandra Vargeese ,  David Charles Donnell Butler ,  Sethumadhavan Divakaramenon ,  Genliang Lu ,  Subramanian Marappan

IPC分类号： A61K31/7125 ,  A61P21/00 ,  C12N15/117 ,  A61K31/7088

摘要： The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.

公开(公告)号：US20220396796A1

公开(公告)日：2022-12-15

申请号：US17571893

申请日：2022-01-10

申请人： CureVac AG

发明人： Mariola FOTIN-MLECZEK ,  Aleksandra KOWALCZYK ,  Regina HEIDENREICH ,  Ulrike GNAD-VOGT ,  Ute KLINKHARDT ,  Katja FIEDLER

IPC分类号： C12N15/117 ,  A61K38/20 ,  A61K39/00 ,  A61P35/00

摘要： The present invention relates to RNA, particularly an immunostimulatory RNA (isRNA), a coding RNA or a combination thereof, for use in the treatment or prophylaxis of a disease, in particular a tumor and/or cancer disease. The present invention also provides pharmaceutical compositions, and a kit comprising the RNA(s). Further, the invention also comprises medical uses of the RNA(s) and compositions comprising the RNA(s).