公开(公告)号：US20240158800A1

公开(公告)日：2024-05-16

申请号：US18359665

申请日：2023-07-26

申请人： The Medical College of Wisconsin, Inc.

发明人： Daniel M. Lipinski ,  Chris A. Reid

IPC分类号： C12N15/115 ,  A61K48/00 ,  C12N15/67

CPC分类号： C12N15/115 ,  A61K48/005 ,  A61K48/0075 ,  C12N15/67 ,  C12N2310/141 ,  C12N2310/16 ,  C12N2310/3519 ,  C12N2320/50 ,  C12N2750/14143 ,  C12N2830/00

摘要： The present invention provides constructs comprising modified riboswitches to regulate expression of a transgene within a subject. Methods of treating a disease, specifically an eye disease, are also contemplated.

公开(公告)号：US11980672B2

公开(公告)日：2024-05-14

申请号：US17844877

申请日：2022-06-21

申请人： ModernaTX, Inc.

发明人： David H. Burkhardt ,  Romesh R. Subramanian ,  Christian Cobaugh

IPC分类号： A61K48/00 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P1/16 ,  A61P31/14 ,  C12N15/10 ,  C12N15/67 ,  C12Q1/68

CPC分类号： A61K48/0058 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K48/0066 ,  A61P1/16 ,  A61P31/14 ,  C12N15/1086 ,  C12N15/67 ,  C12N2840/00

摘要： mRNAs containing an exogenous open reading frame (ORF) flanked by a 5′ untranslated region (UTR) and a 3′ UTR is provided, wherein the 5′ and 3′ UTRs are derived from a naturally abundant mRNA in a tissue. Also provided are methods for identifying the 5′ and 3′ UTRs, and methods for making and using the mRNAs.

公开(公告)号：US11970700B1

公开(公告)日：2024-04-30

申请号：US18065194

申请日：2022-12-13

申请人： INTRON BIOTECHNOLOGY, INC.

发明人： Seong Jun Yoon ,  An Sung Kwon ,  Se Hwan Kim ,  Sang Hyeon Kang

IPC分类号： C12N15/67 ,  C12P21/02

CPC分类号： C12N15/67 ,  C12P21/02

摘要： The present invention relates to an intracellular expression enhancing genetic element, which enhances the intracellular expression of target protein encoded in RNA therapeutics such as mRNA vaccines and RNA medicines. In more detail, the present invention relates to the RNA molecule which has the genetic element capable of enhancing the intracellular expression of target protein encoded in RNA therapeutics delivered into cells; the plasmid used to synthesize the RNA molecule having the genetic element “E3”; the method for synthesizing RNA molecule having the genetic element “E3” using the plasmid and utilizing the synthesized RNA molecule as RNA therapeutics.

公开(公告)号：US20240122959A1

公开(公告)日：2024-04-18

申请号：US18543966

申请日：2023-12-18

申请人： Flagship Pioneering Innovations VI, LLC

发明人： Avak KAHVEJIAN ,  Alexandra Sophie De Boer ,  Nicholas McCartney Plugis ,  Erica Gabrielle Weinstein ,  Sebastian Trousil ,  Morag Helen Stewart ,  Ki Young Paek ,  Catherine Cifuentes-Rojas

IPC分类号： A61K31/7088 ,  C07K7/00 ,  C07K7/06 ,  C12N15/67 ,  C12P21/00

CPC分类号： A61K31/7088 ,  C07K7/00 ,  C07K7/06 ,  C12N15/67 ,  C12P21/00

摘要： This invention relates generally to pharmaceutical compositions and preparations of circular polyribonucleotides and uses thereof.

公开(公告)号：US20240033379A1

公开(公告)日：2024-02-01

申请号：US18045805

申请日：2022-10-11

申请人： Moderna TX, Inc.

发明人： Jason P. SCHRUM ,  Suhaib SIDDIQI ,  Kennechi EJEBE

IPC分类号： A61K48/00 ,  C12N15/113 ,  C07H21/02 ,  C07H19/10 ,  C12P21/00 ,  C12N5/071 ,  C12N15/10 ,  C12N15/11 ,  C07K16/00 ,  C07K16/28 ,  G01N33/559 ,  C12N15/67

CPC分类号： A61K48/0066 ,  C12N2310/335 ,  C07H21/02 ,  C07H19/10 ,  C12P21/00 ,  C12N5/0602 ,  C12N15/102 ,  C12N15/11 ,  C07K16/00 ,  C07K16/2887 ,  G01N33/559 ,  C12N15/1136 ,  C12N15/67 ,  C07K2317/24 ,  C12N2310/3341 ,  C12N15/1138

摘要： The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using thereof.

公开(公告)号：US11873496B2

公开(公告)日：2024-01-16

申请号：US16476868

申请日：2018-01-09

申请人： Whitehead Institute for Biomedical Research

发明人： Richard A. Young ,  Abraham S. Weintraub ,  Charles H. Li ,  Alla A. Sigova

IPC分类号： C07H21/02 ,  C12N15/67 ,  C07K14/47 ,  C12N15/113 ,  G01N33/50 ,  C12N15/85

CPC分类号： C12N15/67 ,  C07K14/4702 ,  C12N15/113 ,  C12N15/85 ,  G01N33/5023 ,  C12N2015/859 ,  C12N2310/51 ,  C12N2310/53 ,  C12N2830/001 ,  C12N2830/15

摘要： The invention relates to methods of modulating the expression of one or more genes in a cell by modulating the multimerization of a transcription factor and/or modulating the formation of enhancer-promoter DNA loops, and thereby modulating the expression of the one or more genes. The invention also relates to treating diseases and conditions involving aberrant gene expression by modulating the multimerization of a transcription factor and/or modulating the formation of enhancer-promoter DNA loops. The invention also relates to methods for screening for compounds that modulate expression of one or more genes in a cell.

公开(公告)号：US20240002862A1

公开(公告)日：2024-01-04

申请号：US18355035

申请日：2023-07-19

申请人： MeiraGTx UK II Limited

发明人： Xuecui Guo

IPC分类号： C12N15/115 ,  C12N15/11 ,  C12N15/67

CPC分类号： C12N15/115 ,  C12N15/111 ,  C12N15/67 ,  C12N2310/126 ,  C12N2310/16 ,  C12N2310/3519 ,  C12N2310/531

摘要： The present disclosure provides polynucleotide constructs for the modulation of target gene expression by aptamer-mediated ribonuclease cleavage of the target gene RNA and methods of using the constructs to modulate gene expression in response to the presence or absence of a ligand that binds the aptamer. The polynucleotide constructs contains a ribonuclease substrate sequence (e.g., an RNase P substrate) and a riboswitch comprising an effector region and an aptamer such that when the aptamer binds a ligand, target gene expression occurs.

公开(公告)号：US20230357780A1

公开(公告)日：2023-11-09

申请号：US18317915

申请日：2023-05-15

申请人： CALIFORNIA INSTITUTE OF TECHNOLOGY

发明人： Arash FARHADI ,  Gabrielle H HO ,  Daniel P SAWYER ,  Mikhail SHAPIRO ,  Robert C. HURT ,  Mengtong DUAN

IPC分类号： C12N15/67 ,  C07K14/195

CPC分类号： C12N15/67 ,  C07K14/195 ,  C12N2510/02

摘要： Provided herein are genetically engineered gas vesicle expression systems (GVES) that are configured to express gas vesicles (GVs) in a mammalian cell, related gas vesicle polynucleotide constructs, gas vesicle reporting genetic circuits, vectors, genetically engineered mammalian cells, non-human mammalian hosts, compositions, methods and systems, which in several embodiments can be used together with contrast-enhanced imaging techniques to detect and report biological events in an imaging target site comprising a mammalian cell and/or organism.

公开(公告)号：US11802318B2

公开(公告)日：2023-10-31

申请号：US16323103

申请日：2017-08-04

申请人： Arizona Board of Regents on behalf of Arizona State University

发明人： Alexander Green ,  Duo Ma

IPC分类号： C12Q1/6897 ,  C12Q1/70 ,  C12N15/113 ,  C12Q1/68 ,  C12N15/67 ,  C12N15/63 ,  C12Q1/6809 ,  C12Q1/6816 ,  C12Q1/689

CPC分类号： C12Q1/6897 ,  C12N15/113 ,  C12N15/63 ,  C12N15/67 ,  C12Q1/68 ,  C12Q1/6809 ,  C12Q1/689 ,  C12Q1/6816 ,  C12Q1/70 ,  C12N2310/531

摘要： Provided herein are synthetic nucleic acid molecules known as loop-mediated riboregulators that have single-nucleotide polymorphism (SNP) sensitivity and ultralow OFF state signal levels. Loop-mediated riboregulators can activate or repress gene expression in response to trigger RNAs bearing completely arbitrary sequences. Also provided herein are methods of using such synthetic nucleic acid molecules for detecting the presence or absence of a particular target RNA in, for example, a biological sample.

公开(公告)号：US20230313207A1

公开(公告)日：2023-10-05

申请号：US18330356

申请日：2023-06-06

申请人： CureVac SE

发明人： Andreas THESS ,  Thomas SCHLAKE ,  Stefanie GRUND

IPC分类号： C12N15/67 ,  A61K39/12 ,  C12N15/85 ,  A61K39/145 ,  A61K39/205 ,  C12N15/68

CPC分类号： C12N15/67 ,  A61K39/12 ,  C12N15/85 ,  A61K39/145 ,  A61K39/205 ,  C12N15/68 ,  A61K48/00

摘要： The invention relates to an artificial nucleic acid molecule comprising an open reading frame and a 3′-UTR comprising at least one poly(A) sequence or a polyadenylation signal. The invention further relates to a vector comprising the artificial nucleic acid molecule comprising an open reading frame and a 3′-UTR comprising at least one poly(A) sequence or a polyadenylation signal, to a cell comprising the artificial nucleic acid molecule or the vector, to a pharmaceutical composition comprising the artificial nucleic acid molecule or the vector and to a kit comprising the artificial nucleic acid molecule, the vector and/or the pharmaceutical composition. The invention also relates to a method for increasing protein production from an artificial nucleic acid molecule and to the use of a 3′-UTR for a method for increasing protein production from an artificial nucleic acid molecule. Moreover, the invention concerns the use of the artificial nucleic acid molecule, the vector, the kit or the pharmaceutical composition as a medicament, as a vaccine or in gene therapy.