公开(公告)号：US12121595B2

公开(公告)日：2024-10-22

申请号：US17203232

申请日：2021-03-16

申请人： RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL

发明人： Paul Taylor Martin

IPC分类号： C12N15/86 ,  A61K38/45 ,  A61K48/00 ,  A61P21/00 ,  C12N9/10 ,  C12Q1/68 ,  C07H21/04 ,  C12N15/864

CPC分类号： A61K48/0058 ,  A61K38/45 ,  A61K48/0075 ,  A61P21/00 ,  C12N9/1048 ,  C12N9/1051 ,  C12N15/86 ,  C12Q1/68 ,  C12Y204/01165 ,  C07H21/04 ,  C12N15/8645 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/42

摘要： The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a GALGT2 polynucleotide. The disclosure provides rAAV and methods of using the rAAV for GALGT2 gene therapy of neuromuscular disorders. Exemplary neuromuscular disorders include, but are not limited to, muscular dystrophies such as Duchenne muscular dystrophy, Congenital Muscular Dystrophy 1A and Limb Girdle Muscular Dystrophy 2D.

公开(公告)号：US20240318183A1

公开(公告)日：2024-09-26

申请号：US18489316

申请日：2023-10-18

申请人： Ionis Pharmaceuticals, Inc.

发明人： Susan M. Freier ,  Huynh-Hoa Bui

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04

CPC分类号： C12N15/1137 ,  A61K31/7088 ,  A61K38/36 ,  C07H21/04 ,  C12N15/113 ,  C12Y304/21034 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3525 ,  C12N2320/31 ,  C12N2320/35

摘要： Disclosed herein are antisense compounds and methods for decreasing PKK mRNA and protein expression. Such methods, compounds, and compositions are useful to treat, prevent, or ameliorate PKK-associated diseases, disorders, and conditions.

公开(公告)号：US20240318004A1

公开(公告)日：2024-09-26

申请号：US18678656

申请日：2024-05-30

申请人： SONY GROUP CORPORATION

发明人： Sharat SINGH ,  Tracy MATRAY ,  Michael VANBRUNT ,  John MCCUTCHEON

IPC分类号： C09B69/10 ,  C07H21/04 ,  G01N33/58

CPC分类号： C09B69/102 ,  C07H21/04 ,  C09B69/109 ,  G01N33/582

摘要： Compounds useful as fluorescent or colored dyes are disclosed. The compounds have the following structure (I):




or a stereoisomer, tantomer or salt thereof, wherein R1, R2, R3, R4, R5, L1a, L1b, L2, L3, L4, L5, L6, L7, M1, M2, q, w, m and n are as defined herein. Methods associated with preparation and use of such compounds are also provided.

公开(公告)号：US12084474B2

公开(公告)日：2024-09-10

申请号：US16411913

申请日：2019-05-14

申请人： Illumina, Inc. ,  Illumina Cambridge Limited

发明人： Xiaolin Wu ,  Randall Smith ,  Peyton Shieh ,  John M. Beierle ,  Wayne N. George ,  Elliot John Lawrence ,  Jie Mao ,  Xiaohai Liu

IPC分类号： C07H21/04 ,  C12Q1/6806 ,  C12Q1/6876

CPC分类号： C07H21/04 ,  C12Q1/6806 ,  C12Q1/6876 ,  C12Q1/6806 ,  C12Q2523/107 ,  C12Q2525/113 ,  C12Q2565/543

摘要： Embodiments of the present disclosure relate to methods of preparation of templates for polynucleotide sequencing. In particular, the disclosure relates to linearization of clustered polynucleotides in preparation for sequencing by cleavage of one or more first strands of double-stranded polynucleotides immobilized on a solid support by a transition metal complex, for example, a palladium complex or a nickel complex. Further disclosure relate to linearization of clustered polynucleotides by cleaving one or more second strands of double double-stranded polynucleotides immobilized on a solid support comprising azobenzene linker by Na2S2O4. Nucleotides and oligonucleotides comprising a 3′ phosphate moiety blocking group, and methods of removing the same using a fluoride reagent are also disclosed.

公开(公告)号：US20240263230A1

公开(公告)日：2024-08-08

申请号：US18592843

申请日：2024-03-01

申请人： Complete Genomics, Inc

发明人： Radoje Drmanac ,  Matthew J. Callow ,  Snezana Drmanac ,  Brian K. Hauser ,  George Yeung

IPC分类号： C12Q1/6874 ,  C07H21/04 ,  C07K1/04 ,  C12Q1/6806 ,  C12Q1/682 ,  C12Q1/6837 ,  C12Q1/6869 ,  G01N15/1404 ,  G01N15/1434

CPC分类号： C12Q1/6874 ,  C07H21/04 ,  C07K1/047 ,  C12Q1/6806 ,  C12Q1/682 ,  C12Q1/6837 ,  C12Q1/6869 ,  G01N15/1404 ,  G01N15/1434 ,  C12Q2525/151 ,  C12Q2525/313 ,  C12Q2531/125 ,  C12Q2565/513 ,  Y10S977/778 ,  Y10S977/789 ,  Y10S977/792 ,  Y10S977/88 ,  Y10S977/882

摘要： Random arrays of single molecules are provided for carrying out large scale analyzes, particularly of biomolecules, such as genomic DNA, cDNAs, proteins, and the like. In one aspect, arrays of the invention comprise concatemers of DNA fragments that are randomly disposed on a regular array of discrete spaced apart regions, such that substantially all such regions contain no more than a single concatemer. Preferably, such regions have areas substantially less than 1 μm2 and have nearest neighbor distances that permit optical resolution of on the order of 109 single molecules per cm2. Many analytical chemistries can be applied to random arrays of the invention, including sequencing by hybridization chemistries, sequencing by synthesis chemistries, SNP detection chemistries, and the like, to greatly expand the scale and potential applications of such techniques.

公开(公告)号：US12036231B2

公开(公告)日：2024-07-16

申请号：US17809190

申请日：2022-06-27

申请人： Board of Regents, The University of Texas System

发明人： Maria Emilia Di Francesco ,  Philip Jones ,  Michael A. Curran

IPC分类号： A61K31/7084 ,  A61P35/00 ,  C07D473/02 ,  C07D473/16 ,  C07D473/26 ,  C07D473/32 ,  C07D473/34 ,  C07H21/02 ,  C07H21/04

CPC分类号： A61K31/7084 ,  A61P35/00 ,  C07D473/02 ,  C07D473/16 ,  C07D473/26 ,  C07D473/32 ,  C07D473/34 ,  C07H21/02 ,  C07H21/04

摘要： Disclosed herein are new cyclic dinucleotide compounds and compositions and their application as pharmaceuticals for the treatment of disease. Methods of modulation of immune response to disease, and induce Stimulator of Interferon Genes (STING) dependent type I interferon production and co-regulated genes in a human or animal subject are also provided for the treatment diseases such as cancer, particularly metastatic solid tumors and lymphomas, inflammation, allergic and autoimmune disease, infectious disease, and for use as anti-viral agents and vaccine adjuvants.

公开(公告)号：US12030911B2

公开(公告)日：2024-07-09

申请号：US17251608

申请日：2019-06-14

申请人： The Regents of the University of Colorado, a body corporate

发明人： Ayman Alawneh ,  Marvin Caruthers

IPC分类号： C07H21/04 ,  C07H1/00 ,  C07H19/073

CPC分类号： C07H21/04 ,  C07H1/00 ,  C07H19/073

摘要： Oligonucleotides comprising phosphoramidimidate internucleotide linkages, and methods of efficiently synthesizing these oligonucleotides with high yield are provided. These oligonucleotides form duplexes with complementary DNA or RNA that are more stable than natural DNA or DNA/RNA complexes, are active with RNAse H1, and may be transfected into cells using standard lipid reagents. These analogues are therefore useful for numerous therapeutic antisense applications.

公开(公告)号：US12030910B2

公开(公告)日：2024-07-09

申请号：US17021510

申请日：2020-09-15

申请人： Ionis Pharmaceuticals, Inc.

发明人： Stanley T. Crooke ,  Xue-hai Liang ,  Wen Shen

IPC分类号： C07H21/04 ,  A61K31/7088 ,  C07H21/00 ,  C12N15/113 ,  C12N15/67 ,  C12P19/34

CPC分类号： C07H21/04 ,  A61K31/7088 ,  C07H21/00 ,  C12N15/113 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/67 ,  C12P19/34 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/346 ,  C12Y301/26004 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/321 ,  C12N2310/3525

摘要： In certain embodiments, the present disclosure provides compounds and methods of increasing the amount or activity of a target protein in a cell. In certain embodiments, the compounds comprise a translation suppression element inhibitor. In certain embodiments, the translation suppression element inhibitor is a uORF inhibitor. In certain embodiments, the uORF inhibitor is an antisense compound.

公开(公告)号：US12018044B2

公开(公告)日：2024-06-25

申请号：US17157150

申请日：2021-01-25

申请人： GLAXOSMITHKLINE BIOLOGICALS SA

发明人： Francesco Berlanda Scorza ,  Yingxia Wen ,  Andrew Geall ,  Frederick Porter

IPC分类号： C12Q1/68 ,  B01D15/36 ,  B01D15/38 ,  B01D61/14 ,  C07H1/06 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/10

CPC分类号： C07H21/02 ,  B01D15/36 ,  B01D15/38 ,  B01D15/3847 ,  B01D61/14 ,  C07H1/06 ,  C07H21/00 ,  C07H21/04 ,  C12N15/101 ,  C12N15/1017 ,  B01D2015/3838

摘要： Methods for purifying RNA from a sample, comprising one or more steps of tangential flow filtration, hydroxyapatite chromatography, core bead flow-through chromatography, or any combinations thereof. These techniques are useful individually, but show very high efficiency when used in combination, or when performed in particular orders. The methods can purify RNA in a highly efficient manner without unduly compromising potency or stability, to provide compositions in which RNA is substantially cleared of contaminants. Moreover, they can be performed without the need for organic solvents.

公开(公告)号：US11993791B2

公开(公告)日：2024-05-28

申请号：US17120973

申请日：2020-12-14

申请人： Temple University-Of The Commonwealth System of Higher Education

发明人： Richard T. Pomerantz ,  Tatiana Kent

IPC分类号： C12N9/12 ,  C07H21/04 ,  C12P19/34 ,  C12Q1/68 ,  C12Q1/6806 ,  C12Q1/6827 ,  C12Q1/6844 ,  C12Q1/6853

CPC分类号： C12N9/1252 ,  C12P19/34 ,  C12Q1/68 ,  C12Q1/6806 ,  C12Q1/6827 ,  C12Q1/6844 ,  C12Q1/6853 ,  C07H21/04 ,  C12N2310/344 ,  C12Q2521/101 ,  C12Q2525/101 ,  C12Q2563/137 ,  C12Y207/07007 ,  C12Q1/6844 ,  C12Q2521/101 ,  C12Q2521/101 ,  C12Q2563/137 ,  C12Q1/6853 ,  C12Q2521/101 ,  C12Q2521/131 ,  C12Q2525/101 ,  C12Q2525/117 ,  C12Q2525/121 ,  C12Q2525/173 ,  C12Q2527/125 ,  C12Q2563/107 ,  C12Q1/6806 ,  C12Q2521/101 ,  C12Q2521/131 ,  C12Q2525/101 ,  C12Q2525/117 ,  C12Q2525/121 ,  C12Q2525/173 ,  C12Q2527/125 ,  C12Q2563/107

摘要： The invention provides compositions and methods for modifying the 3′-terminal ends of nucleic acids using DNA polymerase θ terminal transferase activity.