公开(公告)号：US12121595B2

公开(公告)日：2024-10-22

申请号：US17203232

申请日：2021-03-16

Applicant: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL

Inventor： Paul Taylor Martin

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

CPC classification number: 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

Abstract: 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

Applicant: Ionis Pharmaceuticals, Inc.

Inventor： Susan M. Freier ,  Huynh-Hoa Bui

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

CPC classification number: 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

Abstract: 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

Applicant: SONY GROUP CORPORATION

Inventor： Sharat SINGH ,  Tracy MATRAY ,  Michael VANBRUNT ,  John MCCUTCHEON

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

CPC classification number: C09B69/102 ,  C07H21/04 ,  C09B69/109 ,  G01N33/582

Abstract: 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

Applicant: Illumina, Inc. ,  Illumina Cambridge Limited

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

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

CPC classification number: C07H21/04 ,  C12Q1/6806 ,  C12Q1/6876 ,  C12Q1/6806 ,  C12Q2523/107 ,  C12Q2525/113 ,  C12Q2565/543

Abstract: 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

Applicant: Complete Genomics, Inc

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

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

CPC classification number: 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

Abstract: 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

Applicant: Board of Regents, The University of Texas System

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

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

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

Abstract: 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

Applicant: The Regents of the University of Colorado, a body corporate

Inventor： Ayman Alawneh ,  Marvin Caruthers

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

CPC classification number: C07H21/04 ,  C07H1/00 ,  C07H19/073

Abstract: 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

Applicant: Ionis Pharmaceuticals, Inc.

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

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

CPC classification number: 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

Abstract: 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

Applicant: GLAXOSMITHKLINE BIOLOGICALS SA

Inventor： Francesco Berlanda Scorza ,  Yingxia Wen ,  Andrew Geall ,  Frederick Porter

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

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

Abstract: 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

Applicant: Temple University-Of The Commonwealth System of Higher Education

Inventor： Richard T. Pomerantz ,  Tatiana Kent

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

CPC classification number: 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

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