公开(公告)号：US12129467B2

公开(公告)日：2024-10-29

申请号：US18410136

申请日：2024-01-11

申请人： ARBUTUS BIOPHARMA CORPORATION

发明人： Ed Yaworski ,  Lloyd B. Jeffs ,  Lorne R. Palmer

IPC分类号： C07H21/04 ,  A61K9/107 ,  A61K9/50 ,  A61K9/51 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/712 ,  A61K31/713 ,  A61K47/14 ,  A61K47/54 ,  C12N15/113 ,  C12N15/88 ,  A61K9/127

CPC分类号： C12N15/113 ,  A61K9/1075 ,  A61K9/5015 ,  A61K9/5123 ,  A61K31/7088 ,  A61K31/7105 ,  A61K31/712 ,  A61K31/713 ,  A61K47/14 ,  A61K47/543 ,  C12N15/88 ,  A61K9/1272 ,  A61K9/1274 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/32

摘要： The present invention provides novel, stable lipid particles having a non-lamellar structure and comprising one or more active agents or therapeutic agents, methods of making such lipid particles, and methods of delivering and/or administering such lipid particles. More particularly, the present invention provides stable nucleic acid-lipid particles (SNALP) that have a non-lamellar structure and that comprise a nucleic acid (such as one or more interfering RNA), methods of making the SNALP, and methods of delivering and/or administering the SNALP.

公开(公告)号：US20240352455A1

公开(公告)日：2024-10-24

申请号：US18501779

申请日：2023-11-03

申请人： Ionis Pharmaceuticals, Inc.

发明人： Thazha P. PRAKASH ,  Punit P. SETH ,  Eric E. SWAYZE ,  Mark J. GRAHAM

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61K47/54 ,  C07H21/04 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61K47/549 ,  C07H21/04 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/17 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3511 ,  C12N2310/3513 ,  C12N2310/3515 ,  C12N2310/3525 ,  C12N2310/353 ,  C12N2320/32

摘要： Provided herein are oligomeric compounds with conjugate groups targeting apoplipoprotein (a) [apo(a)]. In certain embodiments, the apo(a) targeting oligomeric compounds are conjugated to N-Acetylgalactosamine. Also disclosed herein are conjugated oligomeric compounds targeting apo(a) for use in decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) and/or Lp(a). Certain diseases, disorders or conditions related to apo(a) and/or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The conjugated oligomeric compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

公开(公告)号：US20240350662A1

公开(公告)日：2024-10-24

申请号：US18512804

申请日：2023-11-17

申请人： DNARx

发明人： Robert James DEBS ,  Timothy D. HEATH ,  Chakkrapong HANDUMRONGKUL

IPC分类号： A61K48/00 ,  A61K9/127 ,  A61K31/19 ,  A61K31/522 ,  A61K31/573 ,  A61K38/19 ,  A61K38/48 ,  A61K39/395 ,  A61K47/69 ,  C07K16/00 ,  C07K16/28 ,  A61K31/00 ,  A61K39/00 ,  C07H21/04 ,  C12N15/85

CPC分类号： A61K48/0016 ,  A61K9/1272 ,  A61K31/19 ,  A61K31/522 ,  A61K31/573 ,  A61K38/193 ,  A61K38/4846 ,  A61K39/395 ,  A61K47/6911 ,  A61K48/0066 ,  A61K48/0083 ,  C07K16/00 ,  C07K16/2887 ,  C12Y304/21022 ,  A61K31/00 ,  A61K2039/505 ,  A61K2039/53 ,  A61K48/005 ,  C07H21/04 ,  C07K2317/24 ,  C12N15/85

摘要： The present invention provides compositions, systems, kits, and methods for generating expression of one or more proteins and/or biologically active nucleic acid molecules in a subject (e.g., at therapeutic levels for extended periods required to produce therapeutic effects). In certain embodiments, systems and kits are provided that comprise a first composition comprising a first amount of polycationic structures, and a second composition comprising a therapeutically effective amount of expression vectors (e.g., non-viral expression vectors not associated with liposomes) that are CpG-free or CpG-reduced, where the expression vectors comprise a first nucleic acid sequence encoding: i) a first therapeutic protein or proteins, and/or ii) a first biologically active nucleic acid molecule or molecules.

公开(公告)号：US12116589B2

公开(公告)日：2024-10-15

申请号：US17881193

申请日：2022-08-04

申请人： Voyager Therapeutics, Inc. ,  THE CURATORS OF THE UNIVERSITY OF MISSOURI

发明人： Dinah Wen-Yee Sah ,  Qingmin Chen ,  Joan R. Coates ,  Holger Patzke ,  Jinzhao Hou

IPC分类号： C07H21/04 ,  A61K31/713 ,  A61P25/28 ,  C07H21/02 ,  C12N15/113 ,  C12N15/86

CPC分类号： C12N15/86 ,  A61K31/713 ,  A61P25/28 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/30

摘要： The present invention relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and/or canine degenerative myelopathy (DM).

公开(公告)号：US20240317794A1

公开(公告)日：2024-09-26

申请号：US18613537

申请日：2024-03-22

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Anna Balanov ,  Gary Shen ,  Fred Briones ,  Ligang Zhang ,  Lubomir Nechev

IPC分类号： C07H21/04 ,  C12N15/10

CPC分类号： C07H21/04 ,  C12N15/101

摘要： The disclosure relates to a method of deprotecting a 3′-hydroxyl of an oligonucleotide having the 3′-hydroxyl protected by a silyl protecting group, comprising contacting the oligonucleotide with a mixture comprising DMSO and a deprotecting reagent. The disclosure also relates to a method of recovering a synthesized oligonucleotide from a solid support based on the method of deprotecting a 3′-hydroxyl of an oligonucleotide. The disclosure also relates to a 3′-hydroxyl protected oligonucleotide intermediate involved during the method of deprotecting the oligonucleotide.

公开(公告)号：US20240294962A1

公开(公告)日：2024-09-05

申请号：US18532609

申请日：2023-12-07

申请人： The Scripps Research Institute

发明人： Floyd E. ROMESBERG ,  Vivian T. DIEN ,  Aaron W. FELDMAN ,  Rebekah J. KARADEEMA ,  Lingjun LI ,  Michael P. LEDBETTER ,  Anne Xiaozhou ZHOU

IPC分类号： C12P19/34 ,  C07H19/24 ,  C07H21/04

CPC分类号： C12P19/34 ,  C07H19/24 ,  C07H21/04

摘要： Disclosed herein are compositions, methods, cells, engineered microorganisms, and kits for increasing the production of proteins or polypeptides comprising one or more unnatural amino acids. Further provided are compositions, cells, engineered microorganisms, and kits for increasing the retention of unnatural nucleic acids encoding the unnatural amino acids in an engineered cell, or semi-synthetic organism.

公开(公告)号：US12071662B2

公开(公告)日：2024-08-27

申请号：US18313227

申请日：2023-05-05

申请人： Illumina Cambridge Limited

发明人： Elena Cressina ,  Antoine Francais ,  Xiaohai Liu

IPC分类号： C12Q1/68 ,  C07H19/06 ,  C07H19/073 ,  C07H19/10 ,  C07H19/14 ,  C07H19/16 ,  C07H19/20 ,  C07H21/00 ,  C07H21/04 ,  C12Q1/6809 ,  C12Q1/6816 ,  C12Q1/6869

CPC分类号： C12Q1/6869 ,  C07H19/06 ,  C07H19/073 ,  C07H19/10 ,  C07H19/14 ,  C07H19/16 ,  C07H19/20 ,  C07H21/00 ,  C07H21/04 ,  C12Q1/6809 ,  C12Q1/6816 ,  C12Q1/6869 ,  C12Q2525/117 ,  C12Q2535/122

摘要： The present disclosure relates to new nucleotide and oligonucleotide compounds and their use in nucleic acid sequencing applications.

公开(公告)号：US12071652B2

公开(公告)日：2024-08-27

申请号：US17323695

申请日：2021-05-18

申请人： Purdue Research Foundation

发明人： Casey J. Krusemark ,  Kyle Denton ,  Dongwook Kim ,  Rachael Jetson

IPC分类号： C12Q1/6823 ,  C07H21/04 ,  C12N15/10 ,  C12Q1/686 ,  G01N33/68

CPC分类号： C12Q1/6823 ,  C07H21/04 ,  C12N15/1065 ,  C12N15/1075 ,  C12Q1/686 ,  G01N33/68 ,  C12N2310/3517 ,  C12N2320/10 ,  C12N15/1075 ,  C12Q2521/537 ,  C12Q2563/179 ,  C12Q1/6823 ,  C12Q2521/537 ,  C12Q2563/179

摘要： Systems, kits, and methods for detecting and quantifying proteomic activity using DNA-encoded probes are provided, where the proteomic activity may be enzymatic activity or ligand binding affinity. Such systems and methods encode quantitative proteomic activity information into DNA sequence populations and utilize DNA-linked substrates or ligands as activity probes. The systems, kits, and methods that are directed to detecting ligand affinity further include crosslinking steps to ensure the integrity of the DNA-linked ligands during purification and washing. Signal detection involves the chemical manipulation of a probe population downstream of sample exposure and application of purifying, selective pressure for desired products. Selection-induced changes in DNA abundance between the initial pool and the purified pool indicate sample activity.

公开(公告)号：US12071624B2

公开(公告)日：2024-08-27

申请号：US17250806

申请日：2019-09-04

申请人： OSAKA UNIVERSITY ,  NATIONAL INSTITUTES OF BIOMEDICAL INNOVATION, HEALTH AND NUTRITION ,  NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY

发明人： Akira Kikuchi ,  Shinji Matsumoto ,  Satoshi Obika ,  Yuya Kasahara ,  Takumi Fukumoto

IPC分类号： C07H21/04 ,  A61P35/04 ,  C12N15/113

CPC分类号： C12N15/1137 ,  A61P35/04 ,  C12N2310/11 ,  C12N2310/315

摘要： The purpose of the present invention is to provide an antisense oligonucleotide that targets an ARL4C molecule and exerts an antitumor effect in vivo, and a nucleic acid drug using the antisense oligonucleotide. An antisense oligonucleotide that has a base sequence consisting of at least 10 consecutive bases contained in the base sequence represented by SEQ ID NO: 1. This antisense oligonucleotide targets an ARL4C molecule and thus can inhibit the expression of ARL4C in a tumor cell in vitro and suppress the migration and proliferation thereof. When systemically administered, moreover, the antisense oligonucleotide can exert an excellent antitumor effect in vivo too.

公开(公告)号：US12071623B2

公开(公告)日：2024-08-27

申请号：US18246845

申请日：2022-05-17

申请人： Nogra Pharma Limited

发明人： Francesca Viti ,  Marie McNulty ,  Salvatore Bellinvia

IPC分类号： C07H21/02 ,  C07H21/04 ,  C12N15/113

CPC分类号： C12N15/1136 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/3341

摘要： Disclosed herein polynucleotides complementary to IL-34, including IL-34 antisense oligonucleotides and IL-34 NAs, and methods for treating inflammatory diseases, such as an inflammatory bowel disease, and/or fibrosis, associated with elevated activity or expression of IL-34. Also disclosed are pharmaceutical compositions containing a polynucleotide complementary to IL-34, for example, an IL-34 antisense oligonucleotide or an IL-34 siRNA, useful for treating inflammatory diseases and/or fibrosis and manufacture of medicaments containing a disclosed polynucleotide to be used in treating inflammatory diseases and/or fibrosis.