公开(公告)号：US20050113325A1

公开(公告)日：2005-05-26

申请号：US10938184

申请日：2004-09-09

申请人： Sergei Gryaznov ,  Krisztina Pongracz

发明人： Sergei Gryaznov ,  Krisztina Pongracz

IPC分类号： A01N43/04 ,  A61K48/00 ,  C07H21/02 ,  C07H21/04 ,  C12N20060101 ,  C12N5/00 ,  C12N15/113

CPC分类号： C12N15/115 ,  A61K47/542 ,  A61K47/543 ,  A61K47/544 ,  A61K47/554 ,  C07C233/18 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/314 ,  C12N2310/351 ,  C12N2310/3515 ,  C12N2320/30 ,  C12Y207/07049

摘要： Compounds comprising an oligonucleotide moiety covalently linked to a lipid moiety are disclosed. The oligonucleotide moiety comprises a sequence that is complementary to the RNA component of human telomerase. The compounds inhibit telomerase activity in cells with a high potency and have superior cellular uptake characteristics.

摘要翻译： 公开了包含与脂质部分共价连接的寡核苷酸部分的化合物。 寡核苷酸部分包含与人端粒酶的RNA成分互补的序列。 该化合物在高效能细胞中抑制端粒酶活性并具有优异的细胞摄取特征。

公开(公告)号：US20030176374A1

公开(公告)日：2003-09-18

申请号：US09851871

申请日：2001-05-09

发明人： C. Frank Bennett ,  Timothy A. Vickers ,  James G. Karras

IPC分类号： A61K048/00

CPC分类号： C12N15/1138 ,  A61K31/711 ,  A61K38/00 ,  C07H21/00 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3515 ,  Y02P20/582 ,  C12N2310/3525 ,  C12N2310/3521

摘要： Compositions and methods for the treatment of inflammatory skin disorders, such as psoriasis, with oligonucleotides which specifically hybridize with nucleic acids encoding B7 proteins.

摘要翻译： 用于治疗炎性皮肤病，如牛皮癣的组合物和方法，其与与编码B7蛋白的核酸特异性杂交的寡核苷酸。

公开(公告)号：US06518017B1

公开(公告)日：2003-02-11

申请号：US09136080

申请日：1998-08-18

申请人： Timothy A. Riley ,  Bob D. Brown ,  Lyle J. Arnold

发明人： Timothy A. Riley ,  Bob D. Brown ,  Lyle J. Arnold

IPC分类号： C12Q168

CPC分类号： C12N15/1093 ,  C12N15/113 ,  C12N15/1135 ,  C12N15/1137 ,  C12N2310/12 ,  C12N2310/31 ,  C12N2310/3125 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/318 ,  C12N2310/3181 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/3533 ,  C12N2310/3521

摘要： Combinatorial libraries comprise first oligonucleotide analogs and second oligonucleotide analogs which are coupled together to form antisense molecules capable of binding target polynucleotides and activating an RNase, and ribozymes capable of cleaving polynucleotides.

摘要翻译： 组合文库包含第一寡核苷酸类似物和第二寡核苷酸类似物，其偶联在一起以形成能够结合靶多核苷酸并激活核糖核酸酶的反义分子，以及能够切割多核苷酸的核酶。

公开(公告)号：US06319906B1

公开(公告)日：2001-11-20

申请号：US09326186

申请日：1999-06-04

申请人： C. Frank Bennett ,  Timothy A. Vickers

发明人： C. Frank Bennett ,  Timothy A. Vickers

IPC分类号： A61K317115

CPC分类号： C12N15/1138 ,  A61K31/711 ,  A61K38/00 ,  C07H21/00 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3515 ,  Y02P20/582 ,  C12N2310/3525 ,  C12N2310/3521

摘要： Compositions and methods for the diagnosis, prevention and treatment of immune states and disorders amenable to treatment through modulation of T cell activation are provided. In accordance with preferred embodiments, oligonucleotides are provided which are specifically hybridizable with nucleic acids encoding B7 proteins.

摘要翻译： 提供了用于诊断，预防和治疗适于通过调节T细胞活化治疗的免疫状态和障碍的组合物和方法。 根据优选实施方案，提供了与编码B7蛋白的核酸特异性杂交的寡核苷酸。

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：US12116575B2

公开(公告)日：2024-10-15

申请号：US18322487

申请日：2023-05-23

申请人： Universitat de València ,  ARTHEX BIOTECH S.L.

发明人： Rubén Artero Allepuz ,  Nerea Moreno Cervera ,  Irene González Martínez ,  Estefanía Cerro Herreros ,  Eric G. Marcusson ,  María Beatriz Llamusí Troisi

IPC分类号： C12N15/113 ,  A61P21/00

CPC分类号： C12N15/113 ,  A61P21/00 ,  C12N2310/141 ,  C12N2310/31

摘要： The invention provides oligonucleotide and/or oligonucleotide analogue molecules that are antagonists of a microRNA, preferably antagonists of human microRNAs hsa-miR-23b-3p and hsa-miR-218-5p, that comprise a mixture of phosphorothioate and phosphodiester linkages, and that are conjugated to at least one oleic acid molecule. Inhibiting these microRNAs allows to increase the endogenous levels of the corresponding proteins MBNL1 and/or MBNL2. The present invention further provides compositions comprising said oligonucleotides and/or oligonucleotide analogue molecules and their uses for the treatment and prevention of DM in a subject in need thereof.

公开(公告)号：US12104156B2

公开(公告)日：2024-10-01

申请号：US18052899

申请日：2022-11-04

申请人： Avidity Biosciences, Inc.

发明人： Barbora Malecova ,  Rob Burke ,  David Sala Cano ,  Beatrice Diana Darimont

IPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/68 ,  A61K48/00 ,  A61P21/00 ,  A61P21/06 ,  C07K16/28 ,  C07K19/00

CPC分类号： C12N15/113 ,  A61K31/7125 ,  A61K47/6807 ,  A61K47/6849 ,  A61K47/6889 ,  A61K48/005 ,  A61P21/00 ,  A61P21/06 ,  C07K16/2881 ,  C07K19/00 ,  C07K2317/55 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/313 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3513 ,  C12N2320/32

摘要： Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.

公开(公告)号：US20240091383A1

公开(公告)日：2024-03-21

申请号：US18274496

申请日：2022-01-28

申请人： JOINT STOCK COMPANY «BIOCAD»

发明人： Dmitriy Aleksandrovich MADERA ,  Anna Sergeevna VESELOVA ,  Aleksei Sergeevich SIUTKIN ,  Pavel Mikhailovich GERSHOVICH ,  Dmitry Valentinovich MOROZOV

IPC分类号： A61K48/00 ,  C12N15/86

CPC分类号： A61K48/0058 ,  C12N15/86 ,  C12N2310/141 ,  C12N2310/31 ,  C12N2750/14143

摘要： The present application relates to the fields of biotechnology, virology, genetics, and molecular biology. More specifically, the present invention relates to an isolated nucleic acid for producing a gene therapy viral product, said isolated nucleic acid comprising a nucleic acid that encodes the SMN1 protein having the amino acid sequence of SEQ ID NO: 1, and a nucleic acid that encodes the microRNA miR-23a, an expression cassette and a vector based thereon, as well as an AAV9-based recombinant virus for expressing the SMN1 gene in target cells, a pharmaceutical composition that includes said recombinant virus, and various uses of the above recombinant virus and the above composition.

公开(公告)号：US11761000B2

公开(公告)日：2023-09-19

申请号：US17352806

申请日：2021-06-21

申请人： IONIS PHARMACEUTICALS, Inc.

发明人： Susan M. Freier

IPC分类号： C07H21/04 ,  C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/335 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US20230256001A1

公开(公告)日：2023-08-17

申请号：US17937256

申请日：2022-09-30

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： Mark K. SCHLEGEL ,  Maja JANAS ,  Vasant R. JADHAV ,  Muthiah MANOHARAN ,  Kallanthottathil G. RAJEEV ,  Muthusamy JAYARAMAN ,  Alexander V. KEL'IN ,  Shigeo MATSUDA ,  Klaus CHARISSE ,  Jayaprakash K. NAIR ,  Martin A. MAIER ,  Alfica SEHGAL ,  Christopher BROWN ,  Christopher THEILE ,  Stuart MILSTEIN

IPC分类号： A61K31/713 ,  C12N15/113

CPC分类号： A61K31/713 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/31 ,  C12N2310/351

摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The antisense strand of the dsRNA molecule comprises at least one thermally destabilizing nucleotide occurring at a seed region; the dsRNA comprises at least four 2′-fluoro modifications, and the sense strand of the dsRNA molecule comprises ligand, wherein the ligand is an ASGPR ligand. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.