ECSP21053104A - Moduladores de la expresión de hsd17b13 - Google Patents

Moduladores de la expresión de hsd17b13

Info

Publication number
ECSP21053104A
ECSP21053104A ECSENADI202153104A ECDI202153104A ECSP21053104A EC SP21053104 A ECSP21053104 A EC SP21053104A EC SENADI202153104 A ECSENADI202153104 A EC SENADI202153104A EC DI202153104 A ECDI202153104 A EC DI202153104A EC SP21053104 A ECSP21053104 A EC SP21053104A
Authority
EC
Ecuador
Prior art keywords
hsd17b13
expression modulators
hsd17b13 expression
compounds
methods
Prior art date
Application number
ECSENADI202153104A
Other languages
English (en)
Inventor
Susan Murray
Susan Freier
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=71102009&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ECSP21053104(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of ECSP21053104A publication Critical patent/ECSP21053104A/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • C12Q1/6886—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Amplifiers (AREA)

Abstract

Se proporcionan procedimientos, compuestos y composiciones útiles para inhibir la expresión de HSD17B13 . Tales compuestos, composiciones y procedimientos son útiles para tratar, prevenir o mejorar una enfermedad asociada con HSD17B13.
ECSENADI202153104A 2018-12-21 2021-07-19 Moduladores de la expresión de hsd17b13 ECSP21053104A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862783680P 2018-12-21 2018-12-21
US201962825581P 2019-03-28 2019-03-28
US201962827524P 2019-04-01 2019-04-01

Publications (1)

Publication Number Publication Date
ECSP21053104A true ECSP21053104A (es) 2021-08-31

Family

ID=71102009

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202153104A ECSP21053104A (es) 2018-12-21 2021-07-19 Moduladores de la expresión de hsd17b13

Country Status (23)

Country Link
US (2) US20220273691A1 (es)
EP (1) EP3899024A4 (es)
JP (2) JP7394137B2 (es)
KR (1) KR20210107027A (es)
CN (1) CN113544272A (es)
AU (2) AU2019403447B2 (es)
BR (1) BR112021011899A2 (es)
CA (1) CA3122432A1 (es)
CL (1) CL2021001643A1 (es)
CO (1) CO2021009009A2 (es)
CR (1) CR20210395A (es)
DO (2) DOP2021000130A (es)
EC (1) ECSP21053104A (es)
IL (2) IL316212A (es)
JO (1) JOP20210158A1 (es)
MX (1) MX2021007503A (es)
NI (1) NI202100056A (es)
PE (1) PE20211755A1 (es)
PH (1) PH12021551463A1 (es)
SG (1) SG11202106378VA (es)
TW (1) TW202039846A (es)
UA (1) UA128236C2 (es)
WO (1) WO2020132564A1 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210000906A1 (en) * 2018-03-21 2021-01-07 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
SG11202106378VA (en) 2018-12-21 2021-07-29 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
WO2021178377A2 (en) * 2020-03-05 2021-09-10 Synerk Inc. Compositions and methods for inhibiting asah1 gene expression
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
CN112635049A (zh) * 2020-12-23 2021-04-09 无锡市第二人民医院 一种研究HSD17B13 rs72613567基因变异和肾功能损伤之间关系的方法
PE20250120A1 (es) * 2021-06-24 2025-01-16 Lilly Co Eli Restos de administracion terapeutica novedosos y usos de estos
PE20250353A1 (es) * 2021-06-24 2025-02-06 Lilly Co Eli Terapeutica con arn novedosos y usos de estos
CA3229569A1 (en) 2021-08-20 2023-02-23 Enanta Pharmaceuticals, Inc. 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors and methods of use thereof
JP2024545602A (ja) * 2021-11-19 2024-12-10 ウェイブ ライフ サイエンシズ リミテッド Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法
CN118541487A (zh) * 2022-01-20 2024-08-23 上海拓界生物医药科技有限公司 一种dsRNA、其应用及制备方法
TW202400791A (zh) * 2022-05-06 2024-01-01 大陸商蘇州瑞博生物技術股份有限公司 一種核酸、含有該核酸的組合物與綴合物及製備方法和用途
WO2026026937A1 (zh) * 2024-07-31 2026-02-05 前沿生物药业(南京)股份有限公司 一种用于抑制HSD17B13基因表达的siRNA及其修饰物和应用
WO2026082165A1 (zh) * 2024-10-18 2026-04-23 正大天晴药业集团股份有限公司 修饰的双链核糖核酸

Family Cites Families (139)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US4751219A (en) 1985-02-05 1988-06-14 Nederlandse Centrale Organisatie Voor Toegepast-Natuur-Wetenschappelijk Onderzoek Synthetic glycolipides, a process for the preparation thereof and several uses for these synthetic glycolipides
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5859221A (en) 1990-01-11 1999-01-12 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5948903A (en) 1991-01-11 1999-09-07 Isis Pharmaceuticals, Inc. Synthesis of 3-deazapurines
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
EP0637965B1 (en) 1991-11-26 2002-10-16 Isis Pharmaceuticals, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
TW520293B (en) 1995-11-22 2003-02-11 Univ Johns Hopkins Med Delivery system to enhance cellular uptake of biomolecules
US20030119724A1 (en) 1995-11-22 2003-06-26 Ts`O Paul O.P. Ligands to enhance cellular uptake of biomolecules
US6620916B1 (en) 1996-09-26 2003-09-16 Ajinomoto Co., Inc. Modified physiologically active proteins and medicinal compositions containing the same
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
EP1015469B2 (en) 1997-09-12 2015-11-18 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
IL145778A0 (en) 1999-04-21 2002-07-25 American Home Prod Methods and compositions for inhibiting the function of polynucleotide sequences
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US6383812B1 (en) 1999-05-28 2002-05-07 Academia Sinica Anti liver disease drug R-YEEE and method of synthesizing branched galactose-terminal glycoproteins
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
AU2001250572A1 (en) * 2000-04-07 2001-10-23 Epigenomics Ag Detection of single nucleotide polymorphisms (snp's) and cytosine-methylations
CN100406065C (zh) 2000-12-01 2008-07-30 细胞工厂治疗公司 糖基化/半乳糖基化肽、双官能接头和核苷酸单体/多聚体的缀合物以及相关的组合物和使用方法
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US20030175906A1 (en) 2001-07-03 2003-09-18 Muthiah Manoharan Nuclease resistant chimeric oligonucleotides
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US20100240730A1 (en) 2002-02-20 2010-09-23 Merck Sharp And Dohme Corp. RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA)
CA2498772A1 (en) 2002-09-11 2004-03-25 Santaris Pharma A/S Modified pna molecules
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US6673661B1 (en) 2002-12-20 2004-01-06 Taiwan Semiconductor Manufacturing Co., Ltd. Self-aligned method for forming dual gate thin film transistor (TFT) device
EP2669377A3 (en) 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
WO2004101619A1 (ja) 2003-05-15 2004-11-25 Shionogi Co., Ltd. 機能的糖ペプチドの合理的設計および合成
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
WO2005027962A1 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. 4’-thionucleosides and oligomeric compounds
EP2990410A1 (en) 2004-08-10 2016-03-02 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
WO2006031461A2 (en) 2004-09-09 2006-03-23 Isis Pharmaceuticals, Inc. Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1841867A1 (en) 2005-01-24 2007-10-10 Avaris AB COMPLEX CONTAINING SiRNA, ShRNA OR ANTISENSE MOLECULE AND FUNCTIONAL ENTITY, FOR IMPROVED SPECIFICITY AND DELIVERY
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
DE602007009487D1 (de) 2006-01-27 2010-11-11 Isis Pharmaceutical Inc 6-modifizierte bicyclische nukleinsäureanaloga
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
EP2606911A1 (en) 2007-02-16 2013-06-26 KTB Tumorforschungsgesellschaft mbH Receptor And Antigen Targeted Prodrug
WO2008131419A2 (en) 2007-04-23 2008-10-30 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2188298B1 (en) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
CA2713379A1 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009120878A2 (en) 2008-03-26 2009-10-01 Alnylam Pharmaceuticals, Inc. Non-natural ribonucleotides, and methods of use thereof
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2010039548A2 (en) 2008-09-23 2010-04-08 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
EP2391343B1 (en) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
WO2011003071A1 (en) * 2009-07-02 2011-01-06 The United States Of America, As Represented By The Secretary, Department Of Health & Human Services Methods of modulating hepatitis c virus infection
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011038356A2 (en) 2009-09-25 2011-03-31 Johns Hopkins University Novel liver-targeting agents and their synthesis
TWI388338B (zh) 2009-10-26 2013-03-11 Iner Aec Executive Yuan 對聚合醣鏈進行放射標誌以作為肝受體造影劑之方法
TWI391144B (zh) 2009-10-26 2013-04-01 Iner Aec Executive Yuan 一種定量肝殘餘功能的檢驗方法與其新穎肝受體造影檢驗藥劑
WO2011072290A2 (en) 2009-12-11 2011-06-16 The Regents Of The University Of Michigan Targeted dendrimer-drug conjugates
US9198972B2 (en) 2010-01-28 2015-12-01 Alnylam Pharmaceuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
PH12012501596A1 (en) 2010-02-24 2017-02-03 Arrowhead Res Corporation Compositions for targeted delivery of sirna
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
US20130109817A1 (en) 2010-03-26 2013-05-02 Mersana Therapeutics, Inc. Modified Polymers for Delivery of Polynucleotides, Method of Manufacture, and Methods of Use Thereof
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
WO2011163121A1 (en) 2010-06-21 2011-12-29 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
US9290760B2 (en) 2010-09-15 2016-03-22 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
WO2012068187A1 (en) 2010-11-19 2012-05-24 Merck Sharp & Dohme Corp. Poly(amide) polymers for the delivery of oligonucleotides
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
RU2013117288A (ru) 2010-12-17 2015-01-27 Эрроухэд Рисерч Корпорейшн СОДЕРЖАЩАЯ ГАЛАКТОЗНЫЙ КЛАСТЕР НАЦЕЛИВАЮЩАЯ ГРУППА ДЛЯ миРНК, МОДУЛИРУЮЩАЯ ФОРМАКОКИНЕТИЧЕСКИЕ СВОЙСТВА
RU2582235C2 (ru) 2010-12-29 2016-04-20 Ф.Хоффманн-Ля Рош Аг Низкомолекулярные конъюгаты для внутриклеточной доставки нуклеиновых кислот
EP2723756B1 (en) 2011-06-21 2020-03-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
CA2842039A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
US10023861B2 (en) 2011-08-29 2018-07-17 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
UA118649C2 (uk) 2011-11-18 2019-02-25 Елнілем Фармасьютікалз, Інк. ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)
EP3919620A1 (en) 2012-05-02 2021-12-08 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
TWI595885B (zh) 2012-05-02 2017-08-21 喜納製藥公司 包含四ｇａｌｎａｃ之新穎結合物及傳送寡核苷酸之方法
RU2624028C2 (ru) * 2012-05-24 2017-06-30 Ионис Фармасьютикалз, Инк. Способы и композиции для модулирования экспрессии аполипопротеина (а)
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
CN103520724B (zh) * 2013-10-23 2016-05-25 江苏美迪森生物医药有限公司 Hsd17b13蛋白或其编码基因的抑制剂的新用途
WO2015106128A2 (en) 2014-01-09 2015-07-16 Alnylam Pharmaceuticals, Inc. MODIFIED RNAi AGENTS
DK3234131T3 (da) 2014-12-16 2020-06-29 Roche Innovation Ct Copenhagen As Fremgangsmåde til screening af kiral toksicitet
CA2991894A1 (en) 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
CN117965536A (zh) 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
AU2017214656B2 (en) 2016-02-05 2022-08-25 Inovio Pharmaceuticals, Inc. Cancer vaccines and methods of treatment using the same
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
CA3059348A1 (en) 2017-04-11 2018-10-18 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
AU2018348195B2 (en) * 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
MY207352A (en) * 2018-03-21 2025-02-21 Regeneron Pharma 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
US20210000906A1 (en) 2018-03-21 2021-01-07 Ionis Pharmaceuticals, Inc. Modulation of hsd17b13 expression
WO2019200172A1 (en) * 2018-04-11 2019-10-17 Ionis Pharmaceuticals, Inc. Modulators of ezh2 expression
US12534728B2 (en) * 2018-09-19 2026-01-27 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of 17beta-HSD type 13 (HSD17B13), compositions thereof, and methods of use
SG11202106378VA (en) 2018-12-21 2021-07-29 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
CA3230382A1 (en) 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
JP2024545602A (ja) 2021-11-19 2024-12-10 ウェイブ ライフ サイエンシズ リミテッド Ｈｓｄ１７ｂ１３関連二本鎖オリゴヌクレオチド組成物及びそれに関連する方法

Also Published As

Publication number Publication date
CA3122432A1 (en) 2020-06-25
CL2021001643A1 (es) 2022-01-21
IL283967B2 (en) 2025-03-01
UA128236C2 (uk) 2024-05-15
IL316212A (en) 2024-12-01
NI202100056A (es) 2021-12-01
SG11202106378VA (en) 2021-07-29
MX2021007503A (es) 2021-08-05
DOP2023000251A (es) 2023-12-29
WO2020132564A1 (en) 2020-06-25
IL283967B1 (en) 2024-11-01
US12409188B2 (en) 2025-09-09
AU2019403447A1 (en) 2021-07-29
JP2022514657A (ja) 2022-02-14
JP7640651B2 (ja) 2025-03-05
DOP2021000130A (es) 2021-09-30
CN113544272A (zh) 2021-10-22
US20230310483A1 (en) 2023-10-05
TW202039846A (zh) 2020-11-01
EP3899024A4 (en) 2023-05-31
AU2019403447B2 (en) 2023-07-27
KR20210107027A (ko) 2021-08-31
PE20211755A1 (es) 2021-09-07
JP7394137B2 (ja) 2023-12-07
PH12021551463A1 (en) 2022-05-02
CO2021009009A2 (es) 2021-07-30
IL283967A (en) 2021-07-29
JP2024037737A (ja) 2024-03-19
AU2023251525A1 (en) 2023-11-16
US20220273691A1 (en) 2022-09-01
BR112021011899A2 (pt) 2021-10-26
EP3899024A1 (en) 2021-10-27
JOP20210158A1 (ar) 2023-01-30
CR20210395A (es) 2021-11-05

Similar Documents

Publication Publication Date Title
CO2021009009A2 (es) Moduladores de la expresión de hsd17b13
NI202100018A (es) Moduladores de la expresión de pnpla3
PH12019502174A1 (en) Modulators of pcsk9 expression
BR112018004620A2 (pt) moduladores da expressão de kras
CO2017013065A2 (es) Moduladores de diaciglicerol aciltransferasa 2 (dgat2)
MX2025003294A (es) Moduladores de la expresion de apol1
CO2020008769A2 (es) Moduladores de la expresión dnm2
MX2021010152A (es) Moduladores de la expresion de malat1.
CO2020003134A2 (es) Moduladores de la expresión de enac
UY38472A (es) Moduladores de la expresión de foxp3
EA202191657A1 (ru) Модуляторы экспрессии hsd17b13
AR117079A1 (es) Moduladores de la expresión de foxp3