ES2844593T3 - Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3 - Google Patents

Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3 Download PDF

Info

Publication number
ES2844593T3
ES2844593T3 ES15786207T ES15786207T ES2844593T3 ES 2844593 T3 ES2844593 T3 ES 2844593T3 ES 15786207 T ES15786207 T ES 15786207T ES 15786207 T ES15786207 T ES 15786207T ES 2844593 T3 ES2844593 T3 ES 2844593T3
Authority
ES
Spain
Prior art keywords
vol
antisense
chem
compound
angptl3
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
ES15786207T
Other languages
English (en)
Spanish (es)
Inventor
Thazha P Prakash
Punit P Seth
Eric E Swayze
Susan M Freier
Mark J Graham
Rosanne M Crooke
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
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
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Application granted granted Critical
Publication of ES2844593T3 publication Critical patent/ES2844593T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
    • 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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/555—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound pre-targeting systems involving an organic compound, other than a peptide, protein or antibody, for targeting specific cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/12—Antihypertensives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N2310/33—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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Emergency Medicine (AREA)
  • Child & Adolescent Psychology (AREA)
ES15786207T 2014-05-01 2015-05-01 Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3 Active ES2844593T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461987467P 2014-05-01 2014-05-01
US201462049230P 2014-09-11 2014-09-11
PCT/US2015/028837 WO2015168589A2 (en) 2014-05-01 2015-05-01 Compositions and methods for modulating angiopoietin-like 3 expression

Publications (1)

Publication Number Publication Date
ES2844593T3 true ES2844593T3 (es) 2021-07-22

Family

ID=54354816

Family Applications (1)

Application Number Title Priority Date Filing Date
ES15786207T Active ES2844593T3 (es) 2014-05-01 2015-05-01 Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3

Country Status (18)

Country Link
US (4) US9382540B2 (enExample)
EP (2) EP3137605B1 (enExample)
JP (4) JP2017521045A (enExample)
KR (1) KR102356388B1 (enExample)
CN (2) CN111961669A (enExample)
AU (2) AU2015252895B2 (enExample)
BR (2) BR122020024443B1 (enExample)
CA (1) CA2946003A1 (enExample)
DK (1) DK3137605T3 (enExample)
ES (1) ES2844593T3 (enExample)
HU (1) HUE052931T2 (enExample)
IL (1) IL248269B (enExample)
MX (2) MX382230B (enExample)
PL (1) PL3137605T3 (enExample)
PT (1) PT3137605T (enExample)
RU (1) RU2734658C2 (enExample)
SI (1) SI3137605T1 (enExample)
WO (1) WO2015168589A2 (enExample)

Families Citing this family (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
JO3412B1 (ar) 2011-06-17 2019-10-20 Regeneron Pharma أجسام مضادة ل angptl3 واستخداماتها
EP3444348A1 (en) 2011-06-21 2019-02-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2839006B1 (en) * 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
DK2872485T3 (da) 2012-07-13 2021-03-08 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
CA2921162A1 (en) 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
HUE052243T2 (hu) * 2013-12-24 2021-04-28 Ionis Pharmaceuticals Inc Az angiopoietin-szerû 3 expressziójának modulációja
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
KR102423317B1 (ko) 2014-01-16 2022-07-22 웨이브 라이프 사이언시스 리미티드 키랄 디자인
AU2015252841B2 (en) 2014-05-01 2020-03-19 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
MX382230B (es) * 2014-05-01 2025-03-13 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3.
US20170327524A1 (en) 2014-10-10 2017-11-16 Hoffmann-La Roche, Inc. Galnac phosphoramidites, nucleic acid conjugates thereof and their use
KR20250005546A (ko) 2015-04-13 2025-01-09 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법
WO2016209862A1 (en) * 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
PL3331892T3 (pl) 2015-08-06 2019-11-29 Hoffmann La Roche Sposób wytwarzania pochodnych kwasu GalNAc
EP3359523A4 (en) 2015-10-09 2019-07-24 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR
EP3370708A4 (en) * 2015-11-06 2019-06-26 Ionis Pharmaceuticals, Inc. MODULATION OF APOLIPOPROTEIN (A) EXPRESSION
SI4119569T1 (sl) * 2015-11-06 2024-10-30 Ionis Pharmaceuticals, Inc. Konjugirane protismiselne spojine za uporabo v terapiji
ES2848122T3 (es) * 2015-11-16 2021-08-05 Hoffmann La Roche Fosforamidita de agrupación de GalNAc
MX2018010401A (es) 2016-03-03 2019-03-06 Regeneron Pharma Metodos para el tratamiento de pacientes con hiperlipidemia administrando un inhibidor de pcsk9 en combinacion con un inhibidor de angptl3.
EP3228326A1 (en) 2016-04-05 2017-10-11 Silence Therapeutics GmbH Nucleic acid linked to a trivalent glycoconjugate
AU2017258105B2 (en) * 2016-04-28 2024-09-05 Regeneron Pharmaceuticals, Inc. Methods for treating patients with familial hypercholesterolemia
EP3484908A4 (en) 2016-07-15 2020-04-08 AM Chemicals Llc NON-NUCLEOSIDIC SOLID CARRIERS AND PHOSPHORAMIDITE BUILDING BLOCKS FOR OLIGONUCLEOTID SYNTHESIS
WO2018035380A1 (en) 2016-08-17 2018-02-22 Solstice Biologics, Ltd. Polynucleotide constructs
KR102645243B1 (ko) * 2016-11-23 2024-03-11 알닐람 파마슈티칼스 인코포레이티드 표적외 효과가 감소된 변형 rna 작용제
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
CN119351474A (zh) * 2017-02-22 2025-01-24 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
WO2018215049A1 (en) 2017-05-23 2018-11-29 F. Hoffmann-La Roche Ag Process for galnac oligonucleotide conjugates
CN111050806A (zh) * 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
WO2019006455A1 (en) 2017-06-30 2019-01-03 Solstice Biologics, Ltd. AUXILIARIES OF CHIRAL PHOSPHORAMIDITIS AND METHODS OF USE THEREOF
US11965004B2 (en) 2017-07-10 2024-04-23 Sri International Molecular guide system peptides and uses thereof
CN118240025A (zh) 2017-07-10 2024-06-25 斯坦福国际研究院 用于治疗癌症的肽皂草素缀合物
PE20201287A1 (es) * 2017-09-14 2020-11-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso
CN118236391A (zh) 2017-12-01 2024-06-25 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110997919B (zh) 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
JP7261494B2 (ja) 2017-12-01 2023-04-20 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
WO2019105435A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
US11660347B2 (en) 2017-12-01 2023-05-30 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
WO2019127004A1 (zh) * 2017-12-26 2019-07-04 广州市锐博生物科技有限公司 修饰的寡核苷酸和可用于合成修饰的寡核苷酸的化合物
CN110959011B (zh) 2017-12-29 2023-03-28 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
WO2019217397A2 (en) 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for improving strand biased
JP7438135B2 (ja) * 2018-05-09 2024-02-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｆｘｉの発現を低下させるための化合物及び方法
SG11202010131QA (en) 2018-05-11 2020-11-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof
EP3842534A4 (en) 2018-08-21 2022-07-06 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND CONJUGATE WITH NUCLEIC ACID AND THEIR USE
EP3862024A4 (en) 2018-09-30 2022-08-17 Suzhou Ribo Life Science Co., Ltd. SIRNA CONJUGATE, METHOD FOR ITS PRODUCTION AND ITS USE
AU2019377275A1 (en) * 2018-11-09 2021-03-25 Novartis Ag Method for reducing the risk of a cardiovascular event with conjugated antisense compounds targeting apo(a)
WO2020099482A2 (en) * 2018-11-13 2020-05-22 Lipigon Pharmaceuticals Ab Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism
CN112423794A (zh) 2018-12-28 2021-02-26 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
JP2022519184A (ja) 2019-01-22 2022-03-22 コロ バイオ， インコーポレイテッド Ｒｎａ編集オリゴヌクレオチド及びその使用
CA3126947A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
JP7663951B2 (ja) 2019-05-22 2025-04-17 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
CN113811613B (zh) 2019-05-22 2024-04-05 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
WO2020238766A1 (zh) 2019-05-24 2020-12-03 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
US12559551B2 (en) 2019-05-24 2026-02-24 Regeneron Pharmaceuticals, Inc. Stabilized formulations containing anti-ANGPTL3 antibodies
US20220380764A1 (en) * 2019-10-01 2022-12-01 Sri International Molecular guide system peptides and uses thereof
CN111067913B (zh) * 2019-12-11 2021-02-05 大连医科大学 薯蓣皂苷在制备肺动脉高压保护药物中的应用
US20230287425A1 (en) * 2020-03-18 2023-09-14 Dicerna Pharmacuticals Inc. Compositions and methods for inhibiting angptl3 expression
CN116333013B (zh) * 2020-09-30 2025-09-02 纳肽得(青岛)生物医药有限公司 一种靶向配体
JP7820374B2 (ja) * 2020-11-18 2026-02-25 アイオーニス ファーマシューティカルズ， インコーポレーテッド アンジオテンシノーゲン発現を調節するための化合物及び方法
WO2022178146A1 (en) * 2021-02-18 2022-08-25 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing nlrp3 expression
AU2022231003A1 (en) 2021-03-04 2023-09-14 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
CA3241896A1 (en) 2021-12-22 2023-06-29 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors
US12037616B2 (en) 2022-03-01 2024-07-16 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (ANGPTL3) related conditions
WO2024102913A1 (en) 2022-11-10 2024-05-16 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with a combination of angiopoietin like 3 (angptl3) inhibitors and solute carrier family 5 member 2 (slc5a2) inhibitors
WO2024145615A2 (en) * 2022-12-30 2024-07-04 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of angptl3 expression
WO2024164008A1 (en) * 2023-02-03 2024-08-08 Sirnaomics, Inc. Products and compositions
WO2025232842A1 (zh) * 2024-05-09 2025-11-13 石药集团中奇制药技术（石家庄）有限公司 一种抑制angptl3基因表达的核酸分子

Family Cites Families (216)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US101207A (en) 1870-03-29 Improvement in screw and screw-driver
US2699508A (en) 1951-12-21 1955-01-11 Selectronics Inc Method of mounting and construction of mounting for low frequency piezoelectric crystals
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
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
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
DE3851889T2 (de) 1987-06-24 1995-04-13 Florey Howard Inst Nukleosid-derivate.
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
DK0942000T3 (da) 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US5223618A (en) 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
DE69126530T2 (de) 1990-07-27 1998-02-05 Isis Pharmaceutical, Inc., Carlsbad, Calif. Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
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
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
EP1256589A3 (en) 1991-11-26 2003-09-17 Isis Pharmaceuticals, Inc. 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
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
AU678769B2 (en) 1992-07-27 1997-06-12 Hybridon, Inc. Oligonucleotide alkylphosphonothioates
AU6165494A (en) 1993-01-25 1994-08-15 Hybridon, Inc. Oligonucleotide alkylphosphonates and alkylphosphonothioates
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304620D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Compounds
WO1994022864A1 (en) 1993-03-30 1994-10-13 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
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
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US6172045B1 (en) 1994-12-07 2001-01-09 Neorx Corporation Cluster clearing agents
US6908903B1 (en) 1994-12-07 2005-06-21 Aletheon Pharmaceuticals, Inc. Cluster clearing agents
JP2000501414A (ja) 1995-11-22 2000-02-08 ザ・ジョンズ・ホプキンス・ユニバーシティー 生体分子の細胞取り込みを高めるリガンド
US20030119724A1 (en) 1995-11-22 2003-06-26 Ts`O Paul O.P. Ligands to enhance cellular uptake of biomolecules
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US20080119427A1 (en) 1996-06-06 2008-05-22 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
EP0950665A4 (en) 1996-09-26 2004-05-06 Ajinomoto Kk PHYSIOLOGICALLY ACTIVE MODIFIED PROTEINS AND DRUG COMPOSITIONS CONTAINING THEM
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6300319B1 (en) 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US6166239A (en) 1998-09-04 2000-12-26 Isis Pharmaceuticals, Inc. Oligonucleotide protecting groups
US20030170249A1 (en) 1999-02-19 2003-09-11 Hakomori Sen-Itiroh Vaccines directed to cancer-associated carbohydrate antigens
CA2370628A1 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As L-ribo-LNA analogues
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
US20080281041A1 (en) 1999-06-07 2008-11-13 Rozema David B Reversibly Masked Polymers
US8541548B2 (en) 1999-06-07 2013-09-24 Arrowhead Madison Inc. Compounds and methods for reversible modification of biologically active molecules
AU6605900A (en) 1999-07-16 2001-02-05 Hyseq, Inc. Novel angiopoietin materials and methods
MXPA02005758A (es) 1999-12-09 2002-09-18 Sankyo Co Metodo para evaluar un remedio o agente preventivo para hiperlipidemia.
ATE322493T1 (de) 1999-12-30 2006-04-15 Leuven K U Res & Dev Cyclohexennukleinsäuren
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
WO2002043771A2 (en) 2000-12-01 2002-06-06 Cell Works Inc. Conjugates of glycosylated/galactosylated peptide
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
EP1403367A4 (en) 2001-06-08 2005-08-17 Sankyo Co A MEDICAMENT TEST METHOD FOR TREATING OR PREVENTING DISEASES SUCH AS HYPERLIPEMIA
JP2005504020A (ja) 2001-07-03 2005-02-10 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 ヌクレアーゼ耐性キメラオリゴヌクレオチド
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
US7259150B2 (en) 2001-08-07 2007-08-21 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein (a) expression
US7439043B2 (en) 2001-10-10 2008-10-21 Neose Technologies, Inc. Galactosyl nucleotide sugars
ES2429034T3 (es) 2001-11-16 2013-11-12 Genentech, Inc. Uso de antagonistas de ANGPTL3 para el tratamiento de enfermedades hepáticas
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)
AU2003260289A1 (en) 2002-09-11 2004-04-30 Santaris Pharma A/S Modified pna molecules
CA2502649A1 (en) 2002-10-18 2004-04-29 Nucleonics Inc. Double-stranded rna structures and constructs, and methods for generating and using the same
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004043979A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20060009410A1 (en) 2002-11-13 2006-01-12 Crooke Rosanne M Effects of apolipoprotein B inhibition on gene expression profiles in animals
US7511131B2 (en) 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
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
JP4152955B2 (ja) 2003-01-09 2008-09-17 ポステック・ファウンデーション 新規フォスフォアミダイト化合物
WO2004072046A2 (en) * 2003-02-12 2004-08-26 Carex S.A. Quinoline derivatives and their use for modulation of lxr activity
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
JP4597976B2 (ja) 2003-04-17 2010-12-15 アルナイラム ファーマシューティカルズ インコーポレイテッド 修飾ｉＲＮＡ剤
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
JPWO2004101619A1 (ja) 2003-05-15 2006-10-26 塩野義製薬株式会社 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
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
JPWO2005097155A1 (ja) 2004-04-08 2008-02-28 タカラバイオ株式会社 神経突起伸長誘導剤
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
EP1771474B1 (en) * 2004-07-20 2010-01-27 Genentech, Inc. Inhibitors of angiopoietin-like 4 protein, combinations, and their use
CA2574791A1 (en) 2004-07-20 2006-02-09 Genentech, Inc. Compositions and methods of using angiopoietin-like 4 protein
JP5192234B2 (ja) 2004-08-10 2013-05-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド 化学修飾オリゴヌクレオチド
DK1786472T3 (da) 2004-08-10 2013-04-15 Genzyme Corp Antisense-modulering af apolipoprotein B-ekspression
US20090203132A1 (en) 2004-09-09 2009-08-13 Swayze Eric E Pyrrolidinyl groups for attaching conjugates to oligomeric compounds
EP1812569A2 (en) 2004-11-08 2007-08-01 K.U. Leuven Research and Development Modified nucleosides for rna interference
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
JP2008527993A (ja) 2005-01-24 2008-07-31 アヴァリス・アクチエボラーグ 特異性及び送達の改善のためのｓｉＲＮＡ分子、ｓｈＲＮＡ分子又はアンチセンス分子及び機能的実体を含む複合体
CA2623772A1 (en) 2005-09-19 2007-03-29 Johnson & Johnson Pharmaceutical Research & Development, L.L.C. Modulation of glucocorticoid receptor expression
ES2351674T3 (es) 2006-01-27 2011-02-09 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6.
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
DK2021472T3 (da) 2006-05-05 2011-09-19 Isis Pharmaceuticals Inc Forbindelser og fremgangsmåder til modulering af genekspression
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
JP2010500290A (ja) 2006-08-04 2010-01-07 アイシス ファーマシューティカルズ， インコーポレーテッド Ｊｎｋタンパク質を調節するための組成物および方法
US8658211B2 (en) 2006-08-18 2014-02-25 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
WO2008073300A2 (en) 2006-12-08 2008-06-19 Lexicon Pharmaceuticals, Inc. Monoclonal antibodies against angptl3
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
EP2604283A1 (en) 2007-02-16 2013-06-19 KTB Tumorforschungsgesellschaft mbH Receptor And Antigen Targeted Prodrug
CA2685127C (en) 2007-04-23 2019-01-08 Alnylam Pharmaceuticals, Inc. Glycoconjugates of rna interference agents
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
US20090004140A1 (en) 2007-06-26 2009-01-01 Yao-Ling Qiu 4-substituted pyrrolidine as anti-infectives
EP2176280B2 (en) 2007-07-05 2015-06-24 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
BRPI0907008A2 (pt) 2008-01-31 2015-07-07 Alnylam Pharmaceuticals Inc Métodos otimizados para liberação de dsrna alvejando o gene pcsk9
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009142822A2 (en) 2008-03-26 2009-11-26 Alnylam Pharmaceuticals, Inc. 2-f modified rna interference agents
EP3604533A1 (en) 2008-04-11 2020-02-05 Arbutus Biopharma Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
WO2009143369A2 (en) 2008-05-22 2009-11-26 Isis Pharmaceuticals, Inc. Method of preparing nucleosides and analogs thereof without using chromatography
EP2342616A2 (en) 2008-09-23 2011-07-13 Alnylam Pharmaceuticals Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
US8501805B2 (en) 2008-09-24 2013-08-06 Isis Pharmaceuticals, Inc. Substituted alpha-L-bicyclic nucleosides
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
CA2741294C (en) 2008-10-24 2018-04-24 Isis Pharmaceuticals, Inc. 5' and 2' bis-substituted nucleosides and oligomeric compounds prepared therefrom
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
EP4241767B1 (en) 2008-11-10 2024-10-23 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
WO2010083615A1 (en) 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
EP3424939A1 (en) 2009-03-02 2019-01-09 Alnylam Pharmaceuticals Inc. Nucleic acid chemical modifications
FR2943060B1 (fr) 2009-03-13 2013-01-04 Commissariat Energie Atomique Agents chelatants d'ions metalliques, leurs procedes de preparation et leurs applications
SG176553A1 (en) 2009-05-05 2012-01-30 Muthiah Manoharan Lipid compositions
TR201811076T4 (tr) 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
AU2010260148A1 (en) 2009-06-15 2012-02-02 Alnylam Pharmaceuticals, Inc. Lipid formulated dsRNA targeting the PCSK9 gene
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011005861A1 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
TWI458493B (zh) 2009-09-25 2014-11-01 Iner Aec Executive Yuan 新穎肝標靶藥劑與合成方法
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
CN111700901A (zh) * 2010-01-08 2020-09-25 Ionis制药公司 血管生成素样3表达的调节
WO2011100131A2 (en) 2010-01-28 2011-08-18 Alnylam Pharmacuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
EP3023495B1 (en) 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Compositions for targeted delivery of sirna
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2553019A1 (en) 2010-03-26 2013-02-06 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
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
EP2601204B1 (en) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
US8603994B2 (en) 2010-11-11 2013-12-10 Valted, Llc Transcriptional repression leading to Parkinson's disease
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
BR112013013434A2 (pt) 2010-12-17 2016-08-16 Arrowhead Res Corp metade que tem por alvo modulador fármaco cinético em cacho de galactose para sirna
ES2605990T3 (es) 2010-12-29 2017-03-17 F. Hoffmann-La Roche Ag Conjugados de molécula pequeña para la administración intracelular de ácidos nucleicos
RS58489B1 (sr) 2011-04-01 2019-04-30 Ionis Pharmaceuticals Inc Modulacija eksprimiranja transduktora signala i aktivatora transkripcije 3 (stat3)
US8642752B2 (en) 2011-04-21 2014-02-04 Isis Pharmaceuticals, Inc. Modulation of Hepatitis B virus (HBV) expression
TW201303013A (zh) 2011-04-21 2013-01-16 Isis Pharmaceuticals Inc Ｂ型肝炎病毒（ｈｂｖ）表現之調節
US9157082B2 (en) 2011-04-27 2015-10-13 Isis Pharmaceuticals, Inc. Modulation of apolipoprotein CIII (ApoCIII) expression
KR20230084331A (ko) 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
EP3444348A1 (en) * 2011-06-21 2019-02-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
US20130017250A1 (en) 2011-07-15 2013-01-17 The Trustees Of Columbia University In The City Of New York Methods For High Density Lipoprotein Cholesterol Regulation
KR20140051357A (ko) 2011-08-26 2014-04-30 애로우헤드 리서치 코오포레이션 Ｉｎ Ｖｉｖｏ 핵산 전달용 폴리(비닐 에스테르) 고분자
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
MY167390A (en) 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
CA2863958A1 (en) 2012-02-08 2013-08-15 Isis Pharmaceuticals, Inc. Methods and compositions for modulating factor vii expression
WO2013142571A2 (en) 2012-03-20 2013-09-26 Cornell University Assays for the identification of compounds that modulate lipid homeostasis
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
TW201808342A (zh) 2012-05-02 2018-03-16 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
EP3358013B1 (en) 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
WO2013173789A2 (en) 2012-05-17 2013-11-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
HUE051680T2 (hu) 2012-05-24 2021-03-29 Ionis Pharmaceuticals Inc Apolipoprotein(a) expressziójának módosítására szolgáló eljárások és készítmények
SG10201804331TA (en) 2012-11-15 2018-07-30 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
US20150368642A1 (en) 2013-01-30 2015-12-24 Hoffmann-La Roche Inc. Lna oligonucleotide carbohydrate conjugates
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
CA2921162A1 (en) 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
AU2014300981B2 (en) 2013-06-27 2017-08-10 Ricc A/S Antisense oligomers and conjugates targeting PCSK9
MX382230B (es) * 2014-05-01 2025-03-13 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3.
JP6394289B2 (ja) 2014-11-04 2018-09-26 富士通株式会社 蒸発器、冷却装置、及び電子機器
US10120707B2 (en) 2015-12-08 2018-11-06 Paypal, Inc. Deployment of development environments

Also Published As

Publication number Publication date
HUE052931T2 (hu) 2021-05-28
RU2016146817A (ru) 2018-06-01
RU2016146817A3 (enExample) 2018-12-26
JP2020096623A (ja) 2020-06-25
CN106255755B (zh) 2020-07-24
KR102356388B1 (ko) 2022-01-26
US9957292B2 (en) 2018-05-01
MX382230B (es) 2025-03-13
US20150315594A1 (en) 2015-11-05
EP3137605A4 (en) 2017-12-27
DK3137605T3 (da) 2020-12-14
JP2017521045A (ja) 2017-08-03
JP2021087441A (ja) 2021-06-10
JP2020074784A (ja) 2020-05-21
US10875884B2 (en) 2020-12-29
AU2015252895B2 (en) 2021-07-15
EP3137605A2 (en) 2017-03-08
WO2015168589A2 (en) 2015-11-05
BR122020024443B1 (pt) 2022-02-22
BR112016024850B1 (pt) 2021-07-06
PL3137605T3 (pl) 2021-04-19
KR20160147803A (ko) 2016-12-23
AU2015252895A1 (en) 2016-10-20
RU2734658C2 (ru) 2020-10-21
CN111961669A (zh) 2020-11-20
US20160194349A1 (en) 2016-07-07
JP6842578B2 (ja) 2021-03-17
MX2021005187A (es) 2021-07-15
WO2015168589A3 (en) 2015-12-30
IL248269A0 (en) 2016-11-30
EP3845547A1 (en) 2021-07-07
BR112016024850A2 (pt) 2017-10-24
SI3137605T1 (sl) 2021-02-26
US20190016748A1 (en) 2019-01-17
CA2946003A1 (en) 2015-11-05
PT3137605T (pt) 2020-12-18
IL248269B (en) 2020-08-31
MX2016014102A (es) 2017-05-03
AU2021240211A1 (en) 2021-10-28
US20220324900A1 (en) 2022-10-13
EP3137605B1 (en) 2020-10-28
US9382540B2 (en) 2016-07-05
CN106255755A (zh) 2016-12-21

Similar Documents

Publication Publication Date Title
EP3137605B1 (en) Compositions and methods for modulating angiopoietin-like 3 expression
ES2830257T3 (es) Modulación de la expresión de la proteína de tipo angiopoyetina 3
ES2819213T3 (es) Composiciones y métodos para modular la expresión de apolipoproteína (a)
ES2677969T3 (es) Modulación de la expresión tipo angiopoyetina 3
US10364433B2 (en) Modulation of AGPAT5 expression
HK40045338A (en) Galnac3 conjugated modified oligonucleotide for modulating angiopoietin-like 3 expression
HK1234435B (en) Compositions and methods for modulating angiopoietin-like 3 expression
HK1234435A1 (en) Compositions and methods for modulating angiopoietin-like 3 expression
HK40042399A (en) Compositions and methods for modulating angiopoietin-like 3 expression
HK40033664A (en) Modulation of angiopoietin-like 3 expression
HK1230644A1 (en) Modulation of angiopoietin-like 3 expression
HK1230644B (en) Modulation of angiopoietin-like 3 expression
HK1221403B (en) Compositions and methods for modulating apolipoprotein c-iii expression