AU2007306361A1 - RNA antagonist compounds for the modulation of PCSK9 - Google Patents

RNA antagonist compounds for the modulation of PCSK9 Download PDF

Info

Publication number
AU2007306361A1
AU2007306361A1 AU2007306361A AU2007306361A AU2007306361A1 AU 2007306361 A1 AU2007306361 A1 AU 2007306361A1 AU 2007306361 A AU2007306361 A AU 2007306361A AU 2007306361 A AU2007306361 A AU 2007306361A AU 2007306361 A1 AU2007306361 A1 AU 2007306361A1
Authority
AU
Australia
Prior art keywords
seq
oligomer
pcsk9
nucleobases
sequence
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.)
Abandoned
Application number
AU2007306361A
Other languages
English (en)
Inventor
Niels Fisker Nielsen
Ellen Marie Straarup
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.)
Roche Innovation Center Copenhagen AS
Original Assignee
Santaris Pharma AS
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 Santaris Pharma AS filed Critical Santaris Pharma AS
Publication of AU2007306361A1 publication Critical patent/AU2007306361A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61P1/14—Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/06—Antiasthmatics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • A61P15/16—Masculine contraceptives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
    • A61P3/14—Drugs for disorders of the metabolism for electrolyte homeostasis for calcium homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/10—Drugs for disorders of the endocrine system of the posterior pituitary hormones, e.g. oxytocin, ADH
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21112—Site-1 protease (3.4.21.112), i.e. subtilisin kexin isozyme-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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Endocrinology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Obesity (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Rheumatology (AREA)
  • Neurosurgery (AREA)
  • Pulmonology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
AU2007306361A 2006-10-09 2007-10-09 RNA antagonist compounds for the modulation of PCSK9 Abandoned AU2007306361A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US82873506P 2006-10-09 2006-10-09
US60/828,735 2006-10-09
US97293207P 2007-09-17 2007-09-17
US60/972,932 2007-09-17
US97740907P 2007-10-04 2007-10-04
US60/977,409 2007-10-04
PCT/EP2007/060703 WO2008043753A2 (en) 2006-10-09 2007-10-09 Rna antagonist compounds for the modulation of pcsk9

Publications (1)

Publication Number Publication Date
AU2007306361A1 true AU2007306361A1 (en) 2008-04-17

Family

ID=39125211

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2007306361A Abandoned AU2007306361A1 (en) 2006-10-09 2007-10-09 RNA antagonist compounds for the modulation of PCSK9

Country Status (9)

Country Link
US (1) US20100216864A1 (https=)
EP (2) EP2444494B1 (https=)
JP (1) JP2010505432A (https=)
AU (1) AU2007306361A1 (https=)
CA (1) CA2666191C (https=)
ES (1) ES2603379T3 (https=)
IL (1) IL197668A0 (https=)
MX (1) MX2009003729A (https=)
WO (1) WO2008043753A2 (https=)

Families Citing this family (74)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
JP2010510807A (ja) * 2006-11-27 2010-04-08 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 高コレステロール血症を治療するための方法
CA2681406A1 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S Rna antagonist compounds for the inhibition of apo-b100 expression
WO2009027527A2 (en) * 2007-08-30 2009-03-05 Santaris Pharma A/S Rna antagonist compounds for the modulation of fabp4/ap2
US20110224280A1 (en) * 2008-04-16 2011-09-15 Niels Fisker Nielsen Pharmaceutical Composition Comprising Anti PCSK9 Oligomers
WO2009143367A2 (en) * 2008-05-22 2009-11-26 Schering Corporation Egf-a domain-mediated modulation of pcsk9
WO2009148605A2 (en) * 2008-06-04 2009-12-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
US8563528B2 (en) 2009-07-21 2013-10-22 Santaris Pharma A/S Antisense oligomers targeting PCSK9
EP2490699A1 (en) 2009-10-20 2012-08-29 Santaris Pharma A/S Oral delivery of therapeutically effective lna oligonucleotides
GB2481373A (en) * 2010-06-21 2011-12-28 Weiming Xu Treatment of hypercholesterolaemia by ubiquitination of PCSK9
JP5875006B2 (ja) * 2010-08-31 2016-03-02 国立大学法人大阪大学 オリゴヌクレオチド、およびオリゴヌクレオチドを有効成分として含有する脂質異常症治療剤
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
AU2012205718B2 (en) 2011-01-10 2017-07-06 The Regents Of The University Of Michigan Stem cell factor inhibitor
WO2013001372A2 (en) 2011-06-30 2013-01-03 University Of Oslo Methods and compositions for inhibition of activation of regulatory t cells
MX340536B (es) 2011-07-06 2016-07-13 Sykehuset Sorlandet Hf Terapia direccionada al receptor del factor de crecimiento epidermico (egfr).
HK1200484A1 (zh) 2011-09-14 2015-08-07 Translate Bio Ma, Inc. 多聚寡核苷酸化合物
AU2012353330B2 (en) 2011-12-16 2018-04-19 National University Corporation Tokyo Medical And Dental University Chimeric double-stranded nucleic acid
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
JP2015165772A (ja) * 2012-07-04 2015-09-24 国立大学法人大阪大学 オリゴヌクレオチド、およびオリゴヌクレオチドを有効成分として含有する脂質異常症治療剤
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
ES2762326T5 (es) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Métodos para modular la expresión de C9ORF72
HK1212597A1 (zh) 2012-10-15 2016-06-17 Isis Pharmaceuticals, Inc. 用於調節c90rf72表達的組合物
US10058630B2 (en) 2012-10-22 2018-08-28 Concievalve, Llc Methods for inhibiting stenosis, obstruction, or calcification of a stented heart valve or bioprosthesis
US10077443B2 (en) 2012-11-15 2018-09-18 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
EP2935333A1 (en) 2012-12-21 2015-10-28 Sykehuset Sorlandet HF Egfr targeted therapy of neurological disorders and pain
CN104955952A (zh) 2013-01-30 2015-09-30 弗·哈夫曼-拉罗切有限公司 Lna寡核苷酸碳水化合物缀合物
CA2901983A1 (en) 2013-03-01 2014-09-04 National University Corporation Tokyo Medical And Dental University Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
ES2700277T3 (es) 2013-05-30 2019-02-14 Univ Nat Corp Tokyo Medical & Dental Agentes de doble cadena para el suministro de oligonucleótidos terapéuticos
CN105324119A (zh) 2013-06-16 2016-02-10 国立大学法人东京医科齿科大学 具有外显子跳跃效应的双链反义核酸
ES2770667T3 (es) 2013-06-27 2020-07-02 Roche Innovation Ct Copenhagen As Oligómeros antisentido y conjugados que se dirigen a PCSK9
WO2015006554A1 (en) 2013-07-10 2015-01-15 The Regents Of The University Of Michigan Therapeutic antibodies and uses thereof
EP4166667A3 (en) 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
US10301624B2 (en) 2014-06-25 2019-05-28 The General Hospital Corporation Targeting human satellite II (HSATII)
EP3973958A3 (en) * 2015-03-20 2022-06-22 Aarhus Universitet Inhibitors of pcsk9 for treatment of lipoprotein metabolism disorders
EP3283012A4 (en) * 2015-04-15 2018-11-21 Concievalve LLC Devices and methods for inhibiting stenosis, obstruction, or calcification of a native heart valve, stented heart valve or bioprosthesis
RS60230B1 (sr) 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
WO2017142054A1 (ja) 2016-02-17 2017-08-24 国立大学法人東京工業大学 人工ヌクレオシド及び人工ヌクレオチド並びに人工オリゴヌクレオチド
EP3517610A4 (en) 2016-09-23 2020-05-27 National University Corporation Tokyo Medical and Dental University THROUGH HETERODUPLEX NUCLEIC ACID THROUGH BLOOD BRAIN CABINET
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
CN110337495B (zh) 2017-02-06 2024-03-29 日产化学株式会社 单链寡核苷酸
US11273222B2 (en) 2017-05-26 2022-03-15 National Cerebral And Cardiovascular Center Antisense nucleic acid targeting PCSK9
WO2019004420A1 (ja) 2017-06-30 2019-01-03 国立大学法人東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
AU2018307294B2 (en) 2017-07-26 2025-04-17 Nissan Chemical Corporation Single-stranded oligonucleotide
JP7360170B2 (ja) 2018-02-28 2023-10-12 国立大学法人 東京医科歯科大学 虚血病変部位特異的な遺伝子治療法
EP3766972B1 (en) 2018-03-14 2025-04-23 Institute of Science Tokyo Nucleic acid complex
US11851654B2 (en) 2018-03-19 2023-12-26 National University Corporation Tokyo Medical And Dental University Nucleic acid with reduced toxicity
MX2020009765A (es) 2018-03-20 2021-01-08 Tokyo Inst Tech Oligonucleotido antisentido reducido en toxicidad.
JP7262816B2 (ja) 2018-03-22 2023-04-24 国立大学法人 東京医科歯科大学 ヘテロ核酸のｂｂｂ通過脂質リガンド
US20210308170A1 (en) 2018-07-27 2021-10-07 Osaka University Composition for suppression of aging, prevention, amelioration, or treatment of an age-related disease or symptom, or extension of lifespan
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
JP7530106B2 (ja) 2019-03-14 2024-08-07 レナセラピューティクス株式会社 Ｉｈｈ発現を調節するための核酸複合体
EP3954395A4 (en) 2019-04-08 2023-06-28 National University Corporation Tokyo Medical and Dental University Pharmaceutical composition for muscle disease treatment
US20220370491A1 (en) 2019-09-18 2022-11-24 National University Corporation Tokyo Medical And Dental University Nucleic acid complex
JP7793144B2 (ja) 2019-10-11 2026-01-05 国立大学法人東京科学大学 修飾ヘテロ核酸
EP4098747A4 (en) 2020-01-31 2025-03-26 Sanwa Kagaku Kenkyusho Co., Ltd. ATN1 ANTISENSE OLIGONUCLEOTIDE
JPWO2021177418A1 (https=) 2020-03-04 2021-09-10
US20230174981A1 (en) 2020-03-16 2023-06-08 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing morpholino nucleic acid
US20240117356A1 (en) 2020-12-31 2024-04-11 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20240240183A1 (en) 2021-05-25 2024-07-18 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing scpBNA or AmNA
CA3222167A1 (en) 2021-05-31 2022-12-08 Rena Therapeutics Inc. Ligand-binding nucleic acid complex
US20250339459A1 (en) 2021-08-19 2025-11-06 National University Corporation Tokyo Medical And Dental University Modified heteronucleic acid containing morpholino nucleic acid
US20240390508A1 (en) 2021-08-21 2024-11-28 Takeda Pharmaceutical Company Limited Human transferrin receptor binding peptide-drug conjugate
WO2023042876A1 (ja) 2021-09-15 2023-03-23 国立大学法人東京医科歯科大学 ２'－修飾ヌクレオシドを含むヘテロ核酸
JPWO2023080159A1 (https=) 2021-11-02 2023-05-11
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
JPWO2024071099A1 (https=) 2022-09-29 2024-04-04
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
WO2025047953A1 (ja) 2023-08-30 2025-03-06 株式会社Stratoimmune ＲａｓＧＲＰ４のアンチセンスオリゴヌクレオチド

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3719766A1 (de) * 1987-06-13 1988-12-22 Heidelberger Druckmasch Ag Registermesssystem
US4914210A (en) * 1987-10-02 1990-04-03 Cetus Corporation Oligonucleotide functionalizing reagents
US6423489B1 (en) * 1992-09-10 2002-07-23 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of Hepatitis C virus-associated diseases
CA2143678A1 (en) * 1992-09-10 1994-03-17 Kevin P. Anderson Compositions and methods for treatment of hepatitis c virus-associated diseases
US6433159B1 (en) * 1992-09-10 2002-08-13 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of Hepatitis C virus associated diseases
ATE233734T1 (de) * 1995-06-07 2003-03-15 Pfizer Biphenyl-2-carbonsäure-tetrahydro-isochinolin-6 yl amid derivate, deren hestellung und deren verwendung als inhibitoren des mikrosomalen triglycerid-transfer-proteins und/oder der apolipoprotein b (apo b) sekretion
NZ335162A (en) * 1996-11-27 2000-01-28 Pfizer Apo b-secretion/mtp inhibitory amides
JP2001514508A (ja) * 1997-03-05 2001-09-11 ユニバーシティー オブ ワシントン Ｃ型肝炎ウイルスの複製を選択的に抑制する薬剤を同定するための新規なスクリーニング法
US6410323B1 (en) * 1999-08-31 2002-06-25 Isis Pharmaceuticals, Inc. Antisense modulation of human Rho family gene expression
US7029895B2 (en) * 1999-09-27 2006-04-18 Millennium Pharmaceuticals, Inc. 27411, a novel human PGP synthase
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
CA2399727A1 (en) 2000-02-07 2001-08-09 Millennium Pharmaceuticals, Inc. Narc-1, subtilase-like homologs
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
WO2003085110A2 (en) * 2002-04-05 2003-10-16 Santaris Pharma A/S Oligomeric compounds for the modulation hif-1alpha expression
WO2003097662A1 (en) 2002-05-15 2003-11-27 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein b expression
WO2004014852A2 (en) * 2002-08-12 2004-02-19 Bristol-Myers Squibb Company Iminothiazolidinones as inhibitors of hcv replication
US7087229B2 (en) * 2003-05-30 2006-08-08 Enzon Pharmaceuticals, Inc. Releasable polymeric conjugates based on aliphatic biodegradable linkers
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
US9045518B2 (en) 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
FR2848572B1 (fr) * 2002-12-12 2005-12-09 Univ Joseph Fourier Molecules inhibitrices de la synthese proteique du virus de l'hepatite c et procede de criblage desdites molecules inhibitrices
EP1471152A1 (en) 2003-04-25 2004-10-27 Institut National De La Sante Et De La Recherche Medicale (Inserm) Mutations in the human PCSK9 gene associated to hypercholesterolemia
US7683036B2 (en) * 2003-07-31 2010-03-23 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
WO2005073378A1 (en) 2004-01-30 2005-08-11 Santaris Pharma A/S MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)
PL1786472T3 (pl) * 2004-08-10 2013-06-28 Kastle Therapeutics Llc Antysensowna modulacja ekspresji apolipoproteiny B
US20060185027A1 (en) * 2004-12-23 2006-08-17 David Bartel Systems and methods for identifying miRNA targets and for altering miRNA and target expression
AU2007275365A1 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. RNA interference mediated inhibition of Proprotein Convertase Subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)

Also Published As

Publication number Publication date
ES2603379T3 (es) 2017-02-27
CA2666191C (en) 2017-07-11
WO2008043753A8 (en) 2008-06-26
CA2666191A1 (en) 2008-04-17
MX2009003729A (es) 2009-04-22
EP2444494A1 (en) 2012-04-25
WO2008043753A3 (en) 2008-09-25
EP2076597A2 (en) 2009-07-08
JP2010505432A (ja) 2010-02-25
EP2444494B1 (en) 2016-09-28
IL197668A0 (en) 2011-08-01
WO2008043753A2 (en) 2008-04-17
US20100216864A1 (en) 2010-08-26

Similar Documents

Publication Publication Date Title
CA2666191C (en) Rna antagonist compounds for the modulation of pcsk9
AU2006291836B2 (en) RNA antagonist compounds for the inhibition of Apo-Bl00 expression
US8563528B2 (en) Antisense oligomers targeting PCSK9
EP2225377B1 (en) Lna antagonists targeting the androgen receptor
EP2225376B1 (en) Rna antagonist compounds for the modulation of pik3ca expression
US20110224280A1 (en) Pharmaceutical Composition Comprising Anti PCSK9 Oligomers
CN102076854A (zh) 用于抑制线粒体3-磷酸甘油酰基转移酶1(mtgpat1)表达的rna拮抗剂化合物
WO2010007522A1 (en) Rna antagonists targeting gli2
EP2440215B1 (en) New potent anti apob antisense compounds
US20110054011A1 (en) RNA Antagonist Compounds for the Modulation of FABP4/AP2
EP2490767A1 (en) Rna antagonists targeting gli2 for the treatment of leukemia
WO2009153302A2 (en) Rna antagonists targeting hsp70-2
MX2008003198A (es) Compuestos antagonistas de acido ribonucleico (arn) para la inhibicion de expresion apo-b1oo

Legal Events

Date Code Title Description
CB4 Opposition filed under section 104(4)

Opponent name: PREFERENCE MANUFACTURING (AUST) PTY LTD

MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application