BRPI0920263A2 - composto, uso do composto, composição, e, método - Google Patents

composto, uso do composto, composição, e, método

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Publication number
BRPI0920263A2
BRPI0920263A2 BRPI0920263A BRPI0920263A BRPI0920263A2 BR PI0920263 A2 BRPI0920263 A2 BR PI0920263A2 BR PI0920263 A BRPI0920263 A BR PI0920263A BR PI0920263 A BRPI0920263 A BR PI0920263A BR PI0920263 A2 BRPI0920263 A2 BR PI0920263A2
Authority
BR
Brazil
Prior art keywords
compound
composition
Prior art date
Application number
BRPI0920263A
Other languages
English (en)
Inventor
Andrew M Siwkowski
Brett P Monia
Chenguang Zhao
Hong Zhang
Jeffrey R Crosby
Susan M Freier
Original Assignee
Isis 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 Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of BRPI0920263A2 publication Critical patent/BRPI0920263A2/pt
Publication of BRPI0920263B1 publication Critical patent/BRPI0920263B1/pt

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/365Lactones
    • A61K31/366Lactones having six-membered rings, e.g. delta-lactones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4365Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system having sulfur as a ring hetero atom, e.g. ticlopidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • A61K31/4523Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems
    • A61K31/4545Non condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. pipamperone, anabasine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/726Glycosaminoglycans, i.e. mucopolysaccharides
    • A61K31/727Heparin; Heparan
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/737Sulfated polysaccharides, e.g. chondroitin sulfate, dermatan sulfate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • AHUMAN NECESSITIES
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    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/14Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
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    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21Serine endopeptidases (3.4.21)
    • C12Y304/21027Coagulation factor XIa (3.4.21.27)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Dermatology (AREA)
  • Virology (AREA)
  • Vascular Medicine (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pulmonology (AREA)
BRPI0920263-3A 2008-10-15 2009-10-15 Compostos compreendendo um oligonucleotídeo modificado, composição e uso dos mesmos BRPI0920263B1 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10577208P 2008-10-15 2008-10-15
US61/105,772 2008-10-15
US17446109P 2009-04-30 2009-04-30
US61/174,461 2009-04-30
PCT/US2009/060922 WO2010045509A2 (en) 2008-10-15 2009-10-15 Modulation of factor 11 expression

Publications (2)

Publication Number Publication Date
BRPI0920263A2 true BRPI0920263A2 (pt) 2016-07-12
BRPI0920263B1 BRPI0920263B1 (pt) 2021-09-21

Family

ID=42107256

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0920263-3A BRPI0920263B1 (pt) 2008-10-15 2009-10-15 Compostos compreendendo um oligonucleotídeo modificado, composição e uso dos mesmos

Country Status (23)

Country Link
US (7) US8334372B2 (pt)
EP (2) EP2379084B1 (pt)
JP (4) JP5809058B2 (pt)
KR (2) KR101979134B1 (pt)
CN (4) CN109797150A (pt)
AU (2) AU2009305636A1 (pt)
BR (1) BRPI0920263B1 (pt)
CA (2) CA2966011C (pt)
CY (1) CY1119909T1 (pt)
DK (1) DK2379084T3 (pt)
ES (1) ES2657679T3 (pt)
HR (1) HRP20171969T1 (pt)
HU (1) HUE035888T2 (pt)
IL (2) IL212267A (pt)
LT (1) LT2379084T (pt)
MX (1) MX2011004097A (pt)
NO (1) NO2379084T3 (pt)
NZ (2) NZ592203A (pt)
PL (1) PL2379084T3 (pt)
PT (1) PT2379084T (pt)
RU (2) RU2535964C2 (pt)
SI (1) SI2379084T1 (pt)
WO (1) WO2010045509A2 (pt)

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SI2379084T1 (en) 2008-10-15 2018-04-30 Ionis Pharmaceuticals, Inc. Modulation of factor expression 11
CA2758927A1 (en) * 2009-04-15 2010-10-21 Isis Pharmaceuticals, Inc. Modulation of inflammatory responses by factor xi
WO2012064758A2 (en) * 2010-11-08 2012-05-18 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
US20120295961A1 (en) * 2011-04-21 2012-11-22 Swayze Eric E Modulation of hepatitis b virus (hbv) expression
KR102247463B1 (ko) 2011-04-21 2021-05-04 아이오니스 파마수티컬즈, 인코포레이티드 B형 간염 바이러스(hbv) 발현 조절
WO2012170947A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
WO2013070771A1 (en) * 2011-11-07 2013-05-16 Isis Pharmaceuticals, Inc. Administration of factor xi antisense oligonucleotides
EP3971571A3 (en) 2012-05-25 2022-06-08 The University of Vermont and State Agriculture College Compositions and methods for assaying platelet reactivity and treatment selection
RU2019110030A (ru) 2013-05-01 2019-05-06 Ионис Фармасьютикалз, Инк. Композиции и способы
WO2015168618A2 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Compositions and methods for modulating growth hormone receptor expression
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
WO2016020880A2 (en) 2014-08-07 2016-02-11 Novartis Ag Angiopoietin-like 4 antibodies and methods of use
US9988443B2 (en) 2014-08-07 2018-06-05 Novartis Ag Angiopoetin-like 4 (ANGPTL4) antibodies and methods of use
JOP20200312A1 (ar) 2015-06-26 2017-06-16 Novartis Ag الأجسام المضادة للعامل xi وطرق الاستخدام
TW201802121A (zh) 2016-05-25 2018-01-16 諾華公司 抗因子XI/XIa抗體之逆轉結合劑及其用途
KR20190065341A (ko) 2016-10-06 2019-06-11 아이오니스 파마수티컬즈, 인코포레이티드 올리고머 화합물들의 접합 방법
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
US11168147B2 (en) 2016-12-23 2021-11-09 Novartis Ag Factor XI antibodies and methods of use
JP7365052B2 (ja) 2017-12-01 2023-10-19 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
EP3719128A4 (en) 2017-12-01 2021-10-27 Suzhou Ribo Life Science Co., Ltd. DOUBLE STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE WITH DOUBLE STRANDED OLIGONUCLEOTIDE, METHOD OF MANUFACTURING THEREOF AND USE THEREOF
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
KR20210005100A (ko) * 2018-04-20 2021-01-13 더 유니버시티 오브 버몬트 앤드 스테이트 애그리컬쳐 칼리지 선택된 환자에서 심혈관 질환을 치료하기 위한 조성물 및 방법
US20210355497A1 (en) * 2018-05-09 2021-11-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fxi expression
JP2021533800A (ja) 2018-08-21 2021-12-09 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Suzhou Ribo Life Science Co., Ltd. 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用
WO2020063198A1 (zh) 2018-09-30 2020-04-02 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
EP3974532A4 (en) * 2019-05-22 2024-01-24 Suzhou Ribo Life Science Co Ltd NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PROCESS OF PREPARATION AND USE
WO2022028457A1 (zh) * 2020-08-04 2022-02-10 上海拓界生物医药科技有限公司 抑制凝血因子XI表达的siRNA、组合物及其医药用途
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TW202333751A (zh) * 2021-12-16 2023-09-01 大陸商上海拓界生物醫藥科技有限公司 一種dsrna、其製備方法及應用
TW202334425A (zh) * 2021-12-16 2023-09-01 大陸商上海拓界生物醫藥科技有限公司 一種dsrna、其製備方法及應用

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CN102245186B (zh) 2014-08-27
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US20200000839A1 (en) 2020-01-02
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SI2379084T1 (en) 2018-04-30
LT2379084T (lt) 2018-03-26
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IL212267A0 (en) 2011-06-30
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