CN107973833A - 作为治疗剂的寡核苷酸类似物的设计 - Google Patents

作为治疗剂的寡核苷酸类似物的设计 Download PDF

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Publication number
CN107973833A
CN107973833A CN201711195027.0A CN201711195027A CN107973833A CN 107973833 A CN107973833 A CN 107973833A CN 201711195027 A CN201711195027 A CN 201711195027A CN 107973833 A CN107973833 A CN 107973833A
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China
Prior art keywords
alkyl
aryl
substituted
compound
independently
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CN201711195027.0A
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English (en)
Chinese (zh)
Inventor
R·P·伊耶
J·E·库格林
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F Star Therapeutics Inc
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Spring Bank Pharmaceuticals Inc
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Publication of CN107973833A publication Critical patent/CN107973833A/zh
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1131—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 viruses
    • 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
    • A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • 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
    • A61P31/04—Antibacterial agents
    • 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
    • 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
    • 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
    • A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
    • 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
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P37/04—Immunostimulants
    • 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
    • A61P37/08—Antiallergic agents
    • 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
    • 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
    • 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]
    • 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
    • C12N2310/17—Immunomodulatory nucleic acids
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pulmonology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
CN201711195027.0A 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计 Pending CN107973833A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US40238010P 2010-08-30 2010-08-30
US61/402,380 2010-08-30
CN2011800522858A CN103298475A (zh) 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN2011800522858A Division CN103298475A (zh) 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计

Publications (1)

Publication Number Publication Date
CN107973833A true CN107973833A (zh) 2018-05-01

Family

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Family Applications (2)

Application Number Title Priority Date Filing Date
CN201711195027.0A Pending CN107973833A (zh) 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计
CN2011800522858A Pending CN103298475A (zh) 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计

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CN2011800522858A Pending CN103298475A (zh) 2010-08-30 2011-08-25 作为治疗剂的寡核苷酸类似物的设计

Country Status (6)

Country Link
US (2) US9040234B2 (https=)
EP (2) EP3524252A1 (https=)
JP (3) JP2014500230A (https=)
KR (1) KR101926419B1 (https=)
CN (2) CN107973833A (https=)
WO (1) WO2012030626A2 (https=)

Families Citing this family (14)

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US9040234B2 (en) * 2010-08-30 2015-05-26 Spring Bank Pharmaceuticals, Inc. Oligonucleotide analogs as therapeutic agents
WO2013142157A1 (en) * 2012-03-22 2013-09-26 Alios Biopharma, Inc. Pharmaceutical combinations comprising a thionucleotide analog
JP6502267B2 (ja) * 2013-02-18 2019-04-17 スプリング バンク ファーマシューティカルズ，インコーポレイテッド ワクチンアジュバントおよび治療剤としての短鎖オリゴヌクレオチドの設計
ES2891903T3 (es) * 2015-02-17 2022-01-31 Cipherome Inc Método para seleccionar agente antineoplásico basado en información de daño proteínico de individuos para evitar los efectos secundarios del agente antineoplásico
WO2016164619A2 (en) * 2015-04-07 2016-10-13 Spring Bank Pharmaceuticals, Inc. Compositions and methods for the treatment of hbv infection
EP3317290A4 (en) * 2015-07-02 2019-04-10 Spring Bank Pharmaceuticals, Inc. COMPOSITIONS AND METHOD FOR TREATING VIRUS INFECTION
TW201717968A (zh) * 2015-07-14 2017-06-01 春季銀行製藥公司 誘導ｒｉｇ－ｉ和其他模式辨識受體之化合物及組成物
EP3507367A4 (en) * 2016-07-05 2020-03-25 Aduro BioTech, Inc. CYCLIC DINUCLEOTID COMPOUNDS WITH INCLUDED NUCLEIC ACIDS AND USES THEREOF
WO2018013887A1 (en) * 2016-07-15 2018-01-18 Sperovie Biosciences, Inc. Compounds, compositions, and methods for the treatment of disease
CN109715202A (zh) * 2016-07-15 2019-05-03 斯必乐维生物科学公司 用于治疗疾病的化合物、组合物和方法
CA3033867A1 (en) 2016-08-17 2018-02-22 Solstice Biologics, Ltd. Polynucleotide constructs
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
CN111484541B (zh) * 2019-01-25 2023-06-02 博瑞生物医药（苏州）股份有限公司 双核苷酸前体药物及其制备方法
US20230242918A1 (en) * 2020-05-22 2023-08-03 President And Fellows Of Harvard College Interferon- inducing oligonucleotide duplexes and methods of use

Citations (1)

* Cited by examiner, † Cited by third party
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WO2009146123A2 (en) * 2008-04-03 2009-12-03 Spring Bank Compositions and methods for treating viral infections

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WO2002092006A2 (en) * 2001-05-16 2002-11-21 Micrologix Biotech, Inc. Nucleic acid-based compounds and methods of use thereof
CA2549173A1 (en) 2003-12-08 2005-07-07 Hybridon, Inc. Modulation of immunostimulatory properties by small oligonucleotide-based compounds
JP2007526253A (ja) 2004-02-19 2007-09-13 コーリー ファーマシューティカル グループ，インコーポレイテッド 免疫刺激性ウイルスｒｎａオリゴヌクレオチド
JP2008523785A (ja) 2004-10-12 2008-07-10 カーランテック，インコーポレーテッド 神経変性、自己免疫脱髄、および糖尿病のウイルス病原に対する動物モデル系
US8076303B2 (en) * 2005-12-13 2011-12-13 Spring Bank Pharmaceuticals, Inc. Nucleotide and oligonucleotide prodrugs
ES2911034T3 (es) * 2006-08-08 2022-05-17 Univ Bonn Rheinische Friedrich Wilhelms Estructura y uso de oligonucleótidos 5' fosfato
US20100040576A1 (en) * 2007-12-18 2010-02-18 The Texas A&M University System Modified Oligonucleotides For The Treatment Of Hepatitis C Infection
WO2009141146A1 (en) * 2008-05-21 2009-11-26 Gunther Hartmann 5' triphosphate oligonucleotide with blunt end and uses thereof
US9040234B2 (en) * 2010-08-30 2015-05-26 Spring Bank Pharmaceuticals, Inc. Oligonucleotide analogs as therapeutic agents

Patent Citations (1)

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WO2009146123A2 (en) * 2008-04-03 2009-12-03 Spring Bank Compositions and methods for treating viral infections

Also Published As

Publication number Publication date
US20150329864A1 (en) 2015-11-19
EP3524252A1 (en) 2019-08-14
WO2012030626A3 (en) 2012-05-31
JP2016164148A (ja) 2016-09-08
JP2018010001A (ja) 2018-01-18
US9040234B2 (en) 2015-05-26
WO2012030626A2 (en) 2012-03-08
JP2014500230A (ja) 2014-01-09
EP2611451A2 (en) 2013-07-10
EP2611451A4 (en) 2014-01-15
US20120053226A1 (en) 2012-03-01
CN103298475A (zh) 2013-09-11
KR101926419B1 (ko) 2018-12-10
JP6210392B2 (ja) 2017-10-11
KR20140048068A (ko) 2014-04-23

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