JP2010505741A - カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ - Google Patents

カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ Download PDF

Info

Publication number
JP2010505741A
JP2010505741A JP2009509880A JP2009509880A JP2010505741A JP 2010505741 A JP2010505741 A JP 2010505741A JP 2009509880 A JP2009509880 A JP 2009509880A JP 2009509880 A JP2009509880 A JP 2009509880A JP 2010505741 A JP2010505741 A JP 2010505741A
Authority
JP
Japan
Prior art keywords
oligomer
cationic
seq
bond
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.)
Pending
Application number
JP2009509880A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010505741A5 (https=
Inventor
ドワイト ディー. ウェラー,
ジェド エヌ. ハッシンガー,
Original Assignee
エイブイアイ バイオファーマ, インコーポレイテッド
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
Priority claimed from US11/432,031 external-priority patent/US8084433B2/en
Priority claimed from US11/432,216 external-priority patent/US7625873B2/en
Priority claimed from US11/432,155 external-priority patent/US20070037763A1/en
Priority claimed from US11/431,968 external-priority patent/US8785407B2/en
Priority claimed from US11/433,840 external-priority patent/US7507196B2/en
Priority claimed from US11/433,213 external-priority patent/US20070004661A1/en
Priority claimed from US11/433,033 external-priority patent/US8415313B2/en
Priority claimed from US11/433,257 external-priority patent/US20070037764A1/en
Priority claimed from US11/433,724 external-priority patent/US7888012B2/en
Priority claimed from US11/433,214 external-priority patent/US8067569B2/en
Priority claimed from US11/517,757 external-priority patent/US8524676B2/en
Priority claimed from US11/518,058 external-priority patent/US8329668B2/en
Priority claimed from US11/595,161 external-priority patent/US20070111962A1/en
Priority claimed from US11/715,572 external-priority patent/US7582615B2/en
Application filed by エイブイアイ バイオファーマ, インコーポレイテッド filed Critical エイブイアイ バイオファーマ, インコーポレイテッド
Publication of JP2010505741A publication Critical patent/JP2010505741A/ja
Publication of JP2010505741A5 publication Critical patent/JP2010505741A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1131Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1131Non-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
    • C12N15/1132Non-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 against retroviridae, e.g. HIV
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N15/1135Non-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 oncogenes or tumor suppressor genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • 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
    • 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
    • C12N15/1138Non-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 receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/314Phosphoramidates
    • C12N2310/3145Phosphoramidates with the nitrogen in 3' or 5'-position
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • AIDS & HIV (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
JP2009509880A 2006-05-10 2007-05-10 カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ Pending JP2010505741A (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
US11/431,968 US8785407B2 (en) 2006-05-10 2006-05-10 Antisense antiviral agent and method for treating ssRNA viral infection
US11/432,031 US8084433B2 (en) 2004-09-16 2006-05-10 Antisense antiviral compound and method for treating ssRNA viral infection
US11/432,155 US20070037763A1 (en) 2003-12-24 2006-05-10 Oligonucleotide compound and method for treating nidovirus infections
US11/432,216 US7625873B2 (en) 2004-07-02 2006-05-10 Antisense antibacterial method and compound
US79997606P 2006-05-11 2006-05-11
US80007606P 2006-05-11 2006-05-11
US80014506P 2006-05-11 2006-05-11
US80012006P 2006-05-11 2006-05-11
US11/433,257 US20070037764A1 (en) 2003-10-23 2006-05-11 Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US11/433,840 US7507196B2 (en) 2004-11-01 2006-05-11 Antisense antiviral compounds and methods for treating a filovirus infection
US11/433,214 US8067569B2 (en) 2000-05-04 2006-05-11 Splice-region antisense composition and method
US11/433,213 US20070004661A1 (en) 2004-10-26 2006-05-11 Antisense antiviral compound and method for treating influenza viral infection
US11/433,724 US7888012B2 (en) 2005-02-09 2006-05-11 Antisense composition and method for treating muscle atrophy
US11/433,033 US8415313B2 (en) 2004-01-23 2006-05-11 Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US11/518,058 US8329668B2 (en) 2005-09-08 2006-09-08 Antisense antiviral compound and method for treating picornavirus infection
US11/517,757 US8524676B2 (en) 2005-09-08 2006-09-08 Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US11/595,161 US20070111962A1 (en) 2005-11-08 2006-11-08 Immunosuppression compound and treatment method
US11/715,572 US7582615B2 (en) 2006-03-07 2007-03-07 Antisense antiviral compound and method for treating arenavirus infection
PCT/US2007/011435 WO2008036127A2 (en) 2006-05-10 2007-05-10 Oligonucleotide analogs having cationic intersubunit linkages

Publications (2)

Publication Number Publication Date
JP2010505741A true JP2010505741A (ja) 2010-02-25
JP2010505741A5 JP2010505741A5 (https=) 2010-07-01

Family

ID=40134113

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2009509880A Pending JP2010505741A (ja) 2006-05-10 2007-05-10 カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ

Country Status (14)

Country Link
US (1) US7943762B2 (https=)
EP (2) EP2024499B1 (https=)
JP (1) JP2010505741A (https=)
AU (1) AU2007297861A1 (https=)
CA (1) CA2651881A1 (https=)
CY (2) CY1120409T1 (https=)
DK (2) DK2735568T3 (https=)
ES (2) ES2645410T3 (https=)
HU (2) HUE035799T2 (https=)
LT (2) LT2735568T (https=)
PL (1) PL2735568T3 (https=)
PT (1) PT2735568T (https=)
SI (2) SI2735568T1 (https=)
WO (1) WO2008036127A2 (https=)

Cited By (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011503184A (ja) * 2007-11-15 2011-01-27 エイブイアイ バイオファーマ, インコーポレイテッド モルホリノオリゴマーの合成方法
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
JP2015500204A (ja) * 2011-11-18 2015-01-05 サレプタ セラピューティクス, インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
JP2015504650A (ja) * 2011-11-30 2015-02-16 サレプタ セラピューティクス, インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
JP2015506669A (ja) * 2011-11-30 2015-03-05 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
WO2016060135A1 (ja) * 2014-10-14 2016-04-21 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
JP2018518148A (ja) * 2015-03-18 2018-07-12 サレプタ セラピューティクス,インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
WO2019216433A1 (ja) * 2018-05-10 2019-11-14 日本新薬株式会社 オリゴ核酸化合物の製造方法
JP2020530981A (ja) * 2017-06-27 2020-11-05 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ T細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US10947536B2 (en) 2011-10-04 2021-03-16 Royal Holloway And Bedford New College Oligomers
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス, インコーポレイテッド アンチセンス送達用キメラペプチド

Families Citing this family (110)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US20050171044A1 (en) * 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
CA2553104A1 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
EP2933332A1 (en) 2004-06-28 2015-10-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP2351839A3 (en) 2004-07-02 2011-10-05 AVI BioPharma, Inc. Antisense antibacterial method and compound
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US8524676B2 (en) * 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
WO2009005793A2 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
AU2015200600B2 (en) * 2007-06-29 2017-04-20 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP2181983A4 (en) * 2007-07-25 2013-01-02 Ajinomoto Kk METHOD FOR THE SELECTIVE REMOVAL OF A DIBENZOFULVENE DERIVATIVE
US8076476B2 (en) * 2007-11-15 2011-12-13 Avi Biopharma, Inc. Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits
US8299206B2 (en) 2007-11-15 2012-10-30 Avi Biopharma, Inc. Method of synthesis of morpholino oligomers
EP2235034B1 (en) * 2007-12-28 2016-11-23 Sarepta Therapeutics, Inc. Immunomodulatory agents and methods of use
BR122020021379B1 (pt) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
EP2376633A1 (en) * 2008-12-17 2011-10-19 AVI BioPharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9914745B2 (en) * 2009-08-14 2018-03-13 Indian Association For The Cultivation Of Science Morpholino-based antisense agent
EP2473624B1 (en) * 2009-08-31 2019-05-01 Gen-Probe Incorporated Dengue virus assay
TR201816523T4 (tr) 2009-11-12 2018-11-21 Univ Western Australia Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
EP2499248B1 (en) 2009-11-13 2017-01-04 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
EP2542678B1 (en) 2010-03-04 2017-04-12 InteRNA Technologies B.V. A MiRNA MOLECULE DEFINED BY ITS SOURCE AND ITS THERAPEUTIC USES IN CANCER ASSOCIATED WITH EMT
US9068185B2 (en) 2010-03-12 2015-06-30 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
CA3090304A1 (en) * 2010-05-13 2011-11-17 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
AU2013202731B2 (en) * 2010-05-28 2016-04-21 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
EP2591106A1 (en) 2010-07-06 2013-05-15 InteRNA Technologies B.V. Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
ES2607603T3 (es) * 2010-09-30 2017-04-03 Nippon Shinyaku Co., Ltd. Derivado de ácido morfolino nucleico
EP2474617A1 (en) 2011-01-11 2012-07-11 InteRNA Technologies BV Mir for treating neo-angiogenesis
WO2012100156A1 (en) 2011-01-20 2012-07-26 Hercules Incorporated Enhanced dry strength and drainage performance by combining glyoxalated acrylamide-containing polymers with cationic aqueous dispersion polymers
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CA3092114A1 (en) 2011-05-05 2012-11-08 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US10100305B2 (en) 2011-07-15 2018-10-16 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation of start sites
ES2935606T3 (es) 2011-12-08 2023-03-08 Sarepta Therapeutics Inc Análogos de oligonucleótidos dirigidos a LMNA humana
EP3369818B1 (en) 2011-12-22 2021-06-09 InteRNA Technologies B.V. Mirna for treating head and neck cancer
EP2900821B1 (en) 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
EP2917348A1 (en) 2012-11-06 2015-09-16 InteRNA Technologies B.V. Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated b-raf pathway
HK1216902A1 (zh) 2012-12-20 2016-12-09 Sarepta Therapeutics, Inc. 用作治疗肌肉萎缩的改进外显子跳跃组合物
CN103060325B (zh) * 2013-01-14 2015-01-07 韩健宝 抗猪流行性腹泻病毒pedv的反义核酸及肽核酸pna
CN110218727A (zh) 2013-03-14 2019-09-10 萨勒普塔医疗公司 用于治疗肌营养不良的外显子跳跃组合物
US20140315977A1 (en) 2013-03-14 2014-10-23 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US20140329762A1 (en) 2013-03-15 2014-11-06 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US11236338B2 (en) 2013-09-05 2022-02-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
EP3044315B1 (en) 2013-09-11 2019-02-20 Synthena AG Nucleic acids and methods for the treatment of pompe disease
EP3795687A1 (en) 2014-05-23 2021-03-24 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
MA50829A (fr) 2015-06-01 2018-04-11 Sarepta Therapeutics Inc Exclusion d'exon induite pat technologie antisens dans le collagène de type vii
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
WO2017059131A1 (en) 2015-09-30 2017-04-06 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
JP2019513371A (ja) 2016-04-01 2019-05-30 アビディティー バイオサイエンシーズ エルエルシー 核酸ポリペプチド組成物とその使用
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
MX2018013157A (es) 2016-04-29 2019-05-23 Sarepta Therapeutics Inc Analogos de oligonucleotidos dirigidos a lmna humana.
BR112018074346B1 (pt) * 2016-05-24 2023-01-03 Sarepta Therapeutics, Inc. Composto oligomérico e processo para preparar um composto oligomérico
AU2017330276B2 (en) 2016-09-20 2022-01-20 The Regents Of The University Of Colorado, A Body Corporate Synthesis of backbone modified morpholino oligonucleotides and chimeras using phosphoramidite chemistry
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
MX2019008199A (es) 2017-01-06 2019-11-25 Avidity Biosciences Llc Composiciones de acido nucleico polipeptido y metodos de induccion de la omision de exon.
EP4707403A2 (en) 2017-03-10 2026-03-11 UCL Business Ltd Method relating to myostatin pathway inhibition
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
WO2019060862A1 (en) 2017-09-25 2019-03-28 Sarepta Therapeutics, Inc. METHODS FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS VIA FAST FLOW SYNTHESIS
EP3691657A4 (en) 2017-10-04 2021-07-21 Avidity Biosciences, Inc. NUCLEIC ACID POLYPEPTIDE COMPOSITIONS AND USES THEREOF
WO2019079386A1 (en) * 2017-10-17 2019-04-25 Sarepta Therapeutics, Inc. CELL PENETRATION PEPTIDES FOR ANTISENSE ADMINISTRATION
US12502432B2 (en) 2017-10-17 2025-12-23 Sarepta Therapeutics, Inc. Bicyclic peptide oligonucleotide conjugates
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
CN111566212A (zh) 2017-11-03 2020-08-21 因特尔纳技术有限公司 miRNA分子,等同物,安塔够妙或其来源用于治疗和/或诊断与神经元缺陷相关的病症和/或疾病或用于神经元生成和/或再生
AU2018378812B2 (en) 2017-12-06 2025-05-22 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
JP2021513508A (ja) 2018-02-12 2021-05-27 インテアールエヌエー テクノロジーズ ビー.ヴイ.InteRNA Technologies B.V. 抗がんマイクロrna及びその脂質製剤
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
EP3918069A1 (en) 2019-01-31 2021-12-08 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
US12006499B2 (en) 2019-06-06 2024-06-11 Avidity Biosciences, Inc. Una amidites and uses thereof
CN114375296A (zh) 2019-06-06 2022-04-19 艾维迪提生物科学公司 核酸多肽组合物及其用途
JP2022536495A (ja) 2019-06-19 2022-08-17 サレプタ セラピューティクス, インコーポレイテッド 筋ジストロフィーを治療する方法
CN114901823A (zh) 2019-12-26 2022-08-12 日本新药株式会社 诱导外显子50的跳读的反义核酸
IL295967A (en) 2020-02-28 2022-10-01 Nippon Shinyaku Co Ltd Antisense nucleic acids for splicing on exon 51
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
SI4126066T1 (sl) 2020-03-27 2026-04-30 Avidity Biosciences, Inc. Sestavki in postopki zdravljenja mišične distrofije
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
CN111575409B (zh) * 2020-05-23 2023-04-18 黑龙江八一农垦大学 基于整合素配体识别肽抑制猪流行性腹泻病毒感染的研究方法
EP4247949A1 (en) 2020-11-23 2023-09-27 Alpha Anomeric SAS Nucleic acid duplexes
EP4332223A4 (en) * 2021-04-28 2025-12-31 Nippon Shinyaku Co Ltd ANTIVIRAL NUCLEIC ACID
US12378267B2 (en) 2021-06-17 2025-08-05 Entrada Therapeutics, Inc. Synthesis of FMOC-protected morpholino monomers and oligomers
WO2022270585A1 (ja) 2021-06-23 2022-12-29 日本新薬株式会社 アンチセンスオリゴマーの組み合わせ
TW202308664A (zh) 2021-07-08 2023-03-01 日商日本新藥股份有限公司 腎毒性減輕劑
CN118475355A (zh) 2021-07-08 2024-08-09 日本新药株式会社 肾毒性减轻剂
CA3225573A1 (en) 2021-07-08 2023-01-12 Nippon Shinyaku Co., Ltd. Precipitation suppressing agent
US20250011777A1 (en) 2021-09-03 2025-01-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
WO2023055774A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
EP4419680A1 (en) 2021-10-22 2024-08-28 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
CN119156395A (zh) 2022-04-22 2024-12-17 恩特拉达治疗学股份有限公司 用于递送治疗剂的环肽
JP2025533454A (ja) 2022-09-21 2025-10-07 サレプタ セラピューティクス, インコーポレイテッド Dmdアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
KR20250174651A (ko) 2023-04-06 2025-12-12 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. 5'-포스포네이트 변형 뉴클레오시드 유사체 및 이로부터 제조된 올리고뉴클레오티드
TW202442246A (zh) 2023-04-27 2024-11-01 美商薩羅塔治療公司 用於治療慢性腎病之反義寡聚物
TW202519660A (zh) 2023-08-02 2025-05-16 美商薩羅塔治療公司 針對反義寡聚物遞送之非標準細胞穿透肽
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
CN120058705A (zh) * 2025-04-29 2025-05-30 苏州诺维康生物科技有限公司 一种医药中间体PMO-G(N2-iBu)的合成方法

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5185444A (en) * 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JPH05504563A (ja) * 1989-12-20 1993-07-15 アンチビラルス・インコーポレイテツド リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー
WO2002092617A1 (en) * 2001-05-17 2002-11-21 Avi Biopharma, Inc. Combined approach to treatment of cancer using a c-myc antisense oligomer
WO2005115479A2 (en) * 2004-05-24 2005-12-08 Avi Biopharma, Inc. Peptide conjugated, inosine-substituted antisense oligomer compound and method
WO2006033933A2 (en) * 2004-09-16 2006-03-30 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssrna viral infection
WO2006047683A2 (en) * 2004-10-26 2006-05-04 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1986005519A1 (en) 1985-03-15 1986-09-25 James Summerton Polynucleotide assay reagent and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5852188A (en) 1990-01-11 1998-12-22 Isis Pharmaceuticals, Inc. Oligonucleotides having chiral phosphorus linkages
HUT69956A (en) 1990-08-14 1995-09-28 Isis Pharmaceuticals Inc Methode for inhibition of influenza virus by antiseuse oligonucleotides
AU659482B2 (en) 1991-06-28 1995-05-18 Massachusetts Institute Of Technology Localized oligonucleotide therapy
US5576302A (en) 1991-10-15 1996-11-19 Isis Pharmaceuticals, Inc. Oligonucleotides for modulating hepatitis C virus having phosphorothioate linkages of high chiral purity
JPH07502658A (ja) 1991-12-23 1995-03-23 バイエル コーポレイション 溶液相サンドイッチハイブリダイゼーションアッセイに用いるためのhavプローブ
US6174868B1 (en) 1992-09-10 2001-01-16 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis C virus-associated diseases
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US6060456A (en) 1993-11-16 2000-05-09 Genta Incorporated Chimeric oligonucleoside compounds
US5734039A (en) 1994-09-15 1998-03-31 Thomas Jefferson University Antisense oligonucleotides targeting cooperating oncogenes
GB9510718D0 (en) 1995-05-26 1995-07-19 Sod Conseils Rech Applic Antisense oligonucleotides
US5955318A (en) 1995-08-14 1999-09-21 Abbott Laboratories Reagents and methods useful for controlling the translation of hepatitis GBV proteins
US6686338B1 (en) 1996-02-23 2004-02-03 The Board Of Regents Of The University Of Nebraska Enzyme inhibitors for metabolic redirection
US6245747B1 (en) 1996-03-12 2001-06-12 The Board Of Regents Of The University Of Nebraska Targeted site specific antisense oligodeoxynucleotide delivery method
CN1263473A (zh) 1997-05-21 2000-08-16 利兰·斯坦福青年大学托管委员会 增加跨生物膜转运的组合物和方法
AUPP398498A0 (en) 1998-06-09 1998-07-02 Silverbrook Research Pty Ltd A method of manufacture of an image creation apparatus (ijm44)
US6133246A (en) 1997-08-13 2000-10-17 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the modulation of JNK proteins
US6228579B1 (en) 1997-11-14 2001-05-08 San Diego State University Foundation Method for identifying microbial proliferation genes
CA2337361C (en) 1998-07-13 2010-11-16 The Board Of Regents Of The University Of Nebraska Targeted site specific drug delivery compositions and method of use
US7094765B1 (en) 1999-01-29 2006-08-22 Avi Biopharma, Inc. Antisense restenosis composition and method
KR20010102992A (ko) 1999-01-29 2001-11-17 추후보정 표적 rna를 검출하는 비-침입적 방법
US20030095953A1 (en) 1999-11-12 2003-05-22 Myles C. Cabot Methods of reversing drug resistance in cancer cells
JP2003516151A (ja) 1999-11-29 2003-05-13 エイブイアイ バイオファーマ, インコーポレイテッド 細菌16Sおよび23SrRNAに標的化された、荷電していないアンチセンスオリゴヌクレオチド、ならびにその使用
AU781676B2 (en) 2000-01-04 2005-06-02 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
JP2004523465A (ja) 2000-05-17 2004-08-05 エイブイアイ バイオファーマ, インコーポレイテッド 薬物の薬物動態を改善するためのアンチセンス酵素インヒビター
EP1301525B1 (en) 2000-07-06 2015-09-02 Sarepta Therapeutics, Inc. Transforming growth factor beta (tgf-beta) blocking agent-treated stem cell composition and method
JP2005507660A (ja) 2001-10-16 2005-03-24 アビ バイオファーマ, インコーポレイテッド ssRNAウイルス感染の処置のためのアンチセンス抗ウイルス剤および方法
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
US20030207907A1 (en) 2002-05-03 2003-11-06 Iversen Patrick L. Delivery of microparticle-conjugated drugs for inhibition of stenosis
BR0314236A (pt) 2002-09-13 2005-08-09 Replicor Inc Formulação de oligonucleotìdeo, composição farmacêutica, kit, composto antiviral, preparação de oligonucleotìdeo e métodos para seleção de um oligonucleotìdeo antiviral para uso como um agente antiviral, para profilaxia ou tratamento de uma infecção viral em um paciente, para tratamento profilático de câncer causado por oncovìrus, para identificação de um composto que altera a ligação de um oligonucleotìdeo a pelo menos um componente viral, para purificação da ligação de oligonucleotìdeos a pelo menos um componente viral e para enriquecimento de oligonucleotìdeos a partir de um agrupamento de oligonucleotìdeos
US7115374B2 (en) 2002-10-16 2006-10-03 Gen-Probe Incorporated Compositions and methods for detecting West Nile virus
JP4791043B2 (ja) 2002-12-31 2011-10-12 プロリゴ・エルエルシー 同一固体支持体上で、2以上のオリゴヌクレオチドをタンデムに合成するための方法および組成物
CA2533218C (en) 2003-08-05 2014-11-18 Avi Biopharma, Inc. Oligonucleotide analog and method for treating flavivirus infections
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
CA2553104A1 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
EP2351839A3 (en) 2004-07-02 2011-10-05 AVI BioPharma, Inc. Antisense antibacterial method and compound
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
TW200622777A (en) * 2004-12-31 2006-07-01 Inventec Corp Manufacturing-order giving system and method thereof
US8524676B2 (en) 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
WO2007030576A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5185444A (en) * 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
JPH05504563A (ja) * 1989-12-20 1993-07-15 アンチビラルス・インコーポレイテツド リン含有キラルインターサブユニットリンケージを有する非電荷モルホリノ―基体ポリマー
WO2002092617A1 (en) * 2001-05-17 2002-11-21 Avi Biopharma, Inc. Combined approach to treatment of cancer using a c-myc antisense oligomer
JP2004537517A (ja) * 2001-05-17 2004-12-16 エイブイアイ バイオファーマ, インコーポレイテッド c−mycアンチセンスオリゴマーを使用した、癌を処置するための併用アプローチ
WO2005115479A2 (en) * 2004-05-24 2005-12-08 Avi Biopharma, Inc. Peptide conjugated, inosine-substituted antisense oligomer compound and method
JP2008509701A (ja) * 2004-05-24 2008-04-03 アビ バイオファーマ, インコーポレイテッド ペプチドが結合された、イノシン置換アンチセンスオリゴマー化合物および方法
WO2006033933A2 (en) * 2004-09-16 2006-03-30 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssrna viral infection
JP2008513012A (ja) * 2004-09-16 2008-05-01 アヴィ バイオファーマ、インコーポレイテッド アンチセンス抗ウイルス化合物及びssRNAウイルス感染を治療する方法
WO2006047683A2 (en) * 2004-10-26 2006-05-04 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection

Cited By (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011503184A (ja) * 2007-11-15 2011-01-27 エイブイアイ バイオファーマ, インコーポレイテッド モルホリノオリゴマーの合成方法
KR101829644B1 (ko) 2010-05-28 2018-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP2013530154A (ja) * 2010-05-28 2013-07-25 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
US10760078B2 (en) 2010-05-28 2020-09-01 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP2020018299A (ja) * 2010-05-28 2020-02-06 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
JP2021048886A (ja) * 2010-05-28 2021-04-01 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9469664B2 (en) 2010-05-28 2016-10-18 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP7008056B2 (ja) 2010-05-28 2022-01-25 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
US10202602B2 (en) 2010-05-28 2019-02-12 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
KR20180019751A (ko) * 2010-05-28 2018-02-26 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP2018002728A (ja) * 2010-05-28 2018-01-11 サレプタ セラピューティクス, インコーポレイテッド 修飾されたサブユニット間結合および/または末端基を有するオリゴヌクレオチドアナログ
US11072793B2 (en) 2010-09-03 2021-07-27 Sarepta Therapeutics, Inc. DsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10017763B2 (en) 2010-09-03 2018-07-10 Sarepta Therapeutics, Inc. dsRNA molecules comprising oligonucleotide analogs having modified intersubunit linkages and/or terminal groups
US10947536B2 (en) 2011-10-04 2021-03-16 Royal Holloway And Bedford New College Oligomers
US9278987B2 (en) 2011-11-18 2016-03-08 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
JP2018048208A (ja) * 2011-11-18 2018-03-29 サレプタ セラピューティクス, インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
US11208655B2 (en) 2011-11-18 2021-12-28 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US10344281B2 (en) 2011-11-18 2019-07-09 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
US9790499B2 (en) 2011-11-18 2017-10-17 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
JP2015500204A (ja) * 2011-11-18 2015-01-05 サレプタ セラピューティクス, インコーポレイテッド 機能的に修飾されたオリゴヌクレオチドおよびそのサブユニット
JP2018082717A (ja) * 2011-11-30 2018-05-31 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP7057385B2 (ja) 2011-11-30 2022-04-19 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP2015506669A (ja) * 2011-11-30 2015-03-05 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
JP2015504650A (ja) * 2011-11-30 2015-02-16 サレプタ セラピューティクス, インコーポレイテッド 脊髄性筋萎縮症における誘発されたエクソン包含
JP2020078347A (ja) * 2011-11-30 2020-05-28 サレプタ セラピューティクス, インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
US10472624B2 (en) 2014-10-14 2019-11-12 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
CN106795186B (zh) * 2014-10-14 2021-03-02 味之素株式会社 吗啉代寡核苷酸的制备方法
US11028386B2 (en) 2014-10-14 2021-06-08 Ajinomoto Co., Inc. Morpholino oligonucleotide manufacturing method
JPWO2016060135A1 (ja) * 2014-10-14 2017-07-27 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
CN106795186A (zh) * 2014-10-14 2017-05-31 味之素株式会社 吗啉代寡核苷酸的制备方法
WO2016060135A1 (ja) * 2014-10-14 2016-04-21 味の素株式会社 モルフォリノオリゴヌクレオチドの製造方法
JP2021090458A (ja) * 2015-03-18 2021-06-17 サレプタ セラピューティクス,インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
JP2018518148A (ja) * 2015-03-18 2018-07-12 サレプタ セラピューティクス,インコーポレイテッド ミオスタチンにおけるアンチセンスに誘導されるエクソンの排除
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
US12121532B2 (en) 2015-08-28 2024-10-22 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
US10905709B2 (en) 2015-08-28 2021-02-02 Sarepta Therapeutics, Inc. Modified antisense oligomers for exon inclusion in spinal muscular atrophy
JP7284718B2 (ja) 2017-06-27 2023-05-31 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ T細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
JP2020530981A (ja) * 2017-06-27 2020-11-05 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ T細胞の機能を調節するためのアンチセンスオリゴヌクレオチド
JP2021518361A (ja) * 2018-03-16 2021-08-02 サレプタ セラピューティクス, インコーポレイテッド アンチセンス送達用キメラペプチド
JP2024163336A (ja) * 2018-03-16 2024-11-21 サレプタ セラピューティクス, インコーポレイテッド アンチセンス送達用キメラペプチド
JP7596148B2 (ja) 2018-03-16 2024-12-09 サレプタ セラピューティクス, インコーポレイテッド アンチセンス送達用キメラペプチド
US11560401B2 (en) 2018-05-10 2023-01-24 Nippon Shinyaku Co., Ltd. Method for preparing oligonucleic acid compound
WO2019216433A1 (ja) * 2018-05-10 2019-11-14 日本新薬株式会社 オリゴ核酸化合物の製造方法

Also Published As

Publication number Publication date
DK2024499T3 (da) 2018-01-29
WO2008036127A2 (en) 2008-03-27
HK1198388A1 (en) 2015-04-17
CY1120409T1 (el) 2019-07-10
ES2645410T3 (es) 2017-12-05
EP2024499A2 (en) 2009-02-18
PL2735568T3 (pl) 2018-02-28
CA2651881A1 (en) 2008-03-27
US7943762B2 (en) 2011-05-17
AU2007297861A1 (en) 2008-03-27
WO2008036127A3 (en) 2008-11-20
DK2735568T3 (da) 2017-11-13
ES2657400T3 (es) 2018-03-05
EP2735568A1 (en) 2014-05-28
CY1120486T1 (el) 2019-07-10
EP2735568B1 (en) 2017-08-02
EP2735568B8 (en) 2017-09-27
LT2024499T (lt) 2018-02-26
SI2735568T1 (en) 2018-01-31
HUE035799T2 (en) 2018-05-28
HUE036995T2 (hu) 2018-08-28
SI2024499T1 (en) 2018-04-30
LT2735568T (lt) 2017-11-27
PT2735568T (pt) 2017-11-14
EP2024499B1 (en) 2017-10-25
US20090088562A1 (en) 2009-04-02

Similar Documents

Publication Publication Date Title
JP2010505741A (ja) カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
US11732259B2 (en) Peptide oligonucleotide conjugates
US20210139897A1 (en) Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
JP2019534862A (ja) ホスホルアミダイト化学を使用する骨格修飾モルホリノオリゴヌクレオチド及びキメラの合成
KR20200024793A (ko) 알파-ENaC의 발현을 억제하기 위한 RNAi 작용제 및 사용 방법
CN114846142A (zh) 用于抑制β-ENaC表达的RNAi试剂、其组合物和使用方法
JP2025535376A (ja) DM1タンパク質キナーゼ(DMPK)の発現を阻害するためのRNAi剤、その組成物および使用方法
TW202334416A (zh) 用於抑制基質金屬蛋白酶7 (MMP7)之表現的RNAi藥劑、其組成物及使用方法
WO2021122869A1 (en) Use of scamp3 inhibitors for treating hepatitis b virus infection
EP4473112A2 (en) Rnai agents for inhibiting expression of coronavirus (cov) viral genomes, compositions thereof, and methods of use
CA3218815A1 (en) Rnai agents for inhibiting expression of mucin 5ac (muc5ac), compositions thereof, and methods of use
WO2006121951A2 (en) Antisense antiviral compounds and methods for treating foot and mouth disease
AU2013202322B2 (en) Oligonucleotide analogs having cationic intersubunit linkages
TW202325846A (zh) 用於治療周邊髓鞘蛋白22相關疾病的嗎啉代寡聚物
WO2021122921A1 (en) Use of cops3 inhibitors for treating hepatitis b virus infection
HK1198388B (en) Oligonucleotide analogs having cationic intersubunit linkages
KR20260034076A (ko) 다량체성 RNAi 작용제 접합체를 위한 간 전달 플랫폼 및 그의 사용 방법
JP2026502546A (ja) インフルエンザA型ウイルス遺伝子発現を阻害するためのRNAi剤、その組成物、及び使用方法
CN121127589A (zh) 用于抑制共济失调蛋白-2 (ATXN2)表达的RNAi试剂、其组合物及使用方法
OA21427A (en) RNAI agents for inhibiting expression of mucin 5AC (MUC5AC), compositions thereof, and methods of use.
KR20260030178A (ko) 올리고뉴클레오티드 접합체를 위한 대사적으로 안정화된 탄수화물 표적화 리간드
HK40100193A (zh) Lpa抑制剂及其用途
HK40100193B (zh) Lpa抑制剂及其用途

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100510

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20100510

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20121120

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20121120

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130218

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130225

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20130712