[go: up one dir, main page]

WO2004043977A3 - 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations - Google Patents

2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations Download PDF

Info

Publication number
WO2004043977A3
WO2004043977A3 PCT/US2003/034905 US0334905W WO2004043977A3 WO 2004043977 A3 WO2004043977 A3 WO 2004043977A3 US 0334905 W US0334905 W US 0334905W WO 2004043977 A3 WO2004043977 A3 WO 2004043977A3
Authority
WO
WIPO (PCT)
Prior art keywords
oligomer
compositions
oligomeric compounds
fluoro substituted
hybridizing
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.)
Ceased
Application number
PCT/US2003/034905
Other languages
French (fr)
Other versions
WO2004043977A2 (en
Inventor
Thazha P Prakush
Brenda F Baker
Anne B Eldrup
Muthiah Manoharan
Balkrishen Bhat
Richard H Griffey
Eric E Swayze
Stanley T Crooke
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
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
Priority to AU2003287464A priority Critical patent/AU2003287464A1/en
Publication of WO2004043977A2 publication Critical patent/WO2004043977A2/en
Publication of WO2004043977A3 publication Critical patent/WO2004043977A3/en
Anticipated expiration legal-status Critical
Ceased 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Landscapes

  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)

Abstract

Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2'-F substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleoside of the oligomer has a modified sugar and/or backbone modification.
PCT/US2003/034905 2002-11-05 2003-11-04 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations Ceased WO2004043977A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003287464A AU2003287464A1 (en) 2002-11-05 2003-11-04 2'-fluoro substituted oligomeric compounds and compositions for use in gene modulations

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US42376002P 2002-11-05 2002-11-05
US60/423,760 2002-11-05
US50352103P 2003-09-16 2003-09-16
US60/503,521 2003-09-16

Publications (2)

Publication Number Publication Date
WO2004043977A2 WO2004043977A2 (en) 2004-05-27
WO2004043977A3 true WO2004043977A3 (en) 2005-01-20

Family

ID=32314503

Family Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2003/034905 Ceased WO2004043977A2 (en) 2002-11-05 2003-11-04 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/035087 Ceased WO2004044140A2 (en) 2002-11-05 2003-11-04 2’-substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/034906 Ceased WO2004043978A2 (en) 2002-11-05 2003-11-04 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations

Family Applications After (2)

Application Number Title Priority Date Filing Date
PCT/US2003/035087 Ceased WO2004044140A2 (en) 2002-11-05 2003-11-04 2’-substituted oligomeric compounds and compositions for use in gene modulations
PCT/US2003/034906 Ceased WO2004043978A2 (en) 2002-11-05 2003-11-04 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations

Country Status (4)

Country Link
EP (1) EP1563070A4 (en)
AU (4) AU2003295388A1 (en)
CA (1) CA2504554A1 (en)
WO (3) WO2004043977A2 (en)

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7176304B2 (en) 2002-02-20 2007-02-13 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7456155B2 (en) 2002-06-28 2008-11-25 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7582618B2 (en) 2002-06-28 2009-09-01 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7598373B2 (en) 2002-12-12 2009-10-06 Idenix Pharmaceuticals, Inc. Process for the production of 2-C-methyl-D-ribonolactone
US7608597B2 (en) 2000-05-23 2009-10-27 Idenix Pharmaceuticals, Inc. Methods and compositions for treating hepatitis C virus
US7858625B2 (en) 2001-05-18 2010-12-28 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery

Families Citing this family (56)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
PL359169A1 (en) 2000-05-26 2004-08-23 Idenix (Cayman) Limited Methods and compositions for treating flaviviruses and pestiviruses
WO2005078097A2 (en) 2004-02-10 2005-08-25 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING MULTIFUNCTIONAL SHORT INTERFERING NUCLEIC ACID (Multifunctional siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
GB2413557B (en) * 2002-02-20 2006-08-16 Sirna Therapeutics Inc RNA interference mediated inhibtion of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
GB2406568B (en) * 2002-02-20 2005-09-28 Sirna Therapeutics Inc RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2004044139A2 (en) 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
DK1576138T3 (en) 2002-11-15 2017-05-01 Idenix Pharmaceuticals Llc 2'-METHYL NUCLEOSIDES IN COMBINATION WITH INTERFERON AND FLAVIVIRIDAE MUTATION
US7151089B2 (en) 2003-10-27 2006-12-19 Genelabs Technologies, Inc. Nucleoside compounds for treating viral infections
US7202223B2 (en) 2003-10-27 2007-04-10 Genelabs Technologies, Inc. Nucleoside compounds for treating viral infections
AU2004295291A1 (en) 2003-10-27 2005-06-16 Smithkline Beecham Corporation Nucleoside compounds for treating viral infections
US7169918B2 (en) 2003-10-27 2007-01-30 Genelabs Technologies, Inc. Methods for preparing 7-(2′-substituted-β-D-ribofuranosyl)-4-(NR2R3)-5-(substituted ethyn-1-yl)-pyrrolo[2,3-d]pyrimidine derivatives
US7157434B2 (en) 2003-10-27 2007-01-02 Genelabs Technologies, Inc. Nucleoside compounds for treating viral infections
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
WO2006036916A2 (en) 2004-09-24 2006-04-06 Alnylam Pharmaceuticals, Inc. Rnai modulation of apob and uses thereof
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
EP2609198B8 (en) 2010-08-24 2018-03-28 Sirna Therapeutics, Inc. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
ES2663009T3 (en) 2010-10-29 2018-04-10 Sirna Therapeutics, Inc. Inhibition of RNA-mediated gene expression using short interference nucleic acids (ANic)
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
MA41795A (en) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc EXCLUSION OF AN EXON INDUCED BY ANTISENSE COMPOUNDS IN MYOSTATIN
EP3851531A1 (en) 2015-06-01 2021-07-21 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type vii collagen
CN108699555A (en) 2015-10-09 2018-10-23 萨勒普塔医疗公司 Composition for treating Duchenne's dystrophy and associated disease and method
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
SG11201809468XA (en) 2016-04-29 2018-11-29 Sarepta Therapeutics Inc Oligonucleotide analogues targeting human lmna
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MX390906B (en) 2016-05-24 2025-03-21 Sarepta Therapeutics Inc PROCESSES FOR PREPARING MORPHOLINO PHOSPHORODIAMIDATE OLIGOMERS.
MA45362A (en) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc PROCESSES FOR THE PREPARATION OF MORPHOLINO OLIGOMERS OF PHOSPHORODIAMIDATE
EP3464305B1 (en) 2016-05-24 2024-08-21 Sarepta Therapeutics, Inc. Processes for preparing oligomers
MA45155B1 (en) 2016-05-24 2024-06-28 Sarepta Therapeutics, Inc. PROCESSES FOR THE PREPARATION OF MORPHOLINO OLIGOMERS OF PHOSPHORODIAMIDATE
US20190262375A1 (en) 2016-06-30 2019-08-29 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
KR102810425B1 (en) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 Exon skipping oligomer conjugates for muscular dystrophy
EP3554552B1 (en) 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
PT3554554T (en) 2016-12-19 2022-11-08 Sarepta Therapeutics Inc EXON-SKIPPING OLIGOMER CONJUGATIONS FOR MASCULINE DYSTROPHY
EA201991450A1 (en) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. OLIGOMER CONJUGATES FOR EXONISM SKIP IN MUSCULAR DYSTROPHY
JP2020536060A (en) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド Combination therapy to treat muscular dystrophy
US20200254002A1 (en) 2017-09-28 2020-08-13 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
JP7394753B2 (en) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド antisense oligomer compounds
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
TW202449155A (en) 2018-07-27 2024-12-16 美商薩羅塔治療公司 Exon skipping oligomers for muscular dystrophy
AU2019397461A1 (en) 2018-12-13 2021-07-29 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20220193246A1 (en) 2019-04-18 2022-06-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
JP2024537711A (en) 2021-09-30 2024-10-16 サレプタ セラピューティクス， インコーポレイテッド Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
EP4590311A2 (en) 2022-09-21 2025-07-30 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
TW202442246A (en) 2023-04-27 2024-11-01 美商薩羅塔治療公司 Antisense oligomers for treatment of chronic kidney disease
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6107094A (en) * 1996-06-06 2000-08-22 Isis Pharmaceuticals, Inc. Oligoribonucleotides and ribonucleases for cleaving RNA
US20030143732A1 (en) * 2001-04-05 2003-07-31 Kathy Fosnaugh RNA interference mediated inhibition of adenosine A1 receptor (ADORA1) gene expression using short interfering RNA

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
US5760209A (en) * 1997-03-03 1998-06-02 Isis Pharmaceuticals, Inc. Protecting group for synthesizing oligonucleotide analogs
US5955443A (en) * 1998-03-19 1999-09-21 Isis Pharmaceuticals Inc. Antisense modulation of PECAM-1
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
JP2003526367A (en) * 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド RNA interference method and RNA interference composition
TR200401292T3 (en) * 2000-12-01 2004-07-21 Max@Planck@Gesellschaft�Zur�F�Rderung�Der�Wissenschaften the rnaágirişimineáyoláaçanáküçükárnaámolekül

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6107094A (en) * 1996-06-06 2000-08-22 Isis Pharmaceuticals, Inc. Oligoribonucleotides and ribonucleases for cleaving RNA
US20030143732A1 (en) * 2001-04-05 2003-07-31 Kathy Fosnaugh RNA interference mediated inhibition of adenosine A1 receptor (ADORA1) gene expression using short interfering RNA

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PARRISH ET AL.: "Functional anatomy of a dsRNA trigger: Differential requirement for the two trigger strands in RNA interference", MOLECULAR CELL, vol. 6, November 2000 (2000-11-01), pages 1077 - 1087, XP002226298 *

Cited By (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7608597B2 (en) 2000-05-23 2009-10-27 Idenix Pharmaceuticals, Inc. Methods and compositions for treating hepatitis C virus
US8299038B2 (en) 2000-05-23 2012-10-30 Idenix Pharmaceuticals, Inc. Methods and compositions for treating hepatitis C virus
US7517864B2 (en) 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7858625B2 (en) 2001-05-18 2010-12-28 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7176304B2 (en) 2002-02-20 2007-02-13 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US7456155B2 (en) 2002-06-28 2008-11-25 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7582618B2 (en) 2002-06-28 2009-09-01 Idenix Pharmaceuticals, Inc. 2′-C-methyl-3′-O-L-valine ester ribofuranosyl cytidine for treatment of flaviviridae infections
US7598373B2 (en) 2002-12-12 2009-10-06 Idenix Pharmaceuticals, Inc. Process for the production of 2-C-methyl-D-ribonolactone

Also Published As

Publication number Publication date
AU2003287464A1 (en) 2004-06-03
AU2003291682A1 (en) 2004-06-03
EP1563070A4 (en) 2008-05-28
EP1563070A2 (en) 2005-08-17
WO2004044140A2 (en) 2004-05-27
WO2004043977A2 (en) 2004-05-27
AU2003295388A1 (en) 2004-06-03
AU2003291682A8 (en) 2004-06-03
WO2004044140A3 (en) 2005-04-14
AU2003287464A8 (en) 2004-06-03
CA2504554A1 (en) 2004-05-27
AU2010201712B2 (en) 2012-05-31
WO2004043978A2 (en) 2004-05-27
AU2010201712A1 (en) 2010-05-20
WO2004043978A3 (en) 2005-04-07

Similar Documents

Publication Publication Date Title
WO2004043977A3 (en) 2’-fluoro substituted oligomeric compounds and compositions for use in gene modulations
WO2004043979A3 (en) Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004041889A3 (en) Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2001002423A3 (en) 2'-guanidinyl-substituted oligonucleotides and gene expression modulation therewith
WO2004041924A3 (en) Non-phosphorous-linked oligomeric compounds and their use in gene modulation
EP1445312A4 (en) Method of inhibiting gene expression
CA2296769A1 (en) Modified dorsal tissue affecting factor and compositions
WO2004046160A8 (en) Amino-lna, thio-lna and alpha-l-oxy-ln
WO2001068836A3 (en) Methods and compositions for rna interference
AU6589094A (en) 7-deazapurine modified oligonucleotides
WO2002026967A3 (en) Targeted gene correction by single-stranded oligodeoxynucleotides
WO2000004158A3 (en) Compositions and methods for fumonisin detoxification
EP1443051A3 (en) Sugar modified oligonucleotides that detect and modulate gene expression
EP0977767A4 (en) Aminooxy-modified oligonucleotides
WO2002055692A3 (en) Method for inhibiting the expression of a target gene and medicament for treating a tumor disease
WO2001066553A3 (en) Novel aryl fructose-1,6-bisphosphatase inhibitors
EP1173614A4 (en) Antisense oligonucleotides comprising universal and/or degenerate bases
WO2000066739A3 (en) Chlamydia antigens and corresponding dna fragments and uses thereof
WO2004044134A3 (en) Phosphorous-linked oligomeric compounds and their use in gene modulation
WO2002102825A3 (en) Fluorescently labelled locked nucleic acids
WO2004044135A3 (en) Structural motifs and oligomeric compounds and their use in gene modulation
WO2004078941A3 (en) Modulation of gene expression using dna-rna hybrids
WO2000061594A3 (en) Nucleoside derivatives with photo-unstable protective groups
WO2004044137A3 (en) Modified oligomeric compounds and compositions for use in gene modulation
AU2002305073A1 (en) Synthesis of selenium-derivatized nucleosides, nucleotides, phosphoramidites, triphosphates and nucleic acids

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CO CR CU CZ DE DK DM DZ EC EE EG ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NO NZ OM PG PH PL PT RO RU SC SD SE SG SK SL SY TJ TM TN TR TT TZ UA UG US UZ VC VN YU ZA ZM ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): BW GH GM KE LS MW MZ SD SL SZ TZ UG ZM ZW AM AZ BY KG KZ MD RU TJ TM AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IT LU MC NL PT RO SE SI SK TR BF BJ CF CG CI CM GA GN GQ GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
122 Ep: pct application non-entry in european phase
NENP Non-entry into the national phase

Ref country code: JP

WWW Wipo information: withdrawn in national office

Country of ref document: JP