DK2360253T3 - Fremgangsmåde til fremstilling af knockdown-celler eller knockdown-organismer ved hjælp af RNA-sekvensspecifikke formidlere af RNA-interferens og anvendelser deraf - Google Patents

Fremgangsmåde til fremstilling af knockdown-celler eller knockdown-organismer ved hjælp af RNA-sekvensspecifikke formidlere af RNA-interferens og anvendelser deraf

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Publication number
DK2360253T3
DK2360253T3 DK10184520.4T DK10184520T DK2360253T3 DK 2360253 T3 DK2360253 T3 DK 2360253T3 DK 10184520 T DK10184520 T DK 10184520T DK 2360253 T3 DK2360253 T3 DK 2360253T3
Authority
DK
Denmark
Prior art keywords
rna
knockdown
dsrna
organisms
applications
Prior art date
Application number
DK10184520.4T
Other languages
English (en)
Inventor
Thomas Tuschl
Phillip A Sharp
Phillip D Zamore
David P Bartel
Original Assignee
Whitehead Biomedical Inst
Max Planck Ges Zur Förderung Der Wissenschaften E V
Massachusetts Inst Technology
Univ Massachusetts
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=41655850&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK2360253(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Whitehead Biomedical Inst, Max Planck Ges Zur Förderung Der Wissenschaften E V, Massachusetts Inst Technology, Univ Massachusetts filed Critical Whitehead Biomedical Inst
Priority claimed from EP20080168152 external-priority patent/EP2028278B1/en
Application granted granted Critical
Publication of DK2360253T3 publication Critical patent/DK2360253T3/da

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1079—Screening libraries by altering the phenotype or phenotypic trait of the host
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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 N.A.
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
DK10184520.4T 2000-03-30 2001-03-30 Fremgangsmåde til fremstilling af knockdown-celler eller knockdown-organismer ved hjælp af RNA-sekvensspecifikke formidlere af RNA-interferens og anvendelser deraf DK2360253T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US19359400P 2000-03-30 2000-03-30
EP00126325 2000-12-01
US26523201P 2001-01-31 2001-01-31
EP20080168152 EP2028278B1 (en) 2000-03-30 2001-03-30 RNA sequence-specific mediators of RNA interference

Publications (1)

Publication Number Publication Date
DK2360253T3 true DK2360253T3 (da) 2014-06-16

Family

ID=41655850

Family Applications (7)

Application Number Title Priority Date Filing Date
DK10184520.4T DK2360253T3 (da) 2000-03-30 2001-03-30 Fremgangsmåde til fremstilling af knockdown-celler eller knockdown-organismer ved hjælp af RNA-sekvensspecifikke formidlere af RNA-interferens og anvendelser deraf
DK08168152.0T DK2028278T3 (da) 2000-03-30 2001-03-30 RNA-sekvensspecifikke formidlere af RNA-interferens
DK10184660.8T DK2361981T4 (da) 2000-03-30 2001-03-30 Rna-sekvensspecifikke mediatorer af rna-interferens
DK10184711.9T DK2345742T3 (da) 2000-03-30 2001-03-30 RNA-sekvensspecifikke formidlere af RNA-interferens
DK10179952.6T DK2348133T3 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som formidler RNA-interferens
DK10179947.6T DK2351852T4 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som medierer RNA-interferens
DK10180025.8T DK2348134T3 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som formidler RNA-interferens

Family Applications After (6)

Application Number Title Priority Date Filing Date
DK08168152.0T DK2028278T3 (da) 2000-03-30 2001-03-30 RNA-sekvensspecifikke formidlere af RNA-interferens
DK10184660.8T DK2361981T4 (da) 2000-03-30 2001-03-30 Rna-sekvensspecifikke mediatorer af rna-interferens
DK10184711.9T DK2345742T3 (da) 2000-03-30 2001-03-30 RNA-sekvensspecifikke formidlere af RNA-interferens
DK10179952.6T DK2348133T3 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som formidler RNA-interferens
DK10179947.6T DK2351852T4 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som medierer RNA-interferens
DK10180025.8T DK2348134T3 (da) 2000-12-01 2001-11-29 Små RNA-molekyler, som formidler RNA-interferens

Country Status (11)

Country Link
EP (7) EP2796553B1 (da)
CN (2) CN101654672B (da)
AT (1) ATE542899T1 (da)
BR (4) BRPI0117341B1 (da)
CA (1) CA2791831C (da)
DK (7) DK2360253T3 (da)
ES (9) ES2461715T3 (da)
LT (1) LT2813582T (da)
PL (1) PL218881B1 (da)
PT (8) PT2360253E (da)
SI (4) SI2348134T1 (da)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2404890C (en) 2000-03-30 2013-11-19 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
BRPI0115814B8 (pt) 2000-12-01 2021-05-25 Europaeisches Laboratorium Fuer Molekularbiologie Embl moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
EP3415625A1 (en) 2002-02-01 2018-12-19 Life Technologies Corporation Double-stranded oligonucleotides
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
DE102018001134A1 (de) * 2017-06-26 2018-12-27 Martin-Luther-Universität Halle-Wittenberg Methode zur gezielten Identifizierung hocheffizienter "small interfering RNAs" ("eRNAs") zur Anwendung in Pflanzen und anderen Zielorganismen
CN111902148A (zh) 2017-12-06 2020-11-06 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
EP4121063A1 (en) 2020-03-19 2023-01-25 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS59198885A (ja) 1983-04-25 1984-11-10 Nec Corp 圧電アクチェータ励振回路
US5652355A (en) † 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
EP0743859A4 (en) † 1993-11-16 1998-10-21 Genta Inc CHEMICAL OLIGONUCLEOSIDE COMPOUNDS
US6506559B1 (en) † 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
AU3751299A (en) † 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
DE19956568A1 (de) * 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2003526367A (ja) * 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Ｒｎａ干渉の方法とｒｎａ干渉組成物
CA2404890C (en) * 2000-03-30 2013-11-19 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
BRPI0115814B8 (pt) * 2000-12-01 2021-05-25 Europaeisches Laboratorium Fuer Molekularbiologie Embl moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas

Also Published As

Publication number Publication date
ES2498747T3 (es) 2014-09-25
PT2813582T (pt) 2017-07-17
PT2345742E (pt) 2014-09-03
DK2361981T4 (da) 2019-05-13
CN101643789A (zh) 2010-02-10
PT2360253E (pt) 2014-05-29
ES2500923T3 (es) 2014-10-01
ATE542899T1 (de) 2012-02-15
PT2361981E (pt) 2013-06-12
SI2351852T1 (sl) 2013-12-31
EP2361981B2 (en) 2019-01-23
EP2351852B2 (en) 2020-07-01
CN101654672B (zh) 2013-06-05
ES2410907T3 (es) 2013-07-03
BRPI0117338A2 (pt) 2011-08-02
CN101654673A (zh) 2010-02-24
EP3199631A1 (en) 2017-08-02
SI2351852T2 (sl) 2020-10-30
DK2345742T3 (da) 2014-09-15
ES2632957T3 (es) 2017-09-18
CA2791831A1 (en) 2002-06-06
EP2348134A1 (en) 2011-07-27
EP2348134B1 (en) 2014-04-16
SI2348134T1 (sl) 2014-08-29
BRPI0117339B1 (pt) 2016-03-15
BRPI0117338B1 (pt) 2016-06-21
PT2348133E (pt) 2014-10-14
BRPI0117341A2 (pt) 2011-08-02
BRPI0117341B1 (pt) 2016-06-21
SI2348133T1 (sl) 2014-11-28
CN101643789B (zh) 2013-04-24
EP2796553B1 (en) 2019-06-19
EP2360253A1 (en) 2011-08-24
DK2348133T3 (da) 2014-10-13
ES2437390T5 (es) 2021-02-05
ES2461715T3 (es) 2014-05-21
PL218881B1 (pl) 2015-02-27
ES2380171T3 (es) 2012-05-09
CA2791831C (en) 2015-06-23
EP2361981A1 (en) 2011-08-31
ES2437390T3 (es) 2014-01-10
EP2351852A1 (en) 2011-08-03
LT2813582T (lt) 2017-07-25
DK2028278T3 (da) 2014-06-23
EP2813582B1 (en) 2017-04-12
EP2360253B1 (en) 2014-03-19
CN101654672A (zh) 2010-02-24
EP2796553A1 (en) 2014-10-29
ES2461765T3 (es) 2014-05-21
PT2028278E (pt) 2014-05-28
EP2351852B1 (en) 2013-10-02
DK2351852T3 (da) 2014-01-06
BRPI0117340B1 (pt) 2016-10-18
ES2410907T5 (es) 2019-08-20
EP3199631B1 (en) 2019-01-30
EP2361981B1 (en) 2013-03-06
DK2361981T3 (da) 2013-06-24
PT2348134E (pt) 2014-07-16
ES2462716T3 (es) 2014-05-26
SI2813582T1 (sl) 2017-08-31
DK2351852T4 (da) 2020-08-24
EP2813582A1 (en) 2014-12-17
PT2351852E (pt) 2014-01-03
DK2348134T3 (da) 2014-07-14

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