ATE503014T1 - Rna-amidate und thioamidateur rnai - Google Patents

Rna-amidate und thioamidateur rnai

Info

Publication number
ATE503014T1
ATE503014T1 AT04818592T AT04818592T ATE503014T1 AT E503014 T1 ATE503014 T1 AT E503014T1 AT 04818592 T AT04818592 T AT 04818592T AT 04818592 T AT04818592 T AT 04818592T AT E503014 T1 ATE503014 T1 AT E503014T1
Authority
AT
Austria
Prior art keywords
rna
thioamidates
amidates
thioamidateur
amidate
Prior art date
Application number
AT04818592T
Other languages
English (en)
Inventor
Sergei Gryaznov
Krisztina Pongracz
Original Assignee
Geron Corp
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 Geron Corp filed Critical Geron Corp
Application granted granted Critical
Publication of ATE503014T1 publication Critical patent/ATE503014T1/de

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
    • 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
    • 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
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
    • 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
    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • AIDS & HIV (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
AT04818592T 2003-11-04 2004-11-03 Rna-amidate und thioamidateur rnai ATE503014T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US51676903P 2003-11-04 2003-11-04
PCT/US2004/032780 WO2005047506A1 (en) 2003-11-04 2004-11-03 Rna amidates and thioamidates for rnai

Publications (1)

Publication Number Publication Date
ATE503014T1 true ATE503014T1 (de) 2011-04-15

Family

ID=34590131

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04818592T ATE503014T1 (de) 2003-11-04 2004-11-03 Rna-amidate und thioamidateur rnai

Country Status (7)

Country Link
US (3) US9133233B2 (de)
EP (1) EP1694842B1 (de)
AT (1) ATE503014T1 (de)
AU (1) AU2004289975B2 (de)
CA (1) CA2544349C (de)
DE (1) DE602004031975D1 (de)
WO (1) WO2005047506A1 (de)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7338117B2 (en) 2003-09-25 2008-03-04 W.E.T. Automotive System, Ltd. Ventilated seat
US9133233B2 (en) * 2003-11-04 2015-09-15 Geron Corporation RNA amidates and thioamidates for RNAi
ME02933B (de) 2004-07-02 2018-04-20 Geron Corp Synthese geschützter 3'-aminonukleosidmonomere
US8785409B2 (en) 2007-01-30 2014-07-22 Geron Corporation Compounds having anti-adhesive effects on cancer cells
EP2490699A1 (de) 2009-10-20 2012-08-29 Santaris Pharma A/S Orale freisetzung von therapeutisch wirksamen lna-oligonukleotiden
US9375485B2 (en) 2012-12-07 2016-06-28 Geron Corporation Use of telomerase inhibitors for the treatment of myeloproliferative disorders and myeloproliferative neoplasms
US9708360B2 (en) 2013-09-30 2017-07-18 Geron Corporation Phosphorodiamidate backbone linkage for oligonucleotides
MX2019002960A (es) 2016-09-14 2019-09-18 Janssen Biopharma Inc Oligonucleótidos modificados y métodos de uso.
KR20250151565A (ko) 2017-07-28 2025-10-21 제론 코포레이션 골수형성이상 증후군의 치료 방법
AU2018332214B2 (en) * 2017-09-14 2023-11-02 Janssen Biopharma, Inc. Modified nucleoside phosphoramidites
AU2019389005B2 (en) 2018-11-29 2024-06-20 Geron Corporation Methods of treating myelodysplastic syndrome
WO2021198958A1 (en) * 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
WO2025207561A1 (en) * 2024-03-27 2025-10-02 Cepheid Isothermal amplification using novel synthetic oligonucleotides

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5599922A (en) 1994-03-18 1997-02-04 Lynx Therapeutics, Inc. Oligonucleotide N3'-P5' phosphoramidates: hybridization and nuclease resistance properties
JP3482209B2 (ja) 1994-03-18 2003-12-22 ジェロン・コーポレーション オリゴヌクレオチドｎ３’→ｐ５’ホスホルアミデート：合成および化合物；ハイブリダイゼーションおよびヌクレアーゼ耐性特性
US5726297A (en) 1994-03-18 1998-03-10 Lynx Therapeutics, Inc. Oligodeoxyribonucleotide N3' P5' phosphoramidates
US20030026782A1 (en) * 1995-02-07 2003-02-06 Arthur M. Krieg Immunomodulatory oligonucleotides
US5859233A (en) 1996-02-21 1999-01-12 Lynx Therapeutics, Inc. Synthons for synthesis of oligonucleotide N3-P5 phosphoramidates
US5684143A (en) 1996-02-21 1997-11-04 Lynx Therapeutics, Inc. Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates
EP2314700A1 (de) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Zusammensetzung und Verfahren zur in vivo und in vitro Abschwächung der Genexpression mittels dobbelsträngiger RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
DK1210357T3 (da) 1999-09-10 2008-07-21 Geron Corp Oligonukleotid-N3' -P5' -thiophosphoramidater: syntese og anvendelse deraf
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
EP1272630A2 (de) 2000-03-16 2003-01-08 Genetica, Inc. Methoden und zusammensetzungen zur interferenz durch rna
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU2003210671A1 (en) * 2002-01-24 2003-09-02 Chiron Corporation Nek2 inhibitors
US7897752B2 (en) 2002-02-20 2011-03-01 Sirna Therapeutics, Inc. RNA interference mediated inhibition of telomerase gene expression using short interfering nucleic acid (siNA)
PL375356A1 (en) * 2002-05-20 2005-11-28 Immunex Corporation Claudin polypeptides, polynucleotides, and methods of making and use thereof
AU2003241621A1 (en) * 2002-05-24 2003-12-12 Isis Pharmaceuticals, Inc. Oligonucleotides having modified nucleoside units
WO2004003152A2 (en) * 2002-06-26 2004-01-08 Chiron Corporation Sos1 inhibitors
WO2004014312A2 (en) * 2002-08-08 2004-02-19 Sirna Therapeutics, Inc. Small-mer compositions and methods of use
CA2500478A1 (en) * 2002-09-27 2004-04-08 Biomira, Inc. Glycosylceramide analogues
EP2000160A3 (de) * 2002-10-30 2009-03-11 Gambro Lundia AB Verfahren und Vorrichtungen zur Bestimmung der Dialyseeffizienz
US20060275769A1 (en) * 2003-01-06 2006-12-07 Oregon Health & Science University Methods of treatment and diagnosis of kaposi's sarcoma (ks) and ks related diseases
AU2004220556B2 (en) 2003-03-07 2009-05-07 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
EP2669377A3 (de) * 2003-04-17 2015-10-14 Alnylam Pharmaceuticals Inc. Modifizierte iRNA-Wirkstoffe
WO2005044976A2 (en) * 2003-06-20 2005-05-19 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
AU2004320900A1 (en) * 2003-07-15 2006-02-23 California Institute Of Technology Improved inhibitor nucleic acids
KR101298493B1 (ko) 2003-09-09 2013-08-21 제론 코포레이션 텔로머라제 억제를 위한 변형 올리고뉴클레오티드
US9133233B2 (en) 2003-11-04 2015-09-15 Geron Corporation RNA amidates and thioamidates for RNAi
ME02933B (de) 2004-07-02 2018-04-20 Geron Corp Synthese geschützter 3'-aminonukleosidmonomere
US9228189B2 (en) * 2012-10-26 2016-01-05 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders

Also Published As

Publication number Publication date
AU2004289975B2 (en) 2011-11-03
AU2004289975A1 (en) 2005-05-26
US9822360B2 (en) 2017-11-21
DE602004031975D1 (de) 2011-05-05
CA2544349A1 (en) 2005-05-26
WO2005047506A1 (en) 2005-05-26
CA2544349C (en) 2020-02-18
US20180119147A1 (en) 2018-05-03
US9133233B2 (en) 2015-09-15
EP1694842B1 (de) 2011-03-23
EP1694842A1 (de) 2006-08-30
US20160130580A1 (en) 2016-05-12
US10655127B2 (en) 2020-05-19
US20070275919A1 (en) 2007-11-29

Similar Documents

Publication Publication Date Title
ATE503014T1 (de) Rna-amidate und thioamidateur rnai
WO2005007196A3 (en) Lipid encapsulated interfering rna
WO2006113679A3 (en) Delivery of sirna by neutral lipid compositions
EP1766035A4 (de) Lipidverkapselte interferenz-rna
MX2009003548A (es) Formulaciones que contienen lipidos.
WO2022120388A3 (en) Ionizable cationic lipids and lipid nanoparticles, and methods of synthesis and use thereof
EP3449946A3 (de) Hydrophobe kernträgerzusammensetzungen zur verabreichung von therapeutika, verfahren zur herstellung und verwendung davon
MX2007004321A (es) Micro acidos ribonucleicos.
CY1112420T1 (el) Ρυθμιση της γονιδιακης εκφρασης στα φυτα με μεσολαβηση dsrνα
WO2014127287A8 (en) Method for in vivo targeted mutagenesis
NZ596958A (en) Improved lipid formulation
EP2304026A4 (de) Nanopartikelzusammensetzungen für ein system zur ausgabe von nukleinsäuren
WO2006036916A3 (en) Rnai modulation of apob and uses thereof
WO2008131419A3 (en) Glycoconjugates of rna interference agents
WO2008092081A8 (en) Targeted delivery of sirna
SG165394A1 (en) Modulation of immunostimulatory properties of short interfering ribonucleic acid (sirna) by nucleotide modification
AU2014219019A8 (en) Short interfering nucleic acid (siNA) molecules containing a 2' internucleoside linkage
WO2008094640A3 (en) Compounds having anti-adhesive effects on cancer cells
MX2024011671A (es) Oligonucleotidos modificados
TR201820474T4 (tr) Spesifik immün modülatör profilleri indükleyen tanımlı nükleotitler arası bağlar bağlamında rna sekansı motifleri.
WO2007014075A3 (en) Rnai modulation of the rho-a gene in research models
ATE550024T1 (de) Gezielte oligonucleotid-zusammensetzungen zur modifizierung der genexpression
Su et al. Neutral and negatively charged phosphate modifications altering thermal stability, kinetics of formation and monovalent ion dependence of DNA G‐Quadruplexes
WO2005054439A3 (en) Small interfering rna (sirna)-mediated heritable gene manipulation in plants
EP4591931A3 (de) Gegen muc5b gerichtete antisense-oligonukleotide und zugehörige verfahren zur modulation der mucinexpression

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties