WO1996018736A3 - Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses - Google Patents

Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses Download PDF

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Publication number
WO1996018736A3
WO1996018736A3 PCT/US1995/015516 US9515516W WO9618736A3 WO 1996018736 A3 WO1996018736 A3 WO 1996018736A3 US 9515516 W US9515516 W US 9515516W WO 9618736 A3 WO9618736 A3 WO 9618736A3
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WO
WIPO (PCT)
Prior art keywords
reversal
induction
reagent
promoter
treatment
Prior art date
Application number
PCT/US1995/015516
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French (fr)
Other versions
WO1996018736A2 (en
Inventor
Leonid Beigelman
Daniel T Stinchcomb
Thale Jarvis
Kenneth Draper
Pamela Pavco
James Mcswiggen
John Gustofson
Nassim Usman
Francine Wincott
Jasenka Matulic-Adamic
Alexander Karpeisky
James D Thompson
Anil Modak
Alex Burgin
Original Assignee
Ribozyme Pharm 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
Priority claimed from US08/363,253 external-priority patent/US5891683A/en
Priority claimed from US08/363,254 external-priority patent/US5891684A/en
Priority claimed from US08/390,850 external-priority patent/US5612215A/en
Priority claimed from US08/426,124 external-priority patent/US5716824A/en
Priority claimed from US08/432,874 external-priority patent/US5627053A/en
Priority claimed from US08/512,861 external-priority patent/US6146886A/en
Priority claimed from US08/541,365 external-priority patent/US5672501A/en
Priority to MX9704440A priority Critical patent/MX9704440A/en
Priority to EP95942944A priority patent/EP0799313A2/en
Application filed by Ribozyme Pharm Inc filed Critical Ribozyme Pharm Inc
Priority to JP51903596A priority patent/JP2001526521A/en
Priority to AU44120/96A priority patent/AU4412096A/en
Priority to US08/585,684 priority patent/US5877021A/en
Publication of WO1996018736A2 publication Critical patent/WO1996018736A2/en
Publication of WO1996018736A3 publication Critical patent/WO1996018736A3/en

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    • 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
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
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    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
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    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
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    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • C07H19/20—Purine radicals with the saccharide radical esterified by phosphoric or polyphosphoric 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
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    • 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
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    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/24—Metalloendopeptidases (3.4.24)
    • C12Y304/24017—Stromelysin 1 (3.4.24.17)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
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    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/321—2'-O-R Modification
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    • 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

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  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • Plant Pathology (AREA)
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  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

An enzymatic nucleic acid molecule which cleaves RNA associated with development or maintenance of an arthritic condition, induction of graft tolerance or reversal of an immune response. In particular, the ribozyme sequences are directed to an mRNA encoding B7-1, B7-2, B7-3, CD40 and/or stromelysin. Also provided are ribozymes where the uracil in positions 4 and/or 7 are substituted, as well as methods for the synthesis of 2'-alkylnucleotides, 2'-O-alkylthioalkyl, or 2'-alkylthioalkylnucleotides. The application further describes a method for diprotection of RNA with aqueous ethylamine, a method for synthesis of a basic ribonucleoside mimetics, and transcription units comprising an RNA polymerase II promoter, a U6 small nuclear promoter, or an adenovirus VA1 promoter system.
PCT/US1995/015516 1994-12-13 1995-11-22 Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses WO1996018736A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
AU44120/96A AU4412096A (en) 1994-12-13 1995-11-22 Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
JP51903596A JP2001526521A (en) 1994-12-13 1995-11-22 Methods and reagents for treating arthritic conditions, inducing graft tolerance and reversing the immune response
MX9704440A MX9704440A (en) 1994-12-13 1995-11-22 Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses.
EP95942944A EP0799313A2 (en) 1994-12-13 1995-11-22 Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
US08/585,684 US5877021A (en) 1995-07-07 1996-01-12 B7-1 targeted ribozymes

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US35492094A 1994-12-13 1994-12-13
US08/354,920 1994-12-13
US08/363,253 US5891683A (en) 1993-09-02 1994-12-23 Non-nucleotide containing enzymatic nucleic acid
US08/363,254 US5891684A (en) 1992-10-15 1994-12-23 Base-modified enzymatic nucleic acid
US08/363,253 1994-12-23
US08/363,254 1994-12-23
US08/390,850 US5612215A (en) 1992-12-07 1995-02-17 Stromelysin targeted ribozymes
US08/390,850 1995-02-17
US08/426,124 US5716824A (en) 1995-04-20 1995-04-20 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US08/426,124 1995-04-20
US08/432,874 US5627053A (en) 1994-03-29 1995-05-02 2'deoxy-2'-alkylnucleotide containing nucleic acid
US08/432,874 1995-05-02
US43450995A 1995-05-04 1995-05-04
US08/434,509 1995-05-04
US60/000,974 1995-07-07
US60/000,951 1995-07-07
US08/512,861 US6146886A (en) 1994-08-19 1995-08-07 RNA polymerase III-based expression of therapeutic RNAs
US08/512,861 1995-08-07
US08/541,365 US5672501A (en) 1994-12-23 1995-10-05 Base-modified enzymatic nucleic acid
US08/541,365 1995-10-05

Publications (2)

Publication Number Publication Date
WO1996018736A2 WO1996018736A2 (en) 1996-06-20
WO1996018736A3 true WO1996018736A3 (en) 1996-09-19

Family

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Application Number Title Priority Date Filing Date
PCT/US1995/015516 WO1996018736A2 (en) 1994-12-13 1995-11-22 Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses

Country Status (3)

Country Link
AU (1) AU4412096A (en)
CA (1) CA2207593A1 (en)
WO (1) WO1996018736A2 (en)

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US8957198B2 (en) 2003-02-03 2015-02-17 Medtronic, Inc. Compositions, devices and methods for treatment of Huntington's disease through intracranial delivery of sirna
US9133517B2 (en) 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
US9273356B2 (en) 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
US9375440B2 (en) 2006-11-03 2016-06-28 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity

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US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
WO1998028317A2 (en) * 1996-12-24 1998-07-02 Ribozyme Pharmaceuticals, Inc. Synthesis of nucleosides and polynucleotides
US6319906B1 (en) * 1996-12-31 2001-11-20 Isis Pharmaceuticals Oligonucleotide compositions and methods for the modulation of the expression of B7 protein
JP2001525667A (en) * 1997-05-09 2001-12-11 リボザイム・ファーマシューティカルズ・インコーポレーテッド Enzymatic nucleic acid treatment of diseases or conditions related to the expression level of C-RAF
US6548657B1 (en) 1997-06-09 2003-04-15 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6280936B1 (en) 1997-06-09 2001-08-28 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6183959B1 (en) 1997-07-03 2001-02-06 Ribozyme Pharmaceuticals, Inc. Method for target site selection and discovery
US6316612B1 (en) 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
US6656731B1 (en) 1997-09-22 2003-12-02 Max Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
WO1999016871A2 (en) * 1997-09-22 1999-04-08 Max-Planck-Gesellschaft Zur Forderung Der Wissensc Nucleic acid catalysts with endonuclease activity
US6127535A (en) 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
WO1999054459A2 (en) 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
JP2002512794A (en) * 1998-04-29 2002-05-08 リボザイム・ファーマシューティカルズ・インコーポレーテッド Nucleotide triphosphates and their incorporation into ribozymes
EP1493818A3 (en) * 1998-04-29 2006-02-15 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
EP1114827B1 (en) * 1999-07-15 2010-11-17 Japan Science and Technology Agency Novel nucleic acid base pair
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
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US20050148530A1 (en) 2002-02-20 2005-07-07 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)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20050043256A1 (en) 2001-07-30 2005-02-24 Isis Pharmaceuticals, Inc. Antisense modulation of stearoyl-CoA desaturase expression
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US7618948B2 (en) 2002-11-26 2009-11-17 Medtronic, Inc. Devices, systems and methods for improving and/or cognitive function through brain delivery of siRNA
US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US7732591B2 (en) 2003-11-25 2010-06-08 Medtronic, Inc. Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
US7960355B2 (en) 2003-05-23 2011-06-14 Isis Pharmaceuticals, Inc. Compositions and methods for the modulation of the expression of B7 protein
US7897582B2 (en) 2003-05-23 2011-03-01 Isis Pharmaceuticals, Inc. Oligonucleotide compositions and methods for the modulation of the expression of B7 protein
EP1704250B1 (en) 2003-12-31 2012-09-19 The Penn State Research Foundation Methods for predicting and overcoming resistance to chemotherapy in ovarian cancer
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
EP2298896A1 (en) 2004-06-22 2011-03-23 The Board of Trustees of the University of Illinois Methods of inhibiting tumor cell proliferation with FOXM1 siRNA
US7968521B2 (en) 2005-06-24 2011-06-28 Yamasa Corporation Stable cystal of protected pseudouridine
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US8067391B2 (en) 2005-10-03 2011-11-29 University Health Network ODCase inhibitors for the treatment of malaria
CN103215293B (en) 2006-01-27 2015-10-28 比奥根Ma公司 NOGO receptor antagonist
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EP2056845B1 (en) 2006-08-08 2017-10-11 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
JP5689413B2 (en) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 5 'triphosphate oligonucleotide having blunt ends and uses thereof
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