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WO2001016312A3 - Nucleic acid based modulators of gene expression - Google Patents

Nucleic acid based modulators of gene expression Download PDF

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Publication number
WO2001016312A3
WO2001016312A3 PCT/US2000/023998 US0023998W WO0116312A3 WO 2001016312 A3 WO2001016312 A3 WO 2001016312A3 US 0023998 W US0023998 W US 0023998W WO 0116312 A3 WO0116312 A3 WO 0116312A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
gene expression
acid based
acid molecules
based modulators
Prior art date
Application number
PCT/US2000/023998
Other languages
French (fr)
Other versions
WO2001016312A2 (en
Inventor
James Mcswiggen
Nassim Usman
Lawrence Blatt
Leonid Beigelman
Alex Burgin
Alexander Karpeisky
Jasenka Matulic-Adamic
David Sweedler
Kenneth Draper
Bharat Chowrira
Dan Stinchcomb
Amber Beaudry
Shawn Zinnen
Janos Lugwig
Brian S Sproat
Original Assignee
Ribozyme Pharm Inc
James Mcswiggen
Nassim Usman
Lawrence Blatt
Leonid Beigelman
Alex Burgin
Alexander Karpeisky
Matulic Adamic Jasenka
David Sweedler
Kenneth Draper
Bharat Chowrira
Dan Stinchcomb
Amber Beaudry
Shawn Zinnen
Janos Lugwig
Brian S Sproat
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 US09/474,432 external-priority patent/US6528640B1/en
Priority claimed from US09/476,387 external-priority patent/US6617438B1/en
Application filed by Ribozyme Pharm Inc, James Mcswiggen, Nassim Usman, Lawrence Blatt, Leonid Beigelman, Alex Burgin, Alexander Karpeisky, Matulic Adamic Jasenka, David Sweedler, Kenneth Draper, Bharat Chowrira, Dan Stinchcomb, Amber Beaudry, Shawn Zinnen, Janos Lugwig, Brian S Sproat filed Critical Ribozyme Pharm Inc
Priority to JP2001520859A priority Critical patent/JP2004512810A/en
Priority to CA002403243A priority patent/CA2403243A1/en
Priority to EP00963298A priority patent/EP1212416A2/en
Priority to AU74733/00A priority patent/AU7473300A/en
Publication of WO2001016312A2 publication Critical patent/WO2001016312A2/en
Publication of WO2001016312A3 publication Critical patent/WO2001016312A3/en

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P3/04—Anorexiants; Antiobesity agents
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
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    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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    • 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
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    • 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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    • 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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    • C12P19/00—Preparation of compounds containing saccharide radicals
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)
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    • C12Y301/03048—Protein-tyrosine-phosphatase (3.1.3.48)
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Abstract

Nucleic acid molecules (antisenses or ribozymes) useful as inhibitors of gene expression, especially of HER2, BACE, TERT, PTP-1B, MetAP-2, HBV, phospholamban, presenilin-2 and PKC-alpha. The nucleic acid molecules can be modified in various ways on the sugar and/or base moieties and/or on the phosphate backbone. They are used in pharmaceutical formulations for the treatment of diseases involving increased expression of the target genes. Also disclosed is a method for the synthesis of a modified pyrimidine nucleotide triphosphate and its incorporation into an oligonucleotide.
PCT/US2000/023998 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression WO2001016312A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
JP2001520859A JP2004512810A (en) 1999-08-31 2000-08-30 Modulators of gene expression based on nucleic acids
CA002403243A CA2403243A1 (en) 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression
EP00963298A EP1212416A2 (en) 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression
AU74733/00A AU7473300A (en) 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression

Applications Claiming Priority (28)

Application Number Priority Date Filing Date Title
US15171399P 1999-08-31 1999-08-31
US60/151,713 1999-08-31
US40664399A 1999-09-27 1999-09-27
US15623699P 1999-09-27 1999-09-27
US15646799P 1999-09-27 1999-09-27
US60/156,467 1999-09-27
US60/156,236 1999-09-27
US09/406,643 1999-09-27
US43643099A 1999-11-08 1999-11-08
US09/436,430 1999-11-08
US16910099P 1999-12-06 1999-12-06
US60/169,100 1999-12-06
US17361299P 1999-12-29 1999-12-29
US09/474,432 US6528640B1 (en) 1997-11-05 1999-12-29 Synthetic ribonucleic acids with RNAse activity
US09/474,432 1999-12-29
US60/173,612 1999-12-29
US09/476,387 US6617438B1 (en) 1997-11-05 1999-12-30 Oligoribonucleotides with enzymatic activity
US09/476,387 1999-12-30
US49882400A 2000-02-04 2000-02-04
US09/498,824 2000-02-04
US53102500A 2000-03-20 2000-03-20
US09/531,025 2000-03-20
US19776900P 2000-04-14 2000-04-14
US60/197,769 2000-04-14
US57822300A 2000-05-23 2000-05-23
US09/578,223 2000-05-23
US63638500A 2000-08-09 2000-08-09
US09/636,385 2000-08-09

Publications (2)

Publication Number Publication Date
WO2001016312A2 WO2001016312A2 (en) 2001-03-08
WO2001016312A3 true WO2001016312A3 (en) 2001-08-09

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2000/023998 WO2001016312A2 (en) 1999-08-31 2000-08-30 Nucleic acid based modulators of gene expression

Country Status (4)

Country Link
EP (1) EP1212416A2 (en)
JP (1) JP2004512810A (en)
CA (1) CA2403243A1 (en)
WO (1) WO2001016312A2 (en)

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US9034841B2 (en) 2011-04-21 2015-05-19 Isis Pharmaceuticals, Inc. Modulation of hepatitis B virus (HBV) expression
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

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6482932B1 (en) * 1997-11-05 2002-11-19 Ribozyme Pharmaceuticals, Incorporated Nucleoside triphosphates and their incorporation into oligonucleotides
US6528640B1 (en) * 1997-11-05 2003-03-04 Ribozyme Pharmaceuticals, Incorporated Synthetic ribonucleic acids with RNAse activity
US6617438B1 (en) * 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US20020055479A1 (en) * 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US7179796B2 (en) 2000-01-18 2007-02-20 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
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