WO2003064441A3 - Oligonucleotides comprising alternating segments and uses thereof - Google Patents

Oligonucleotides comprising alternating segments and uses thereof Download PDF

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Publication number
WO2003064441A3
WO2003064441A3 PCT/CA2003/000129 CA0300129W WO03064441A3 WO 2003064441 A3 WO2003064441 A3 WO 2003064441A3 CA 0300129 W CA0300129 W CA 0300129W WO 03064441 A3 WO03064441 A3 WO 03064441A3
Authority
WO
WIPO (PCT)
Prior art keywords
oligonucleotides
alternating segments
sugar
methods
relates
Prior art date
Application number
PCT/CA2003/000129
Other languages
French (fr)
Other versions
WO2003064441A2 (en
Inventor
Masad J Damha
Michael A Parniak
Original Assignee
Univ Mcgill
Masad J Damha
Michael A Parniak
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 to AU2003202376A priority Critical patent/AU2003202376B2/en
Application filed by Univ Mcgill, Masad J Damha, Michael A Parniak filed Critical Univ Mcgill
Priority to US10/503,120 priority patent/US8278103B2/en
Priority to DE60325016T priority patent/DE60325016D1/en
Priority to DK03700774T priority patent/DK1470144T3/en
Priority to EP03700774A priority patent/EP1470144B1/en
Priority to JP2003564061A priority patent/JP2005522997A/en
Priority to CA2474414A priority patent/CA2474414C/en
Priority to MXPA04007403A priority patent/MXPA04007403A/en
Priority to NZ534396A priority patent/NZ534396A/en
Publication of WO2003064441A2 publication Critical patent/WO2003064441A2/en
Publication of WO2003064441A3 publication Critical patent/WO2003064441A3/en
Priority to US13/599,520 priority patent/US9902953B2/en
Priority to US14/847,873 priority patent/US20150368643A1/en

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • 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
    • A61P31/20—Antivirals for DNA viruses
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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

Abstract

The invention relates to oligonucleosides having alternating segments of sugar-modified nucleosides and 2’-deoxynucleosides, and uses thereof. The invention further relates to oligonucleotides having alternating segments of sugar-modified nucleotides and 2’-deoxynucleotides, and uses thereof. Such uses include the preparation of antisense oligonucleotides and their use for the prevention or depletion of function of a target nucleic acid of interest, such as an RNA, in a system. Accordingly, an oligonucleotide of the invention is useful for therapeutic, analytical and diagnostic methods and uses, as well as a component of compositions and commercial packages corresponding to such methods and uses.
PCT/CA2003/000129 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof WO2003064441A2 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
JP2003564061A JP2005522997A (en) 2002-02-01 2003-01-31 Oligonucleotides containing alternating segments and uses thereof
US10/503,120 US8278103B2 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof
DE60325016T DE60325016D1 (en) 2002-02-01 2003-01-31 OLIGONUCLEOTIDES WITH ALTERNATIVE SEGMENTS AND THEIR USES
DK03700774T DK1470144T3 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and applications thereof
EP03700774A EP1470144B1 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof
AU2003202376A AU2003202376B2 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof
CA2474414A CA2474414C (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof
MXPA04007403A MXPA04007403A (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof.
NZ534396A NZ534396A (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments of sugar-modified nucleosides and 2'-deoxynucleosides and uses thereof
US13/599,520 US9902953B2 (en) 2002-02-01 2012-08-30 Oligonucleotides comprising alternating segments and uses thereof
US14/847,873 US20150368643A1 (en) 2002-02-01 2015-09-08 Oligonucleotides comprising alternating segments and uses thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US35287302P 2002-02-01 2002-02-01
US60/352,873 2002-02-01

Related Child Applications (3)

Application Number Title Priority Date Filing Date
US10503120 A-371-Of-International 2003-01-31
US10/503,120 A-371-Of-International US8278103B2 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof
US13/599,520 Continuation US9902953B2 (en) 2002-02-01 2012-08-30 Oligonucleotides comprising alternating segments and uses thereof

Publications (2)

Publication Number Publication Date
WO2003064441A2 WO2003064441A2 (en) 2003-08-07
WO2003064441A3 true WO2003064441A3 (en) 2003-10-02

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/CA2003/000129 WO2003064441A2 (en) 2002-02-01 2003-01-31 Oligonucleotides comprising alternating segments and uses thereof

Country Status (13)

Country Link
US (3) US8278103B2 (en)
EP (1) EP1470144B1 (en)
JP (1) JP2005522997A (en)
AT (1) ATE416183T1 (en)
AU (1) AU2003202376B2 (en)
CA (1) CA2474414C (en)
DE (1) DE60325016D1 (en)
DK (1) DK1470144T3 (en)
ES (1) ES2318106T3 (en)
MX (1) MXPA04007403A (en)
NZ (1) NZ534396A (en)
PT (1) PT1470144E (en)
WO (1) WO2003064441A2 (en)

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JP2005522997A (en) 2005-08-04
US20150368643A1 (en) 2015-12-24
ES2318106T3 (en) 2009-05-01
US20130203977A1 (en) 2013-08-08
ATE416183T1 (en) 2008-12-15
EP1470144B1 (en) 2008-12-03
US8278103B2 (en) 2012-10-02
WO2003064441A2 (en) 2003-08-07
NZ534396A (en) 2006-11-30
DK1470144T3 (en) 2009-04-06
CA2474414A1 (en) 2003-08-07
MXPA04007403A (en) 2005-07-01
US20050142535A1 (en) 2005-06-30
US9902953B2 (en) 2018-02-27
DE60325016D1 (en) 2009-01-15
EP1470144A2 (en) 2004-10-27
CA2474414C (en) 2018-03-06
AU2003202376B2 (en) 2008-10-30
PT1470144E (en) 2009-02-10

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