SG11202112741XA - Modified gapmer oligonucleotides and methods of use - Google Patents

Modified gapmer oligonucleotides and methods of use

Info

Publication number
SG11202112741XA
SG11202112741XA SG11202112741XA SG11202112741XA SG11202112741XA SG 11202112741X A SG11202112741X A SG 11202112741XA SG 11202112741X A SG11202112741X A SG 11202112741XA SG 11202112741X A SG11202112741X A SG 11202112741XA SG 11202112741X A SG11202112741X A SG 11202112741XA
Authority
SG
Singapore
Prior art keywords
methods
gapmer oligonucleotides
modified gapmer
modified
oligonucleotides
Prior art date
Application number
SG11202112741XA
Other languages
English (en)
Inventor
Leonid Beigelman
Rajendra K Pandey
Vivek Kumar Rajwanshi
David Bernard Smith
Jin Hong
Original Assignee
Aligos Therapeutics 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
Application filed by Aligos Therapeutics Inc filed Critical Aligos Therapeutics Inc
Publication of SG11202112741XA publication Critical patent/SG11202112741XA/en

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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • C12N15/113—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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/323—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
SG11202112741XA 2019-05-31 2020-05-29 Modified gapmer oligonucleotides and methods of use SG11202112741XA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201962855793P 2019-05-31 2019-05-31
US201962937760P 2019-11-19 2019-11-19
US201962943532P 2019-12-04 2019-12-04
PCT/US2020/035212 WO2020243490A2 (en) 2019-05-31 2020-05-29 Modified gapmer oligonucleotides and methods of use

Publications (1)

Publication Number Publication Date
SG11202112741XA true SG11202112741XA (en) 2021-12-30

Family

ID=72179172

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202112741XA SG11202112741XA (en) 2019-05-31 2020-05-29 Modified gapmer oligonucleotides and methods of use

Country Status (15)

Country Link
US (1) US11466274B2 (ko)
EP (1) EP3976056A2 (ko)
JP (1) JP2022535735A (ko)
KR (1) KR20220024192A (ko)
CN (1) CN113905744A (ko)
AU (1) AU2020282828A1 (ko)
BR (1) BR112021023488A2 (ko)
CA (1) CA3141874A1 (ko)
CO (1) CO2021018034A2 (ko)
IL (1) IL288259A (ko)
MX (1) MX2021014206A (ko)
PE (1) PE20220574A1 (ko)
SG (1) SG11202112741XA (ko)
TW (1) TW202113079A (ko)
WO (1) WO2020243490A2 (ko)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021119325A1 (en) * 2019-12-12 2021-06-17 Aligos Therapeutics, Inc. S-antigen transport inhibiting oligonucleotide polymers and methods
WO2022026387A1 (en) * 2020-07-27 2022-02-03 Aligos Therapeutics, Inc. Hbv binding oligonucleotides and methods of use
AU2022339846A1 (en) 2021-09-01 2024-03-14 Aligos Therapeutics, Inc. Pnpla3-targeting short interfering rna (sirna) molecules and uses thereof
WO2023039076A1 (en) 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
WO2023177808A1 (en) * 2022-03-17 2023-09-21 Aligos Therapeutics, Inc. Modified gapmer oligomers and methods of use thereof

Family Cites Families (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040127446A1 (en) 1992-05-14 2004-07-01 Lawrence Blatt Oligonucleotide mediated inhibition of hepatitis B virus and hepatitis C virus replication
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
EP1569661B1 (en) 2002-11-18 2009-09-09 Santaris Pharma A/S Antisense design
WO2005009346A2 (en) 2003-06-24 2005-02-03 Mirus Corporation Inhibition of gene function by delivery of polynucleotide-based gene expression inhibitors to mammalian cells in vivo
JP5814505B2 (ja) 2006-04-03 2015-11-17 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブＲｏｃｈｅ Ｉｎｎｏｖａｔｉｏｎ Ｃｅｎｔｅｒ Ｃｏｐｅｎｈａｇｅｎ Ａ／Ｓ ａｎｔｉｍｉＲＮＡアンチセンスオリゴヌクレオチドを含む医薬組成物
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede mRNA
EA201290152A1 (ru) 2009-10-16 2012-12-28 Глаксо Груп Лимитед Антисмысловые ингибиторы вируса гепатита в (hbv)
WO2011052436A1 (ja) 2009-10-29 2011-05-05 国立大学法人大阪大学 架橋型人工ヌクレオシドおよびヌクレオチド
EP2673361B1 (en) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
RU2667524C2 (ru) * 2011-04-21 2018-09-21 Ионис Фармасьютикалз, Инк. Модулирование экспрессии вируса гепатита b (hbv)
EP2839006B1 (en) 2012-04-20 2018-01-03 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP2895434A2 (en) 2012-09-11 2015-07-22 3M Innovative Properties Company Glass granule having a zoned structure
US10377789B2 (en) 2012-09-21 2019-08-13 Osaka University Oligonucleotide and artificial nucleoside having guanidine bridge
MY173826A (en) 2012-11-15 2020-02-24 Roche Innovation Ct Copenhagen As Oligonucleotide conjugates
BR112015018161A2 (pt) 2013-01-30 2017-08-22 Hoffmann La Roche Conjugado de oligômeros antisense de lna, composição farmacêutica e uso de um conjugado de oligômeros antisense de lna
IL284593B2 (en) 2013-05-01 2023-02-01 Ionis Pharmaceuticals Inc Compositions and methods for modulation of hbv and ttr expression
WO2015042447A1 (en) 2013-09-20 2015-03-26 Isis Pharmaceuticals, Inc. Targeted therapeutic nucleosides and their use
WO2015113922A1 (en) 2014-01-30 2015-08-06 Roche Innovation Center Copenhagen A/S Poly oligomer compound with biocleavable conjugates
CN106103463B (zh) 2014-02-18 2018-11-30 国立大学法人大阪大学 桥连型核苷和核苷酸
GB201408623D0 (en) * 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
AU2015329974B2 (en) 2014-10-10 2019-06-20 F. Hoffmann-La Roche Ag GaINAc phosphoramidites, nucleic acid conjugates thereof and their use
JP2017536366A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス Ｌｎａキラルホスホロチオエート
US20210052631A1 (en) 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3587432A4 (en) 2017-02-21 2021-01-06 Osaka University COMPOUND OF NUCLEIC ACID AND OLIGONUCLEOTIDE
EP3587576A4 (en) 2017-02-21 2021-01-06 Osaka University ANTI-SENSE OLIGONUCLEIC ACID
WO2020097342A1 (en) 2018-11-08 2020-05-14 Aligos Therapeutics, Inc. S-antigen transport inhibiting oligonucleotide polymers and methods
AU2020221839A1 (en) 2019-02-14 2021-08-19 Aligos Therapeutics, Inc. Bicyclic sulfonamides

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Publication number Publication date
US20200385735A1 (en) 2020-12-10
CO2021018034A2 (es) 2022-04-19
AU2020282828A1 (en) 2022-01-27
JP2022535735A (ja) 2022-08-10
CN113905744A (zh) 2022-01-07
BR112021023488A2 (pt) 2022-01-18
KR20220024192A (ko) 2022-03-03
WO2020243490A3 (en) 2021-01-21
US11466274B2 (en) 2022-10-11
CA3141874A1 (en) 2020-12-03
TW202113079A (zh) 2021-04-01
EP3976056A2 (en) 2022-04-06
WO2020243490A2 (en) 2020-12-03
PE20220574A1 (es) 2022-04-20
MX2021014206A (es) 2022-01-06
IL288259A (en) 2022-01-01

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