IL265359B2 - Modified oligonucleotides and pharmaceutical preparations containing them - Google Patents

Modified oligonucleotides and pharmaceutical preparations containing them

Info

Publication number
IL265359B2
IL265359B2 IL265359A IL26535919A IL265359B2 IL 265359 B2 IL265359 B2 IL 265359B2 IL 265359 A IL265359 A IL 265359A IL 26535919 A IL26535919 A IL 26535919A IL 265359 B2 IL265359 B2 IL 265359B2
Authority
IL
Israel
Prior art keywords
oligonucleotide
sequence
pharmaceutical composition
target rna
linked
Prior art date
Application number
IL265359A
Other languages
English (en)
Hebrew (he)
Other versions
IL265359A (en
IL265359B1 (en
Original Assignee
Janssen Biopharma Llc
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 Janssen Biopharma Llc filed Critical Janssen Biopharma Llc
Publication of IL265359A publication Critical patent/IL265359A/en
Publication of IL265359B1 publication Critical patent/IL265359B1/en
Publication of IL265359B2 publication Critical patent/IL265359B2/en

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
    • 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
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • 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
    • 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
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/31—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
    • C12N2310/34—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
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • 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
    • C12N2310/3525—MOE, methoxyethoxy
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
IL265359A 2016-09-14 2017-09-14 Modified oligonucleotides and pharmaceutical preparations containing them IL265359B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662394738P 2016-09-14 2016-09-14
US201662394739P 2016-09-14 2016-09-14
PCT/US2017/051644 WO2018053185A1 (en) 2016-09-14 2017-09-14 Modified oligonucleotides and methods of use

Publications (3)

Publication Number Publication Date
IL265359A IL265359A (en) 2019-05-30
IL265359B1 IL265359B1 (en) 2024-10-01
IL265359B2 true IL265359B2 (en) 2025-02-01

Family

ID=59955744

Family Applications (2)

Application Number Title Priority Date Filing Date
IL265359A IL265359B2 (en) 2016-09-14 2017-09-14 Modified oligonucleotides and pharmaceutical preparations containing them
IL315071A IL315071A (en) 2016-09-14 2017-09-14 Modified oligonucleotides and pharmaceutical compositions

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL315071A IL315071A (en) 2016-09-14 2017-09-14 Modified oligonucleotides and pharmaceutical compositions

Country Status (13)

Country Link
US (3) US10793859B2 (enExample)
EP (1) EP3512949A1 (enExample)
JP (3) JP6970187B2 (enExample)
KR (3) KR20240010761A (enExample)
CN (1) CN109952378B (enExample)
AU (2) AU2017326372B2 (enExample)
BR (1) BR112019004911A2 (enExample)
CA (1) CA3037042A1 (enExample)
IL (2) IL265359B2 (enExample)
MA (1) MA46422A (enExample)
MX (2) MX2019002960A (enExample)
TW (1) TWI769177B (enExample)
WO (1) WO2018053185A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018053185A1 (en) 2016-09-14 2018-03-22 Alios Biopharma, Inc. Modified oligonucleotides and methods of use
EA202090719A1 (ru) 2017-09-14 2020-07-01 Янссен Байофарма, Инк. Модифицированные нуклеозидные амидофосфиты
EP3682007A2 (en) * 2017-09-14 2020-07-22 Janssen BioPharma, Inc. Galnac derivatives
BR112021008539A2 (pt) * 2018-11-08 2021-08-03 Aligos Therapeutics, Inc. polímeros de oligonucleotídeos e métodos de inibição de transporte de antígeno s
US20230250125A1 (en) * 2020-04-01 2023-08-10 Janssen Biopharma, Inc. Nucleic acid polymers
US20220056451A1 (en) * 2020-07-27 2022-02-24 Aligos Therapeutics, Inc. Hbv binding oligonucleotides and methods of use
CN114057816B (zh) * 2020-07-30 2025-02-11 浙江柏拉阿图医药科技有限公司 富含腺嘌呤硫代磷酸酯化寡核苷酸及其抗肝炎病毒的应用
US20240073008A1 (en) 2021-01-12 2024-02-29 Nec Corporation Secure computation system, secure computation server apparatus, secure computation method, and secure computation program
WO2023131098A2 (en) * 2022-01-10 2023-07-13 Ausper Biopharma Co., Ltd. Modulation of hepatitis b virus (hbv) expression
TW202508607A (zh) * 2023-05-19 2025-03-01 大陸商北京炫景瑞醫藥科技有限公司 雙股寡核苷酸及其共軛物和用途
US20250345389A1 (en) * 2024-05-13 2025-11-13 Gilead Sciences, Inc. Combination therapies

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001018015A1 (en) * 1999-09-10 2001-03-15 Geron Corporation Oligonucleotide n3'→p5' thiophosphoramidates: their synthesis and use
WO2005047506A1 (en) * 2003-11-04 2005-05-26 Geron Corporation Rna amidates and thioamidates for rnai
WO2007022369A2 (en) * 2005-08-17 2007-02-22 Sirna Therapeutics, Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
WO2013003520A1 (en) * 2011-06-30 2013-01-03 Arrowhead Research Corporation Compositions and methods for inhibiting gene expression of hepatitis b virus
US20140121261A1 (en) * 2012-10-26 2014-05-01 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders
WO2016077321A1 (en) * 2014-11-10 2016-05-19 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) irna compositions and methods of use thereof

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0754242A4 (en) 1994-03-18 1997-06-11 Lynx Therapeutics Inc SYNTHESIS AND SUBSTANCE, HYBRIDIZATION AND NUCLEASE-RESISTANT PROPERTIES OF OLIGONUCLEOTID-N3'-m (7) P5'-PHOSPHORAMIDATE
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
TR200102328T2 (tr) 1999-02-12 2002-01-21 Sankyo Company Limited Yeni nükleosid ve oligonükleotid analogları
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
KR100782896B1 (ko) 1999-05-04 2007-12-06 엑시콘 에이/에스 Ｌ-리보-ｌｎａ 유사체
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
AU2001227965A1 (en) 2000-01-21 2001-07-31 Geron Corporation 2'-arabino-fluorooligonucleotide n3'-p5'phosphoramidates: their synthesis and use
US7563618B2 (en) * 2001-03-23 2009-07-21 Geron Corporation Oligonucleotide conjugates
EP1383782A1 (en) 2001-03-26 2004-01-28 Sirna Therpeutics, Inc. Oligonucleotide mediated inhibition of hepatitis b virus and hepatitis c virus replication
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US7956176B2 (en) * 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20050026198A1 (en) * 2003-06-27 2005-02-03 Tamara Balac Sipes Method of selecting an active oligonucleotide predictive model
US8198253B2 (en) * 2006-07-19 2012-06-12 Isis Pharmaceuticals, Inc. Compositions and their uses directed to HBXIP
EP2512491B1 (en) * 2009-10-16 2017-10-11 Glaxo Group Limited Hbv antisense inhibitors
WO2011060557A1 (en) 2009-11-20 2011-05-26 Governors Of The University Of Alberta Method for selection of aptamers
US20130156845A1 (en) * 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
SG10201604074TA (en) * 2011-04-21 2016-07-28 Isis Pharmaceuticals Inc Modulation of hepatitis b virus (hbv) expression
WO2013159109A1 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Modulation of hepatitis b virus (hbv) expression
CN104736551B (zh) * 2012-08-15 2017-07-28 Ionis制药公司 使用改进的封端方案制备寡聚化合物的方法
US10059940B2 (en) * 2015-01-27 2018-08-28 Minghong Zhong Chemically ligated RNAs for CRISPR/Cas9-lgRNA complexes as antiviral therapeutic agents
WO2018053185A1 (en) 2016-09-14 2018-03-22 Alios Biopharma, Inc. Modified oligonucleotides and methods of use

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001018015A1 (en) * 1999-09-10 2001-03-15 Geron Corporation Oligonucleotide n3'→p5' thiophosphoramidates: their synthesis and use
WO2005047506A1 (en) * 2003-11-04 2005-05-26 Geron Corporation Rna amidates and thioamidates for rnai
WO2007022369A2 (en) * 2005-08-17 2007-02-22 Sirna Therapeutics, Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
WO2013003520A1 (en) * 2011-06-30 2013-01-03 Arrowhead Research Corporation Compositions and methods for inhibiting gene expression of hepatitis b virus
US20140121261A1 (en) * 2012-10-26 2014-05-01 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders
WO2016077321A1 (en) * 2014-11-10 2016-05-19 Alnylam Pharmaceuticals, Inc. Hepatitis b virus (hbv) irna compositions and methods of use thereof

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
GRYAZNOV S M,, OLIGONUCLEOTIDE N3'-> P5' PHOSPHORAMIDATES AS POTENTIAL THERAPEUTIC AGENTS, 10 December 1999 (1999-12-10) *
MATRAY T J GRYAZNOV S M,, SYNTHESIS AND PROPERTIES OF RNA ANALOGS - OLIGORIBONUCLEOTIDES N3' - P5' PHOSPHORAMIDITES, 31 December 1999 (1999-12-31) *
NARINDER S BANAIT ET AL,, DNA AND RNA ANALOGUES - OLIGONUCLEOTIDE PHOSPHORAMIDATES WITH BRIDGING NITROGEN, 1 April 2002 (2002-04-01) *
OBIKA S ET AL,, SYNTHESIS AND PROPERTIES OF 3'-AMINO-2',4'-BNA, A BRIDGED NUCLEIC ACID WITH A N3'-> P5' PHOSPHORAMIDATE LINKAGE, 15 October 2008 (2008-10-15) *

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JP2023011793A (ja) 2023-01-24
US20180135054A1 (en) 2018-05-17
TW201825103A (zh) 2018-07-16
RU2019111020A (ru) 2020-10-15
MA46422A (fr) 2019-07-24
CA3037042A1 (en) 2018-03-22
US10793859B2 (en) 2020-10-06
US12077757B2 (en) 2024-09-03
US20250092400A1 (en) 2025-03-20
TWI769177B (zh) 2022-07-01
IL315071A (en) 2024-10-01
BR112019004911A2 (pt) 2019-06-25
JP2019529406A (ja) 2019-10-17
AU2017326372B2 (en) 2023-12-07
CN109952378A (zh) 2019-06-28
JP7170820B2 (ja) 2022-11-14
WO2018053185A1 (en) 2018-03-22
KR20240010761A (ko) 2024-01-24
KR20190047025A (ko) 2019-05-07
KR102533038B1 (ko) 2023-05-17
AU2017326372A1 (en) 2019-04-04
IL265359A (en) 2019-05-30
US20210040483A1 (en) 2021-02-11
IL265359B1 (en) 2024-10-01
CN109952378B (zh) 2024-09-17
JP6970187B2 (ja) 2021-11-24
JP2022023936A (ja) 2022-02-08
JP7478210B2 (ja) 2024-05-02
EP3512949A1 (en) 2019-07-24
MX2023014145A (es) 2023-12-13
RU2019111020A3 (enExample) 2021-06-22
MX2019002960A (es) 2019-09-18
AU2024201485A1 (en) 2024-03-28
KR102627418B1 (ko) 2024-01-19
KR20230033736A (ko) 2023-03-08

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