AU2018351649B2 - Methods for treating hepatitis B infection - Google Patents

Methods for treating hepatitis B infection Download PDF

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Publication number
AU2018351649B2
AU2018351649B2 AU2018351649A AU2018351649A AU2018351649B2 AU 2018351649 B2 AU2018351649 B2 AU 2018351649B2 AU 2018351649 A AU2018351649 A AU 2018351649A AU 2018351649 A AU2018351649 A AU 2018351649A AU 2018351649 B2 AU2018351649 B2 AU 2018351649B2
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Australia
Prior art keywords
oligonucleotide
nucleotides
hbv
positions
antisense strand
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AU2018351649A
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AU2018351649A1 (en
Inventor
Marc Abrams
Martin KOSER
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Dicerna Pharmaceuticals Inc
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Dicerna Pharmaceuticals Inc
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52Purines, e.g. adenine
    • A61K31/522Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
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    • A61K47/50Medicinal 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
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    • A61K47/59Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
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    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00Modified animals
    • A01K2207/05Animals modified by non-integrating nucleic acids, e.g. antisense, RNAi, morpholino, episomal vector, for non-therapeutic purpose
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0337Animal models for infectious diseases
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

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  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Inorganic Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Professional, Industrial, Or Sporting Protective Garments (AREA)
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AU2018351649A 2017-10-20 2018-10-19 Methods for treating hepatitis B infection Active AU2018351649B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762575358P 2017-10-20 2017-10-20
US62/575,358 2017-10-20
PCT/US2018/056801 WO2019079781A2 (en) 2017-10-20 2018-10-19 METHODS OF TREATING HEPATITIS B TYPE INFECTIONS

Publications (2)

Publication Number Publication Date
AU2018351649A1 AU2018351649A1 (en) 2020-04-23
AU2018351649B2 true AU2018351649B2 (en) 2025-04-24

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AU2018351649A Active AU2018351649B2 (en) 2017-10-20 2018-10-19 Methods for treating hepatitis B infection

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US (5) US10799524B2 (enExample)
EP (2) EP3684377B1 (enExample)
JP (2) JP7353276B2 (enExample)
KR (1) KR102783206B1 (enExample)
CN (2) CN118185936A (enExample)
AU (1) AU2018351649B2 (enExample)
BR (1) BR112020007476A2 (enExample)
CA (1) CA3078960A1 (enExample)
CL (1) CL2020001061A1 (enExample)
CO (1) CO2020005135A2 (enExample)
CR (1) CR20200163A (enExample)
DK (1) DK3684377T3 (enExample)
ES (1) ES2936863T3 (enExample)
FI (1) FI3684377T3 (enExample)
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IL (4) IL282881B2 (enExample)
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MA (1) MA50264B1 (enExample)
MX (1) MX2020004060A (enExample)
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PE (1) PE20220561A1 (enExample)
PH (1) PH12020550591A1 (enExample)
PL (1) PL3684377T3 (enExample)
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SI (1) SI3684377T1 (enExample)
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IL282881B2 (en) 2017-10-20 2024-10-01 Dicerna Pharmaceuticals Inc Methods for treating hepatitis b infection
AU2019300324A1 (en) 2018-07-13 2021-01-21 F. Hoffmann-La Roche Ag Oligonucleotides for modulating RTEL1 expression
AU2020358016A1 (en) * 2019-10-02 2022-04-21 Dicerna Pharmaceuticals, Inc. Chemical modifications of small interfering RNA with minimal fluorine content
EP4077671A1 (en) * 2019-12-19 2022-10-26 F. Hoffmann-La Roche AG Use of saraf inhibitors for treating hepatitis b virus infection
WO2021130266A1 (en) * 2019-12-24 2021-07-01 F. Hoffmann-La Roche Ag Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
EP4081217A1 (en) * 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
US20220056451A1 (en) * 2020-07-27 2022-02-24 Aligos Therapeutics, Inc. Hbv binding oligonucleotides and methods of use
CA3190794A1 (en) * 2020-08-05 2022-02-10 Soren Ottosen Oligonucleotide treatment of hepatitis b patients
WO2022131883A1 (ko) * 2020-12-18 2022-06-23 올릭스 주식회사 HBV 발현을 억제하는 RNAi 제제 및 이의 용도
CN114853829A (zh) * 2021-02-05 2022-08-05 上海拓界生物医药科技有限公司 一种核酸配体、其缀合物、制备方法及用途
CN114940991B (zh) * 2021-04-13 2023-02-03 厦门甘宝利生物医药有限公司 一种抑制乙型肝炎病毒基因表达的rna抑制剂及其应用
CA3213775A1 (en) 2021-04-19 2022-10-27 Utsav SAXENA Compositions and methods for inhibiting nuclear receptor subfamily 1 group h member 3 (nr1h3) expression
AU2022384619A1 (en) 2021-11-11 2024-04-11 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
US20240271141A1 (en) * 2021-11-29 2024-08-15 Shanghai Argo Biopharmaceutical Co., Ltd. Composition and method for inhibiting expression of hepatitis b virus (hbv)protein
EP4201947A1 (en) 2021-12-22 2023-06-28 F. Hoffmann-La Roche AG Process for the backbone deprotection of oligonucleotides containing a terminal alkyl phosphonate group
WO2023116764A1 (zh) * 2021-12-23 2023-06-29 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及其用途
US20240309383A1 (en) * 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
WO2025199466A1 (en) 2024-03-22 2025-09-25 Purdue Research Foundation Liver-specific asialoglycoprotein receptor targeting ligands, conjugates comprising same, and related compositions and methods of use
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use

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