CA3129261A1 - Rnai agents for hepatitis b virus infection - Google Patents

Rnai agents for hepatitis b virus infection Download PDF

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Publication number
CA3129261A1
CA3129261A1 CA3129261A CA3129261A CA3129261A1 CA 3129261 A1 CA3129261 A1 CA 3129261A1 CA 3129261 A CA3129261 A CA 3129261A CA 3129261 A CA3129261 A CA 3129261A CA 3129261 A1 CA3129261 A1 CA 3129261A1
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CA
Canada
Prior art keywords
seq
administered
sequence
hbv
amount
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3129261A
Other languages
English (en)
French (fr)
Inventor
Bruce D. Given
James C. Hamilton
Thomas Schluep
Maria Gloria Beumont
Oliver Lenz
Ronald Cornelis Marie Kalmeijer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
GlaxoSmithKline Intellectual Property No 3 Ltd
Arrowhead Pharmaceuticals Inc
Original Assignee
Janssen Pharmaceuticals Inc
Arrowhead Pharmaceuticals 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 Janssen Pharmaceuticals Inc, Arrowhead Pharmaceuticals Inc filed Critical Janssen Pharmaceuticals Inc
Publication of CA3129261A1 publication Critical patent/CA3129261A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K31/66Phosphorus compounds
    • A61K31/675Phosphorus compounds having nitrogen as a ring hetero atom, e.g. pyridoxal phosphate
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K47/50Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0021Intradermal administration, e.g. through microneedle arrays or needleless injectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
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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
    • 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
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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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    • 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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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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  • Health & Medical Sciences (AREA)
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  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CA3129261A 2019-02-07 2020-02-07 Rnai agents for hepatitis b virus infection Pending CA3129261A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962802614P 2019-02-07 2019-02-07
US62/802,614 2019-02-07
US201962853659P 2019-05-28 2019-05-28
US62/853,659 2019-05-28
US201962932315P 2019-11-07 2019-11-07
US62/932,315 2019-11-07
PCT/US2020/017264 WO2020163747A1 (en) 2019-02-07 2020-02-07 Rnai agents for hepatitis b virus infection

Publications (1)

Publication Number Publication Date
CA3129261A1 true CA3129261A1 (en) 2020-08-13

Family

ID=69771180

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3129261A Pending CA3129261A1 (en) 2019-02-07 2020-02-07 Rnai agents for hepatitis b virus infection

Country Status (15)

Country Link
US (1) US20210395745A1 (https=)
EP (2) EP3920884B1 (https=)
JP (2) JP7585214B2 (https=)
KR (1) KR20210130736A (https=)
CN (1) CN113507920A (https=)
AU (1) AU2020219355A1 (https=)
BR (1) BR112021015361A2 (https=)
CA (1) CA3129261A1 (https=)
IL (1) IL285257A (https=)
JO (1) JOP20210208A1 (https=)
MX (1) MX2021009495A (https=)
PH (1) PH12021551866A1 (https=)
SG (1) SG11202108395SA (https=)
TW (1) TWI872048B (https=)
WO (1) WO2020163747A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016306275A1 (en) 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
JOP20190015A1 (ar) 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
US11021692B2 (en) 2017-12-19 2021-06-01 Janssen Sciences Ireland Unlimited Company Hepatitis B virus (HBV) vaccines and uses thereof
WO2020163747A1 (en) * 2019-02-07 2020-08-13 Arrowhead Pharmaceuticals, Inc. Rnai agents for hepatitis b virus infection
EP4168547A1 (en) * 2020-06-22 2023-04-26 Janssen Pharmaceuticals, Inc. Compositions and methods for treatment of hepatitis d virus infection
WO2022078479A1 (en) * 2020-10-16 2022-04-21 Janssen Pharmaceuticals, Inc. Combination therapy for treating hepatitis b virus infection
US20240124879A1 (en) * 2020-12-18 2024-04-18 Olix Pharmaceuticals, Inc. Rnai agent for inhibiting hbv expression and use thereof
TW202245809A (zh) * 2020-12-18 2022-12-01 美商詹森藥物公司 用於治療b型肝炎病毒感染之組合療法
WO2022152869A1 (en) * 2021-01-15 2022-07-21 Janssen Sciences Ireland Unlimited Company Use of oligonucleotides for individuals with hepatic impairment
JP2024527380A (ja) * 2021-07-09 2024-07-24 グラクソスミスクライン、インテレクチュアル、プロパティー、(ナンバー3)、リミテッド 腎機能障害を有する個体へのオリゴヌクレオチドの使用
CA3235672A1 (en) * 2021-11-29 2023-06-01 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of hepatitis b virus (hbv) protein
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
KR20260008676A (ko) * 2024-07-08 2026-01-16 항저우 티엔롱 파마슈티칼 컴퍼니 리미티드 HBV 유전자 발현을 표적하고 조절하기 위한 이중 가닥 RNAi 제제 및 그 응용

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US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EA024762B9 (ru) 2011-06-30 2017-04-28 Эрроухэд Рисерч Корпорейшн Композиции и способы ингибирования экспрессии генов вируса гепатита в
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
AU2016306275A1 (en) * 2015-08-07 2018-02-08 Arrowhead Pharmaceuticals, Inc. RNAi therapy for Hepatitis B virus infection
JOP20190015A1 (ar) * 2016-08-04 2019-02-04 Arrowhead Pharmaceuticals Inc عوامل (ار ان ايه آي) لعدوى فيروس الالتهاب الكبدي الوبائي ب
WO2020163747A1 (en) 2019-02-07 2020-08-13 Arrowhead Pharmaceuticals, Inc. Rnai agents for hepatitis b virus infection

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Publication number Publication date
IL285257A (en) 2021-09-30
JP2022521155A (ja) 2022-04-06
EP3920884B1 (en) 2026-04-01
TW202045723A (zh) 2020-12-16
WO2020163747A1 (en) 2020-08-13
US20210395745A1 (en) 2021-12-23
MX2021009495A (es) 2021-09-08
SG11202108395SA (en) 2021-08-30
EP3920884A1 (en) 2021-12-15
CN113507920A (zh) 2021-10-15
EP4707394A3 (en) 2026-04-29
JP7763907B2 (ja) 2025-11-04
TWI872048B (zh) 2025-02-11
BR112021015361A2 (pt) 2021-10-05
AU2020219355A9 (en) 2021-10-14
KR20210130736A (ko) 2021-11-01
EP4707394A2 (en) 2026-03-11
JOP20210208A1 (ar) 2023-01-30
JP7585214B2 (ja) 2024-11-18
AU2020219355A1 (en) 2021-09-30
JP2024153797A (ja) 2024-10-29
PH12021551866A1 (en) 2022-05-16

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