JP2025511854A - 非アルコール性脂肪肝疾患の治療 - Google Patents

非アルコール性脂肪肝疾患の治療 Download PDF

Info

Publication number
JP2025511854A
JP2025511854A JP2024559398A JP2024559398A JP2025511854A JP 2025511854 A JP2025511854 A JP 2025511854A JP 2024559398 A JP2024559398 A JP 2024559398A JP 2024559398 A JP2024559398 A JP 2024559398A JP 2025511854 A JP2025511854 A JP 2025511854A
Authority
JP
Japan
Prior art keywords
compound
human subject
dose
methyl
nucleotide sequence
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
JP2024559398A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2023196941A5 (https=
JP2025511854A5 (https=
Inventor
ジェイムズ、シー、ハミルトン
ブルース、ディー、ギブン
Original Assignee
アローヘッド ファーマシューティカルズ インコーポレイテッド
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 アローヘッド ファーマシューティカルズ インコーポレイテッド filed Critical アローヘッド ファーマシューティカルズ インコーポレイテッド
Publication of JP2025511854A publication Critical patent/JP2025511854A/ja
Publication of JPWO2023196941A5 publication Critical patent/JPWO2023196941A5/ja
Publication of JP2025511854A5 publication Critical patent/JP2025511854A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • A61P3/06—Antihyperlipidemics
    • 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
    • C12N15/1137—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 enzymes
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Plant Substances (AREA)
JP2024559398A 2022-04-08 2023-04-07 非アルコール性脂肪肝疾患の治療 Pending JP2025511854A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263329081P 2022-04-08 2022-04-08
US63/329,081 2022-04-08
US202263401858P 2022-08-29 2022-08-29
US63/401,858 2022-08-29
PCT/US2023/065487 WO2023196941A1 (en) 2022-04-08 2023-04-07 Treatment of a non-alcoholic fatty liver disease

Publications (3)

Publication Number Publication Date
JP2025511854A true JP2025511854A (ja) 2025-04-16
JPWO2023196941A5 JPWO2023196941A5 (https=) 2026-04-09
JP2025511854A5 JP2025511854A5 (https=) 2026-04-09

Family

ID=86286479

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024559398A Pending JP2025511854A (ja) 2022-04-08 2023-04-07 非アルコール性脂肪肝疾患の治療

Country Status (6)

Country Link
US (1) US20250283089A1 (https=)
EP (1) EP4504337A1 (https=)
JP (1) JP2025511854A (https=)
CN (1) CN119789859A (https=)
CA (1) CA3247602A1 (https=)
WO (1) WO2023196941A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025074157A1 (en) * 2023-10-06 2025-04-10 Glaxosmithkline Intellectual Property (No.3) Limited Treatment of a non-alcoholic fatty liver disease
WO2025162271A1 (zh) * 2024-02-03 2025-08-07 北京福元医药股份有限公司 用于抑制HSD17B13表达的siRNA、其缀合物和药物组合物及其用途

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102515329B1 (ko) 2016-03-07 2023-03-29 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
MX2018009853A (es) 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
MX2020001912A (es) * 2017-09-14 2020-03-24 Arrowhead Pharmaceuticals Inc Agentes de iarn y composiciones para inhibir la expresion de la angiopoyetina tipo 3 (angptl3) y metodos de uso.
MX2021003077A (es) 2018-09-19 2021-05-27 Arrowhead Pharmaceuticals Inc Agentes de arni para inhibir la expresion de 17beta-hsd tipo 13 (hsd17b13), composiciones de dichos agentes, y metodos de uso.
TW202202153A (zh) * 2020-03-26 2022-01-16 美商艾羅海德製藥公司 用於抑制ＰＮＰＬＡ３表現之ＲＮＡｉ劑、其醫藥組成物及使用方法

Also Published As

Publication number Publication date
US20250283089A1 (en) 2025-09-11
EP4504337A1 (en) 2025-02-12
WO2023196941A1 (en) 2023-10-12
CN119789859A (zh) 2025-04-08
CA3247602A1 (en) 2023-10-12

Similar Documents

Publication Publication Date Title
JP7488254B2 (ja) １７β－ＨＳＤ１３型（ＨＳＤ１７Ｂ１３）の発現を阻害するためのＲＮＡｉ剤、その組成物、および使用方法
JP7697889B2 (ja) アルファ－１アンチトリプシン欠乏症（ａａｔｄ）の治療法
TW201922264A (zh) 用於抑制類血管生成素蛋白-3(ANGPTL3)表現的RNAi藥劑及組合物及使用方法
JP2023519246A (ja) ＰＮＰＬＡ３の発現を阻害するためのＲＮＡｉ薬、その医薬組成物、及び使用方法
JP7829478B2 (ja) Ａｐｏｃ３関連疾患および障害の処置のための方法
JP2025511854A (ja) 非アルコール性脂肪肝疾患の治療
JP7846095B2 (ja) Ｚ－ａａｔタンパク質のレベルを低下させる方法
CN120112639A (zh) Angptl3相关疾病和病症的治疗方法
WO2025103391A1 (zh) 用于抑制lpa的基因表达的sirna，含其的组合物及其用途
CN116670278A (zh) 用于降低z-aat蛋白水平的方法
RU2851259C1 (ru) Способы снижения уровней белка z-aat
RU2847618C2 (ru) Способы лечения дефицита альфа-1-антитрипсина (aatd)
WO2025074157A1 (en) Treatment of a non-alcoholic fatty liver disease
CN121969377A (zh) 非酒精性脂肪性肝病的治疗
TW202430635A (zh) 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法
HK40128585A (zh) Angptl3相关疾病和病症的治疗方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260401

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20260401