TW202220675A - 降低ｚ－ａａｔ蛋白水準之方法 - Google Patents

降低ｚ－ａａｔ蛋白水準之方法 Download PDF

Info

Publication number
TW202220675A
TW202220675A TW110134359A TW110134359A TW202220675A TW 202220675 A TW202220675 A TW 202220675A TW 110134359 A TW110134359 A TW 110134359A TW 110134359 A TW110134359 A TW 110134359A TW 202220675 A TW202220675 A TW 202220675A
Authority
TW
Taiwan
Prior art keywords
aat
reduction
ads
dose
individual
Prior art date
Application number
TW110134359A
Other languages
English (en)
Chinese (zh)
Inventor
棠恩 克絲汀安森
詹姆士 Ｃ 漢彌頓
布魯斯 齊文
馬丁 賈維爾 聖
Original Assignee
美商愛羅海德製藥公司
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 美商愛羅海德製藥公司 filed Critical 美商愛羅海德製藥公司
Publication of TW202220675A publication Critical patent/TW202220675A/zh

Links

Images

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/34—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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
TW110134359A 2020-09-15 2021-09-15 降低ｚ－ａａｔ蛋白水準之方法 TW202220675A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063078658P 2020-09-15 2020-09-15
US63/078,658 2020-09-15
US202163180487P 2021-04-27 2021-04-27
US63/180,487 2021-04-27

Publications (1)

Publication Number Publication Date
TW202220675A true TW202220675A (zh) 2022-06-01

Family

ID=78080560

Family Applications (1)

Application Number Title Priority Date Filing Date
TW110134359A TW202220675A (zh) 2020-09-15 2021-09-15 降低ｚ－ａａｔ蛋白水準之方法

Country Status (11)

Country Link
US (1) US20230348905A1 (https=)
EP (1) EP4214316A2 (https=)
JP (1) JP7846095B2 (https=)
KR (1) KR20230067648A (https=)
AU (1) AU2021345026A1 (https=)
BR (1) BR112023004756A2 (https=)
CA (1) CA3192372A1 (https=)
CO (1) CO2023003958A2 (https=)
MX (1) MX2023003066A (https=)
TW (1) TW202220675A (https=)
WO (1) WO2022060721A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201620526A (zh) 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
WO2012178033A2 (en) * 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
US9458457B2 (en) 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
CA3011946A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
CN110268060B (zh) 2017-01-10 2024-07-26 箭头药业股份有限公司 α-1抗胰蛋白酶(AAT)RNAi物质、包含AAT RNAi物质的组合物和使用方法
MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).

Also Published As

Publication number Publication date
CA3192372A1 (en) 2022-03-24
BR112023004756A2 (pt) 2024-02-06
JP7846095B2 (ja) 2026-04-14
CO2023003958A2 (es) 2023-04-17
AU2021345026A9 (en) 2024-06-13
WO2022060721A3 (en) 2022-04-28
JP2023541170A (ja) 2023-09-28
AU2021345026A1 (en) 2023-05-11
EP4214316A2 (en) 2023-07-26
WO2022060721A2 (en) 2022-03-24
MX2023003066A (es) 2023-04-10
US20230348905A1 (en) 2023-11-02
KR20230067648A (ko) 2023-05-16

Similar Documents

Publication Publication Date Title
TWI823866B (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
TWI870358B (zh) 用於抑制17β-HSD第13型(HSD17B13)表現之RNAi藥劑、其組合物及使用方法
US12582668B2 (en) Methods for the treatment of alpha-1 antitrypsin deficiency (AATD)
JP2020503070A (ja) α−１アンチトリプシン（ＡＡＴ）ＲＮＡｉ剤、ＡＡＴ ＲＮＡｉ剤を含む組成物、及び使用方法。
JP2021522804A (ja) Ｆｘｉの発現を低下させるための化合物及び方法
JP7829478B2 (ja) Ａｐｏｃ３関連疾患および障害の処置のための方法
TW202220675A (zh) 降低ｚ－ａａｔ蛋白水準之方法
CN120112639A (zh) Angptl3相关疾病和病症的治疗方法
JP2025511854A (ja) 非アルコール性脂肪肝疾患の治療
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)
AU2020324974B2 (en) Methods for the treatment of APOC3-related diseases and disorders
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
HK40092078A (zh) 一种核酸、含有该核酸的药物组合物与sirna缀合物及制备方法和用途
CN121969377A (zh) 非酒精性脂肪性肝病的治疗
HK40092078B (zh) 一种核酸、含有该核酸的药物组合物与sirna缀合物及制备方法和用途
HK40075141A (en) Methods for the treatment of apoc3-related diseases and disorders