KR20230067648A - Z-aat 단백질 수준을 감소시키기 위한 방법 - Google Patents

Z-aat 단백질 수준을 감소시키기 위한 방법 Download PDF

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Publication number
KR20230067648A
KR20230067648A KR1020237012414A KR20237012414A KR20230067648A KR 20230067648 A KR20230067648 A KR 20230067648A KR 1020237012414 A KR1020237012414 A KR 1020237012414A KR 20237012414 A KR20237012414 A KR 20237012414A KR 20230067648 A KR20230067648 A KR 20230067648A
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South Korea
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aat
subject
reduction
drug substance
liver
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Pending
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KR1020237012414A
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English (en)
Korean (ko)
Inventor
던 크리스찬슨
제임스 씨. 해밀턴
브루스 기븐
Original Assignee
애로우헤드 파마슈티컬스 인코포레이티드
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Publication of KR20230067648A publication Critical patent/KR20230067648A/ko
Pending legal-status Critical Current

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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (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)
KR1020237012414A 2020-09-15 2021-09-14 Z-aat 단백질 수준을 감소시키기 위한 방법 Pending KR20230067648A (ko)

Applications Claiming Priority (5)

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
PCT/US2021/050247 WO2022060721A2 (en) 2020-09-15 2021-09-14 Methods for the reduction of z-aat protein levels

Publications (1)

Publication Number Publication Date
KR20230067648A true KR20230067648A (ko) 2023-05-16

Family

ID=78080560

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237012414A Pending KR20230067648A (ko) 2020-09-15 2021-09-14 Z-aat 단백질 수준을 감소시키기 위한 방법

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
EP3366312A1 (en) * 2011-06-23 2018-08-29 Alnylam Pharmaceuticals, Inc. Serpina 1 sirnas: compositions of matter and methods of treatment
DK3444350T3 (da) * 2013-07-03 2022-02-07 Dicerna Pharmaceuticals Inc Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna
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.
BR112019014282A2 (pt) 2017-01-10 2020-03-03 Arrowhead Pharmaceuticals, Inc. Agentes de rnai de antitripsina (aat) alfa-1, composições incluindo agentes de rnai de aat, e métodos de uso
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
WO2022060721A3 (en) 2022-04-28
JP2023541170A (ja) 2023-09-28
TW202220675A (zh) 2022-06-01
WO2022060721A2 (en) 2022-03-24
CA3192372A1 (en) 2022-03-24
EP4214316A2 (en) 2023-07-26
CO2023003958A2 (es) 2023-04-17
AU2021345026A1 (en) 2023-05-11
AU2021345026A9 (en) 2024-06-13
US20230348905A1 (en) 2023-11-02
BR112023004756A2 (pt) 2024-02-06
JP7846095B2 (ja) 2026-04-14
MX2023003066A (es) 2023-04-10

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Patent event date: 20230412

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Patent event date: 20240912

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