KR20240139057A - 프리칼리크레인(pkk) 단백질의 발현을 억제하기 위한 조성물 및 방법 - Google Patents

프리칼리크레인(pkk) 단백질의 발현을 억제하기 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20240139057A
KR20240139057A KR1020247025383A KR20247025383A KR20240139057A KR 20240139057 A KR20240139057 A KR 20240139057A KR 1020247025383 A KR1020247025383 A KR 1020247025383A KR 20247025383 A KR20247025383 A KR 20247025383A KR 20240139057 A KR20240139057 A KR 20240139057A
Authority
KR
South Korea
Prior art keywords
pkk
subject
dsrna
nucleotides
paragraph
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
KR1020247025383A
Other languages
English (en)
Korean (ko)
Inventor
동쑤 슈
펑청 패트릭 샤오
시웨이 씨아
Original Assignee
상하이 아르고 바이오파마슈티칼 씨오., 엘티디.
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 상하이 아르고 바이오파마슈티칼 씨오., 엘티디. filed Critical 상하이 아르고 바이오파마슈티칼 씨오., 엘티디.
Publication of KR20240139057A publication Critical patent/KR20240139057A/ko
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21035—Tissue kallikrein (3.4.21.35)
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21008—Kallikrein (3.4.21.8)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21034—Plasma kallikrein (3.4.21.34)
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
KR1020247025383A 2022-01-28 2023-01-28 프리칼리크레인(pkk) 단백질의 발현을 억제하기 위한 조성물 및 방법 Pending KR20240139057A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN2022074535 2022-01-28
CNPCT/CN2022/074535 2022-01-28
PCT/CN2023/073501 WO2023143483A1 (en) 2022-01-28 2023-01-28 Compositions and methods for inhibiting expression of prekallikrein (pkk) protein

Publications (1)

Publication Number Publication Date
KR20240139057A true KR20240139057A (ko) 2024-09-20

Family

ID=87470605

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247025383A Pending KR20240139057A (ko) 2022-01-28 2023-01-28 프리칼리크레인(pkk) 단백질의 발현을 억제하기 위한 조성물 및 방법

Country Status (11)

Country Link
US (1) US20250145999A1 (https=)
EP (1) EP4469570A1 (https=)
JP (1) JP2025508324A (https=)
KR (1) KR20240139057A (https=)
CN (1) CN118510894A (https=)
AU (1) AU2023210979A1 (https=)
CA (1) CA3244243A1 (https=)
IL (1) IL314396A (https=)
MX (1) MX2024009257A (https=)
TW (1) TW202346586A (https=)
WO (1) WO2023143483A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12042509B2 (en) * 2021-10-01 2024-07-23 Adarx Pharmaceuticals, Inc. Prekallikrein-modulating compositions and methods of use thereof

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102365486B1 (ko) * 2013-08-28 2022-02-18 아이오니스 파마수티컬즈, 인코포레이티드 프리칼리크레인 (pkk) 발현의 조절
HUE052709T2 (hu) * 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
JP6695902B2 (ja) * 2015-05-06 2020-05-20 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 第ＸＩＩ因子（ハーゲマン因子）（Ｆ１２）、カリクレインＢ、血漿（フレッチャー因子）１（ＫＬＫＢ１）、及びキニノーゲン１（ＫＮＧ１）ｉＲＮＡ組成物及びその使用方法
CN109762904A (zh) * 2019-03-05 2019-05-17 中国医学科学院北京协和医院 与胰腺神经内分泌肿瘤相关的分子标记物及其应用
US20220315929A1 (en) * 2019-05-24 2022-10-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof
WO2021126734A1 (en) * 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
BR112022015380A2 (pt) * 2020-02-07 2022-09-27 Intellia Therapeutics Inc Composições e métodos para edição de gene de calicreína (klkb1)

Also Published As

Publication number Publication date
TW202346586A (zh) 2023-12-01
CA3244243A1 (en) 2023-08-03
IL314396A (en) 2024-09-01
MX2024009257A (es) 2024-08-06
CN118510894A (zh) 2024-08-16
US20250145999A1 (en) 2025-05-08
JP2025508324A (ja) 2025-03-26
EP4469570A1 (en) 2024-12-04
WO2023143483A1 (en) 2023-08-03
AU2023210979A1 (en) 2024-08-01

Similar Documents

Publication Publication Date Title
JP7833044B2 (ja) タンパク質ＬＰＡ（Ａｐｏ（ａ））の発現を阻害するための組成物及び方法
WO2023045994A1 (en) Compositions and methods for inhibiting expression of angiopoietin-like 3 (angptl3) protein
JP7829035B2 (ja) アンギオテンシノゲン（ａｇｔ）タンパク質の発現を阻害する組成物及び方法
EP4442827A1 (en) Composition and method for inhibiting expression of hepatitis b virus (hbv) protein
WO2023143483A1 (en) Compositions and methods for inhibiting expression of prekallikrein (pkk) protein
AU2024276138A1 (en) Compositions and methods for inhibiting expression of coagulation factor xi (fxi)
RU2854980C2 (ru) Композиции и способы ингибирования экспрессии ангиопоэтин-подобного белка 3 (angptl3)
WO2025113470A1 (en) Compositions and methods for inhibiting expression of transthyretin (ttr)
OA21722A (en) Compositions and methods for inhibiting expression of Prekallikrein (PKK) protein.
WO2025077711A1 (en) Compositions and methods for inhibiting expression of amyloid precursor protein (app)
AU2024359927A1 (en) Compositions and methods for inhibiting expression of amyloid precursor protein (app)
HK40115058A (en) Composition and method for inhibiting angiotensinogen (agt) protein expression
WO2025148896A1 (en) Compositions and methods for inhibiting expression of transmembrane serine protease 6 (tmprss6)
HK40117932A (en) Composition and method for inhibiting expression of hepatitis b virus (hbv) protein
HK40109544A (zh) 用於抑制前激肽释放酶(pkk)蛋白表达的组合物和方法
CN122003498A (zh) 抑制淀粉样前体蛋白(app)表达的组合物和方法

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20240726

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application