KR20240099296A - 매트릭스 메탈로프로테이나제 7 (MMP7)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 - Google Patents

매트릭스 메탈로프로테이나제 7 (MMP7)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 Download PDF

Info

Publication number
KR20240099296A
KR20240099296A KR1020247016352A KR20247016352A KR20240099296A KR 20240099296 A KR20240099296 A KR 20240099296A KR 1020247016352 A KR1020247016352 A KR 1020247016352A KR 20247016352 A KR20247016352 A KR 20247016352A KR 20240099296 A KR20240099296 A KR 20240099296A
Authority
KR
South Korea
Prior art keywords
mmp7
rnai agent
rnai
nucleotides
nucleotide
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
KR1020247016352A
Other languages
English (en)
Korean (ko)
Inventor
앤서니 니콜라스
타오 페이
에릭 더블유. 부쉬
팅팅 위안
Original Assignee
애로우헤드 파마슈티컬스 인코포레이티드
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 애로우헤드 파마슈티컬스 인코포레이티드 filed Critical 애로우헤드 파마슈티컬스 인코포레이티드
Publication of KR20240099296A publication Critical patent/KR20240099296A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1137Non-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
    • 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
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • A61K9/0078Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y304/00Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/24Metalloendopeptidases (3.4.24)
    • C12Y304/24023Matrilysin (3.4.24.23)
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3513Protein; Peptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/35Special therapeutic applications based on a specific dosage / administration regimen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pulmonology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Otolaryngology (AREA)
  • Dispersion Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
KR1020247016352A 2021-10-22 2022-10-21 매트릭스 메탈로프로테이나제 7 (MMP7)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법 Pending KR20240099296A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163270849P 2021-10-22 2021-10-22
US63/270,849 2021-10-22
US202263308289P 2022-02-09 2022-02-09
US63/308,289 2022-02-09
US202263345654P 2022-05-25 2022-05-25
US63/345,654 2022-05-25
PCT/US2022/078498 WO2023070082A2 (en) 2021-10-22 2022-10-21 Rnai agents for inhibiting expression of matrix metalloproteinase 7(mmp7), compositions thereof, and methods of use

Publications (1)

Publication Number Publication Date
KR20240099296A true KR20240099296A (ko) 2024-06-28

Family

ID=86059695

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020247016352A Pending KR20240099296A (ko) 2021-10-22 2022-10-21 매트릭스 메탈로프로테이나제 7 (MMP7)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법

Country Status (13)

Country Link
US (1) US20230265437A1 (https=)
EP (1) EP4419689A2 (https=)
JP (1) JP2024539193A (https=)
KR (1) KR20240099296A (https=)
AU (1) AU2022368927A1 (https=)
CA (1) CA3235224A1 (https=)
CL (1) CL2024001175A1 (https=)
CO (1) CO2024005110A2 (https=)
IL (1) IL312239A (https=)
MX (1) MX2024004912A (https=)
PE (1) PE20241185A1 (https=)
TW (1) TW202334416A (https=)
WO (1) WO2023070082A2 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024302964A1 (en) * 2023-06-13 2025-12-18 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting mmp7, sirna conjugate and medical use thereof
KR20260046154A (ko) 2023-07-28 2026-04-06 장춘 진사이언스 파마슈티컬 씨오., 엘티디. siRNA 및 이의 조성물
WO2025152967A1 (zh) * 2024-01-15 2025-07-24 武汉人福创新药物研发中心有限公司 靶向αvβ6的化合物及其用途
WO2025228156A1 (zh) * 2024-04-30 2025-11-06 北京炫景瑞医药科技有限公司 肾靶向的双链寡核苷酸缀合物及其用途

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030194704A1 (en) * 2002-04-03 2003-10-16 Penn Sharron Gaynor Human genome-derived single exon nucleic acid probes useful for gene expression analysis two
US20050118625A1 (en) * 2003-10-02 2005-06-02 Mounts William M. Nucleic acid arrays for detecting gene expression associated with human osteoarthritis and human proteases
WO2006092795A2 (en) * 2005-03-01 2006-09-08 Qbi Enterprises Ltd. Oligoribonucleotides and methods of use thereof for treatment of ocular scarring and other fibrotic conditions
JP7445594B2 (ja) * 2017-11-01 2024-03-07 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 インテグリンリガンドおよびその使用
US12372530B2 (en) * 2017-11-15 2025-07-29 Beth Israel Deaconess Medical Center, Inc. Markers for the diagnosis and treatment of non-alcoholic steatohepatitis (NASH) and advanced liver fibrosis
JP2023541422A (ja) * 2020-09-11 2023-10-02 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 インテグリン標的化リガンド及びその使用

Also Published As

Publication number Publication date
PE20241185A1 (es) 2024-06-03
CA3235224A1 (en) 2023-04-27
WO2023070082A2 (en) 2023-04-27
TW202334416A (zh) 2023-09-01
CO2024005110A2 (es) 2024-05-30
MX2024004912A (es) 2024-05-06
AU2022368927A1 (en) 2024-04-11
CL2024001175A1 (es) 2024-12-20
US20230265437A1 (en) 2023-08-24
WO2023070082A3 (en) 2023-07-06
IL312239A (en) 2024-06-01
JP2024539193A (ja) 2024-10-28
EP4419689A2 (en) 2024-08-28

Similar Documents

Publication Publication Date Title
KR102723833B1 (ko) 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
KR20240099296A (ko) 매트릭스 메탈로프로테이나제 7 (MMP7)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
US12486502B2 (en) RNAi agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use
JP7777522B2 (ja) ベータENaCの発現を阻害するRNAi剤、その組成物および使用方法
US20250034578A1 (en) Subcutaneous Delivery of RNAi Agents for Inhibiting Expression of Receptor for Advanced Glycation End-products
US20260043029A1 (en) RNAi Agents for Inhibiting Expression of Thymic Stromal Lymphopoietin (TSLP), Compositions Thereof, and Methods of Use
CN111212909A (zh) 用于抑制去唾液酸糖蛋白受体1的表达的RNAi试剂和组合物
KR20240014067A (ko) 뮤신 5AC (MUC5AC)의 발현을 억제하기 위한 RNAi 작용제, 그의 조성물, 및 사용 방법
CN117440817A (zh) 用于抑制晚期糖基化终末产物受体表达的RNAi剂、其组合物和使用方法
CN118541485A (zh) 用于抑制基质金属蛋白酶7(MMP7)表达的RNAi剂、其组合物和使用方法
OA21374A (en) RNAI agents for inhibiting expression of receptor for advanced glycation end-products, compositions thereof, and methods of use.
TW202440926A (zh) 用於抑制前蛋白轉化酶枯草桿菌蛋白酶KEXIN 9 (PCSK9)表現之RNAi 試劑及其醫藥組合物與使用方法
WO2025184301A1 (en) Rnai agents for dual inhibition of expression of apolipoprotein c-iii (apoc3) and proprotein convertase subtilisin kexin 9 (pcsk9), compositions thereof, and methods of use

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20240517

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application