TW202305135A - 用於抑制己酮醣激酶(khk)之組合物及方法 - Google Patents

用於抑制己酮醣激酶(khk)之組合物及方法 Download PDF

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TW202305135A
TW202305135A TW111113704A TW111113704A TW202305135A TW 202305135 A TW202305135 A TW 202305135A TW 111113704 A TW111113704 A TW 111113704A TW 111113704 A TW111113704 A TW 111113704A TW 202305135 A TW202305135 A TW 202305135A
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Taiwan
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seq
nucleotides
khk
antisense strand
oligonucleotide
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TW111113704A
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English (en)
Chinese (zh)
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包柏 戴爾 布朗
亨立克 T 杜迪克
烏薩夫 薩克森納
智惠 朴
馬克 艾布蘭
馬丁 李 克瑟
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德商百靈佳殷格翰國際股份有限公司
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Publication of TW202305135A publication Critical patent/TW202305135A/zh

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    • 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
    • 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
    • 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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Detergent Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
TW111113704A 2021-04-12 2022-04-11 用於抑制己酮醣激酶(khk)之組合物及方法 TW202305135A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202163173775P 2021-04-12 2021-04-12
US63/173,775 2021-04-12
US202163182277P 2021-04-30 2021-04-30
US63/182,277 2021-04-30
EP21196784 2021-09-15
EP21196784.9 2021-09-15

Publications (1)

Publication Number Publication Date
TW202305135A true TW202305135A (zh) 2023-02-01

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US (2) US20220340909A1 (enExample)
EP (1) EP4323518A2 (enExample)
JP (1) JP2024516356A (enExample)
KR (1) KR20230170732A (enExample)
CN (1) CN117120612A (enExample)
AU (1) AU2022256742A1 (enExample)
BR (1) BR112023017367A2 (enExample)
CA (1) CA3214439A1 (enExample)
CL (1) CL2023003017A1 (enExample)
CO (1) CO2023013465A2 (enExample)
CR (1) CR20230482A (enExample)
DO (1) DOP2023000223A (enExample)
EC (1) ECSP23076906A (enExample)
IL (1) IL307315A (enExample)
JO (1) JOP20230250A1 (enExample)
MX (1) MX2023012048A (enExample)
PE (1) PE20250400A1 (enExample)
PH (1) PH12023552830A1 (enExample)
TW (1) TW202305135A (enExample)
WO (1) WO2022218941A2 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3211059A1 (en) * 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
TW202430191A (zh) 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療伴有晚期纖維化及/或硬化之nash的方法
TW202430192A (zh) 2022-10-11 2024-08-01 德商百靈佳殷格翰國際股份有限公司 用於治療nash之劑量方案
WO2024175062A1 (zh) * 2023-02-23 2024-08-29 大睿生物医药科技(上海)有限公司 调控KHK基因活性的dsRNA分子
WO2024260379A1 (zh) * 2023-06-20 2024-12-26 施能康医药科技(苏州)有限公司 靶向已酮糖激酶的核酸及其用途
CN118703500B (zh) * 2024-01-30 2025-06-17 大连医科大学 一种果糖激酶khk的小干扰和短发夹rna序列组合及其应用
WO2025184876A1 (zh) * 2024-03-07 2025-09-12 竣莅医药科技(无锡)有限公司 一种rna干扰触发分子与对应核酸干扰药物制剂及其用途
WO2026149467A1 (zh) * 2025-01-09 2026-07-16 云合智药(苏州)生物科技有限公司 用于抑制己酮糖激酶表达的RNAi剂及其应用

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US20220340909A1 (en) 2022-10-27
IL307315A (en) 2023-11-01
BR112023017367A2 (pt) 2023-12-12
CL2023003017A1 (es) 2024-05-03
DOP2023000223A (es) 2023-11-15
US20250092403A1 (en) 2025-03-20
JP2024516356A (ja) 2024-04-15
KR20230170732A (ko) 2023-12-19
MX2023012048A (es) 2023-10-23
WO2022218941A2 (en) 2022-10-20
PE20250400A1 (es) 2025-02-11
CR20230482A (es) 2023-12-07
WO2022218941A3 (en) 2023-02-23
PH12023552830A1 (en) 2024-05-20
JOP20230250A1 (ar) 2023-10-12
CN117120612A (zh) 2023-11-24
EP4323518A2 (en) 2024-02-21
CO2023013465A2 (es) 2023-10-30
CA3214439A1 (en) 2022-10-20
ECSP23076906A (es) 2023-11-30
AU2022256742A1 (en) 2023-09-07

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