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PE20250400A1 - Composiciones y metodos para inhibir cetohexoquinasa - Google Patents

Composiciones y metodos para inhibir cetohexoquinasa

Info

Publication number
PE20250400A1
PE20250400A1 PE2023002847A PE2023002847A PE20250400A1 PE 20250400 A1 PE20250400 A1 PE 20250400A1 PE 2023002847 A PE2023002847 A PE 2023002847A PE 2023002847 A PE2023002847 A PE 2023002847A PE 20250400 A1 PE20250400 A1 PE 20250400A1
Authority
PE
Peru
Prior art keywords
ketohexokinase
compositions
methods
pharmaceutically acceptable
acceptable salt
Prior art date
Application number
PE2023002847A
Other languages
English (en)
Inventor
Bod Dale Brown
Henryk T Dudek
Utsav Saxena
Jihye Park
Marc Abrams
Martin Lee Koser
Original Assignee
Dicerna Pharmaceuticals Inc
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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of PE20250400A1 publication Critical patent/PE20250400A1/es

Links

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    • 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
    • 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
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (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)

Abstract

La presente invencion proporciona un oligonucleotido de ARNi bicatenario o una sal de este aceptable desde el punto de vista farmaceutico, para reducir la expresion de la cetohexoquinasa (KHK) que es una enzima clave en el metabolismo de la fructosa. Este oligonucleotido comprende una cadena antisentido y una cadena de sentido que forman una region duplex, en donde la cadena antisentido comprende una region de complementariedad de al menos 15 nucleotidos contiguos de longitud con respecto a una secuencia diana de ARNm de KHK. La invencion tambien reivindica una composicion farmaceutica que comprende el oligonucleotido de ARNi o una sal de este aceptable desde el punto de vista farmaceutico, y al menos un portador, entre otros.
PE2023002847A 2021-04-12 2022-04-11 Composiciones y metodos para inhibir cetohexoquinasa PE20250400A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163173775P 2021-04-12 2021-04-12
US202163182277P 2021-04-30 2021-04-30
EP21196784 2021-09-15
PCT/EP2022/059663 WO2022218941A2 (en) 2021-04-12 2022-04-11 Compositions and methods for inhibiting ketohexokinase (khk)

Publications (1)

Publication Number Publication Date
PE20250400A1 true PE20250400A1 (es) 2025-02-11

Family

ID=81448852

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2023002847A PE20250400A1 (es) 2021-04-12 2022-04-11 Composiciones y metodos para inhibir cetohexoquinasa

Country Status (19)

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

Families Citing this family (7)

* 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
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
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
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
CN118546932A (zh) * 2023-06-20 2024-08-27 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

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Also Published As

Publication number Publication date
BR112023017367A2 (pt) 2023-12-12
CO2023013465A2 (es) 2023-10-30
CA3214439A1 (en) 2022-10-20
CN117120612A (zh) 2023-11-24
ECSP23076906A (es) 2023-11-30
CL2023003017A1 (es) 2024-05-03
IL307315A (en) 2023-11-01
DOP2023000223A (es) 2023-11-15
WO2022218941A2 (en) 2022-10-20
JP2024516356A (ja) 2024-04-15
EP4323518A2 (en) 2024-02-21
TW202305135A (zh) 2023-02-01
US20220340909A1 (en) 2022-10-27
MX2023012048A (es) 2023-10-23
WO2022218941A3 (en) 2023-02-23
AU2022256742A1 (en) 2023-09-07
KR20230170732A (ko) 2023-12-19
US20250092403A1 (en) 2025-03-20
PH12023552830A1 (en) 2024-05-20
CR20230482A (es) 2023-12-07

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