ECSP23076906A - Composiciones y métodos para inhibir cetohexoquinasa (khk) - Google Patents

Composiciones y métodos para inhibir cetohexoquinasa (khk)

Info

Publication number
ECSP23076906A
ECSP23076906A ECSENADI202376906A ECDI202376906A ECSP23076906A EC SP23076906 A ECSP23076906 A EC SP23076906A EC SENADI202376906 A ECSENADI202376906 A EC SENADI202376906A EC DI202376906 A ECDI202376906 A EC DI202376906A EC SP23076906 A ECSP23076906 A EC SP23076906A
Authority
EC
Ecuador
Prior art keywords
khk
compositions
methods
ketohexokinase
inhibiting
Prior art date
Application number
ECSENADI202376906A
Other languages
English (en)
Inventor
Marc Abrams
Henryk T Dudek
Jihye Park
Martin Lee Koser
Bob Dale Brown
Utsav Saxena
Original Assignee
Boehringer Ingelheim Int Gmbh [De/De]
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 Boehringer Ingelheim Int Gmbh [De/De] filed Critical Boehringer Ingelheim Int Gmbh [De/De]
Publication of ECSP23076906A publication Critical patent/ECSP23076906A/es

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
    • 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)
  • 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)

Abstract

En la presente se proporcionan oligonucleótidos para inhibir la expresión de KHK. También se proporcionan composiciones que los incluyen y usos de estos, en particular, usos relacionados con el tratamiento de enfermedades, trastornos y/o afecciones asociados con la expresión de KHK.
ECSENADI202376906A 2021-04-12 2023-10-11 Composiciones y métodos para inhibir cetohexoquinasa (khk) ECSP23076906A (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
ECSP23076906A true ECSP23076906A (es) 2023-11-30

Family

ID=81448852

Family Applications (1)

Application Number Title Priority Date Filing Date
ECSENADI202376906A ECSP23076906A (es) 2021-04-12 2023-10-11 Composiciones y métodos para inhibir cetohexoquinasa (khk)

Country Status (20)

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)
JO (1) JOP20230250A1 (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 (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

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
EP1253199A1 (en) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Abasic moieties containing enzymatic nucleic acids
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
CN100523215C (zh) 2000-12-01 2009-08-05 马普科技促进协会 介导rna干涉的小rna分子
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
WO2010033225A2 (en) 2008-09-22 2010-03-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for the specific inhibition of gene expression by dsrna possessing modifications
KR20110110776A (ko) 2008-12-18 2011-10-07 다이서나 파마수이티컬, 인크. 유전자 발현의 특이적 억제를 위한 연장된 다이서 기질 제제 및 방법
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
AU2012328680A1 (en) 2011-10-25 2014-05-01 Ionis Pharmaceuticals, Inc. Antisense modulation of GCCR expression
EP3105331B1 (en) * 2014-02-11 2021-06-23 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
US10351854B2 (en) * 2014-10-10 2019-07-16 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
EP3865576A1 (en) * 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
WO2018045317A1 (en) * 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
EP3684377B1 (en) * 2017-10-20 2022-12-07 Dicerna Pharmaceuticals, Inc. Methods for treating hepatitis b infection
JP2022500003A (ja) * 2018-09-18 2022-01-04 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. ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物およびその使用方法
US20220389430A1 (en) * 2019-10-02 2022-12-08 Dicema Pharmaceuticals, Inc. Chemical modifications of small interfering rna with minimal fluorine content
MX2022011009A (es) * 2020-03-06 2022-10-07 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de ketohexocinasa (khk) y metodos de uso de las mismas.
CA3211059A1 (en) * 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof

Also Published As

Publication number Publication date
US20220340909A1 (en) 2022-10-27
IL307315A (en) 2023-11-01
BR112023017367A2 (pt) 2023-12-12
TW202305135A (zh) 2023-02-01
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
AU2022256742A1 (en) 2023-09-07

Similar Documents

Publication Publication Date Title
DOP2023000223A (es) Composiciones y métodos para inhibir cetohexoquinasa (khk)
ECSP24035717A (es) Compuestos de quinolina como inhibidores de kras
CO2022010547A2 (es) Compuestos y usos de los mismos
ECSP22087539A (es) Inhibidores de kras tricíclicos fusionados
UY40021A (es) Inhibidores lisina acetiltransferasa 6a (kat6a) y usos de los mismos
MX2017011269A (es) Inhibidores de lisil oxidasa tipo 2 fluorinados y usos de los mismos.
MX2021009673A (es) Moduladores de ror-gamma.
MX2019002750A (es) Derivados de pirazolopiridina como moduladores de cinasa 1 progenitora hematopoyetica (hpk1) y usos de los mismos para tratamiento de cancer.
CL2025000629A1 (es) Inhibidores de parp1 de azetidina y pirrolidina y usos de estos
NI201600164A (es) Compuestos y composiciones como agonistas del receptor tipo toll 7
CO2019007409A2 (es) Derivados de la pirimidin enona tricíclica para la inhibición de rorγ y otros usos
MX2016014308A (es) Compuestos y composiciones como agonistas del receptor tipo toll 7.
CL2023000371A1 (es) Composiciones y métodos para inhibir la expresión de plp1.
MX2021004566A (es) Compuestos terapeuticos.
MX2022014924A (es) Moduladores de il-17a.
UY32937A (es) Compuestos y composiciones como moduladores de la actividad de gpr119
CO2022018636A2 (es) Moduladores de il-17a
MX2022013396A (es) Compuestos de tetrahidroisoquinolina como activadores de nrf2.
CL2023003363A1 (es) Composiciones y métodos para inhibir la expresión del componente reductor 1 de amidoxima de mitocondria (marc1)
MX2021000142A (es) Composiciones y metodos para el tratamiento de enfermedades o condiciones relacionadas con inflamasomas.
CO2024017422A2 (es) Composiciones y métodos para inhibir la expresión de serina proteasa transmembrana 6 (tmprss6)
CO2024017526A2 (es) Compuestos de degradación de wee1 y usos de los mismos
CO2026000941A2 (es) Composiciones y métodos para la expresión del receptor del ligando de muerte programada (pd-l1)
MX2022013398A (es) Compuestos de tetrahidroisoquinolina como activadores de nrf2.
ECSP23091984A (es) Compuestos y usos de estos