CL2023002479A1 - Composiciones de arni de ketohexoquinasa (khk) y métodos de uso de éstas - Google Patents

Composiciones de arni de ketohexoquinasa (khk) y métodos de uso de éstas

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Publication number
CL2023002479A1
CL2023002479A1 CL2023002479A CL2023002479A CL2023002479A1 CL 2023002479 A1 CL2023002479 A1 CL 2023002479A1 CL 2023002479 A CL2023002479 A CL 2023002479A CL 2023002479 A CL2023002479 A CL 2023002479A CL 2023002479 A1 CL2023002479 A1 CL 2023002479A1
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CL
Chile
Prior art keywords
khk
methods
ketohexokinase
arni
compositions
Prior art date
Application number
CL2023002479A
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English (en)
Inventor
Tremblay Frederic
D Mcininch James
Castoreno Adam
K Schlegel Mark
Noetzli Leila
Original Assignee
Alnylam Pharmaceuticals Inc
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Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2023002479A1 publication Critical patent/CL2023002479A1/es

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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01—Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01003—Ketohexokinase (2.7.1.3)

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  • Health & Medical Sciences (AREA)
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  • Organic Chemistry (AREA)
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  • Virology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
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  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Urology & Nephrology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

La presente invención se refiere a agentes de ARNi, por ejemplo, agentes de ARNbc, dirigidos al gen ketohexoquinasa (KHK). La invención también se refiere a métodos de uso de dichos agentes de ARNi para inhibir la expresión de un gen KHK y a métodos de tratamiento o prevención de un trastorno asociado a KHK en un sujeto.
CL2023002479A 2021-02-26 2023-08-22 Composiciones de arni de ketohexoquinasa (khk) y métodos de uso de éstas CL2023002479A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163154005P 2021-02-26 2021-02-26
US202163223581P 2021-07-20 2021-07-20
US202163280668P 2021-11-18 2021-11-18

Publications (1)

Publication Number Publication Date
CL2023002479A1 true CL2023002479A1 (es) 2024-04-05

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CL2023002479A CL2023002479A1 (es) 2021-02-26 2023-08-22 Composiciones de arni de ketohexoquinasa (khk) y métodos de uso de éstas

Country Status (13)

Country Link
US (2) US11926832B2 (es)
EP (1) EP4298218A1 (es)
JP (1) JP2024515423A (es)
KR (1) KR20230150844A (es)
AU (1) AU2022226098A1 (es)
BR (1) BR112023016645A2 (es)
CA (1) CA3211059A1 (es)
CL (1) CL2023002479A1 (es)
CO (1) CO2023012562A2 (es)
IL (1) IL305153A (es)
MX (1) MX2023009704A (es)
TW (1) TW202302847A (es)
WO (1) WO2022182574A1 (es)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230150844A (ko) 2021-02-26 2023-10-31 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나아제(KHK) iRNA 조성물 및 이의 사용 방법
AU2022256742A1 (en) * 2021-04-12 2023-09-07 Boehringer Ingelheim International Gmbh Compositions and methods for inhibiting ketohexokinase (khk)
US20240139230A1 (en) 2022-10-11 2024-05-02 Boehringer Ingelheim International Gmbh Dosage regimen for the treatment of nash
WO2024079076A1 (en) 2022-10-11 2024-04-18 Boehringer Ingelheim International Gmbh Methods for the treatment of nash with advanced fibrosis and/or cirrhosis
CN116854754B (zh) * 2023-09-01 2023-12-12 北京悦康科创医药科技股份有限公司 一种含有核糖环或其衍生结构的GalNAc化合物及其寡核苷酸缀合物

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