MX2020000154A - Composiciones y métodos para inhibir la expresión de hmgb1. - Google Patents

Composiciones y métodos para inhibir la expresión de hmgb1.

Info

Publication number
MX2020000154A
MX2020000154A MX2020000154A MX2020000154A MX2020000154A MX 2020000154 A MX2020000154 A MX 2020000154A MX 2020000154 A MX2020000154 A MX 2020000154A MX 2020000154 A MX2020000154 A MX 2020000154A MX 2020000154 A MX2020000154 A MX 2020000154A
Authority
MX
Mexico
Prior art keywords
hmgb1 expression
compositions
methods
oligonucleotides
hepatocytes
Prior art date
Application number
MX2020000154A
Other languages
English (en)
Inventor
Marc Abrams
Girish Chopda
Jihye Park
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 MX2020000154A publication Critical patent/MX2020000154A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • C12N2310/11—Antisense
    • 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 N.A.
    • 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
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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
    • 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
    • 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
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Esta divulgación ser refiere a los oligonucleótidos, las composiciones y métodos útiles para reducir la expresión de HMGB1, particularmente en hepatocitos; los oligonucleótidos descritos para la reducción de la expresión HMGB1 pueden ser de doble cadena o cadena simple y pueden modificarse para las características mejoradas tales como mayor resistencia a nucleasas y menor inmunogenicidad; los oligonucleótidos descritos para la reducción de la expresión de HMGB1 también puede diseñarse para incluir ligandos dirigidos para dirigir una célula u órgano particular, tal como los hepatocitos del hígado y puede usarse para tratar la fibrosis hepática y afecciones relacionadas.
MX2020000154A 2017-06-29 2018-06-29 Composiciones y métodos para inhibir la expresión de hmgb1. MX2020000154A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762526971P 2017-06-29 2017-06-29
PCT/US2018/040410 WO2019006375A1 (en) 2017-06-29 2018-06-29 COMPOSITIONS AND METHODS FOR INHIBITING HMGB1 EXPRESSION

Publications (1)

Publication Number Publication Date
MX2020000154A true MX2020000154A (es) 2020-07-22

Family

ID=64735107

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020000154A MX2020000154A (es) 2017-06-29 2018-06-29 Composiciones y métodos para inhibir la expresión de hmgb1.

Country Status (10)

Country Link
US (2) US10675295B2 (es)
EP (1) EP3645013A4 (es)
JP (1) JP2020529197A (es)
KR (1) KR20200023427A (es)
CN (1) CN111050776A (es)
AU (1) AU2018294415A1 (es)
CA (1) CA3068630A1 (es)
IL (2) IL271680B (es)
MX (1) MX2020000154A (es)
WO (1) WO2019006375A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111050776A (zh) 2017-06-29 2020-04-21 迪克纳制药公司 用于抑制hmgb1表达的组合物和方法
CA3094008A1 (en) * 2018-04-13 2019-10-17 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides
MX2021007001A (es) * 2018-12-12 2021-10-13 Dicerna Pharmaceuticals Inc Moleculas inhibidoras de acido nucleico de cadena doble que contienen un tribucle.
MX2021007855A (es) * 2018-12-28 2021-10-26 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de hmgb1.
TW202300645A (zh) * 2021-04-14 2023-01-01 美商戴瑟納製藥股份有限公司 用於調節ｐｎｐｌａ３表現之組合物及方法
BR112023021614A2 (pt) 2021-04-19 2024-01-16 Dicerna Pharmaceuticals Inc Composições e métodos para inibir a expressão da subfamília 1 de receptores nucleares, grupo h, membro 3 (nr1h3)
WO2023027759A1 (en) * 2021-08-25 2023-03-02 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting αlpha-1 antitrypsin expression
WO2024061202A1 (zh) * 2022-09-20 2024-03-28 北京福元医药股份有限公司 用于抑制hmgb1基因表达的双链核糖核酸及其修饰物、缀合物和用途

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US20080311122A1 (en) 2005-11-28 2008-12-18 Medimmune, Llc Antagonists of Hmgb1 and/or Rage and Methods of Use Thereof
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US20110081362A1 (en) * 2008-01-31 2011-04-07 The Brigham And Women's Hospital, Inc. Treatment of cancer
ES2708944T3 (es) * 2008-09-22 2019-04-12 Dicerna Pharmaceuticals Inc Composiciones y métodos para la inhibición específica de la expresión de genes por DSRNA que tenga modificaciones
WO2011007876A1 (ja) 2009-07-16 2011-01-20 Ｎｅｃソフト株式会社 Ｈｍｇｂ１結合核酸分子およびその用途
US9186371B2 (en) 2010-09-17 2015-11-17 Japan Science And Technology Agency Inhibitor of HMGB protein-mediated immune response activation, and screening method
WO2014028494A1 (en) * 2012-08-13 2014-02-20 The Regents Of The University Of California Detecting and treating liver damage
JP6688292B2 (ja) * 2014-10-10 2020-04-28 ダイセルナ ファーマシューティカルズ， インコーポレイテッドＤｉｃｅｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 乳酸デヒドロゲナーゼ及びその薬剤の治療的阻害
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CN111050776A (zh) 2017-06-29 2020-04-21 迪克纳制药公司 用于抑制hmgb1表达的组合物和方法
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Also Published As

Publication number Publication date
CA3068630A1 (en) 2019-01-03
JP2020529197A (ja) 2020-10-08
US20190000870A1 (en) 2019-01-03
IL271680A (en) 2020-02-27
US11478501B2 (en) 2022-10-25
CN111050776A (zh) 2020-04-21
KR20200023427A (ko) 2020-03-04
US10675295B2 (en) 2020-06-09
AU2018294415A1 (en) 2020-01-16
EP3645013A4 (en) 2021-07-21
IL271680B (en) 2022-09-01
US20200297754A1 (en) 2020-09-24
EP3645013A1 (en) 2020-05-06
IL295086A (en) 2022-09-01
WO2019006375A1 (en) 2019-01-03

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