AR124229A1 - Oligonucleótidos antisentido que actúan sobre atxn3 - Google Patents

Oligonucleótidos antisentido que actúan sobre atxn3

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Publication number
AR124229A1
AR124229A1 ARP210103344A ARP210103344A AR124229A1 AR 124229 A1 AR124229 A1 AR 124229A1 AR P210103344 A ARP210103344 A AR P210103344A AR P210103344 A ARP210103344 A AR P210103344A AR 124229 A1 AR124229 A1 AR 124229A1
Authority
AR
Argentina
Prior art keywords
oligonucleotide
atxn3
conjugate
pharmaceutically acceptable
salt
Prior art date
Application number
ARP210103344A
Other languages
English (en)
Inventor
Heidi Rye Hudlebusch
Erik Funder
Lukasz Kielpinski
Christoffer Sondergaard
Alexander Herbert Stephan
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of AR124229A1 publication Critical patent/AR124229A1/es

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    • CCHEMISTRY; METALLURGY
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    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/314Phosphoramidates
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
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    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
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    • C12N2310/33415-Methylcytosine
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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

Abstract

Reivindicación 1: Un oligonucleótido antisentido seleccionado del grupo que consiste en los compuestos ID Nº 1122_107, 1122_156, 1122_91, 1122_154, 1122_155, 1122_157, 1122_158, 1122_167, 1122_172, 1122_175, 1122_294, 1122_296, 1816_13, 1816_15, 1816_28, 1816_41, 1816_42, 1816_43, 1816_60, 1816_61, 1816_64, 1816_65 y 1816_68, o una sal de estos aceptable desde el punto de vista farmacéutico. Reivindicación 26: Un conjugado que comprende el oligonucleótido de acuerdo con cualquiera de las reivindicaciones 1 - 25, y al menos una porción de conjugado unida de manera covalente a dicho oligonucleótido; o una sal de este aceptable desde el punto de vista farmacéutico. Reivindicación 27: Una composición farmacéutica que comprende el oligonucleótido de acuerdo con las reivindicaciones 1 - 25 o el conjugado de la reivindicación 26 y un diluyente, solvente, portador, sal y/o adyuvante aceptables desde el punto de vista farmacéutico. Reivindicación 28: Un método in vivo o in vitro para modular la expresión de ATXN3 en una célula diana que expresa ATXN3, en donde dicho método comprende administrar a dicha célula un oligonucleótido o sal de acuerdo con cualquiera de las reivindicaciones 1 - 25, el conjugado de acuerdo con la reivindicación 26 o la composición farmacéutica de acuerdo con la reivindicación 27 en una cantidad eficaz.
ARP210103344A 2020-12-03 2021-12-02 Oligonucleótidos antisentido que actúan sobre atxn3 AR124229A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP20211618 2020-12-03

Publications (1)

Publication Number Publication Date
AR124229A1 true AR124229A1 (es) 2023-03-01

Family

ID=73698678

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210103344A AR124229A1 (es) 2020-12-03 2021-12-02 Oligonucleótidos antisentido que actúan sobre atxn3

Country Status (4)

Country Link
US (1) US20220177883A1 (es)
AR (1) AR124229A1 (es)
TW (1) TW202237842A (es)
WO (1) WO2022117745A1 (es)

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Also Published As

Publication number Publication date
US20220177883A1 (en) 2022-06-09
TW202237842A (zh) 2022-10-01
WO2022117745A1 (en) 2022-06-09

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