CR20200626A - Oligonucleótidos para modular la expresión de scn9a - Google Patents

Oligonucleótidos para modular la expresión de scn9a

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Publication number
CR20200626A
CR20200626A CR20200626A CR20200626A CR20200626A CR 20200626 A CR20200626 A CR 20200626A CR 20200626 A CR20200626 A CR 20200626A CR 20200626 A CR20200626 A CR 20200626A CR 20200626 A CR20200626 A CR 20200626A
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CR
Costa Rica
Prior art keywords
oligonucleotides
sub
modulating
voltage
gated sodium
Prior art date
Application number
CR20200626A
Other languages
English (en)
Inventor
Søren Rasmussen
Christoffer Sondergaard
Gianluigi Lichinchi
Lykke Pedersen
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 CR20200626A publication Critical patent/CR20200626A/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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/10—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Pain & Pain Management (AREA)
  • Neurology (AREA)
  • Neurosurgery (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)

Abstract

<p>La presente invención se refiere a oligonucleótidos (oligómeros) que son complementarios a los ácidos nucleicos codificantes de los canales iónicos de sodio dependientes de voltaje, tal como SCN9A, que codifica el canal de sodio dependiente de voltaje Nav1.7. Los oligonucleótidos de la invención pueden inhibir la expresión de los canales iónicos de sodio dependientes de voltaje, tal como Nav1.7 y son útiles en la prevención o el tratamiento del dolor.</p>
CR20200626A 2018-06-22 2019-06-19 Oligonucleótidos para modular la expresión de scn9a CR20200626A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18179339 2018-06-22
EP18194982 2018-09-18
PCT/EP2019/066223 WO2019243430A1 (en) 2018-06-22 2019-06-19 Oligonucleotides for modulating scn9a expression

Publications (1)

Publication Number Publication Date
CR20200626A true CR20200626A (es) 2021-02-22

Family

ID=66867163

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20200626A CR20200626A (es) 2018-06-22 2019-06-19 Oligonucleótidos para modular la expresión de scn9a

Country Status (16)

Country Link
US (1) US12116576B2 (es)
EP (1) EP3810776A1 (es)
JP (2) JP2021527437A (es)
KR (1) KR20210027384A (es)
CN (1) CN112513273A (es)
AU (1) AU2019291050A1 (es)
BR (1) BR112020026169A2 (es)
CA (1) CA3103756A1 (es)
CL (1) CL2020003304A1 (es)
CR (1) CR20200626A (es)
IL (1) IL279024A (es)
MX (1) MX2020013930A (es)
PE (1) PE20210314A1 (es)
SG (1) SG11202012161RA (es)
WO (1) WO2019243430A1 (es)
ZA (1) ZA202007542B (es)

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SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2021123086A1 (en) 2019-12-20 2021-06-24 F. Hoffmann-La Roche Ag Enhanced oligonucleotides for inhibiting scn9a expression
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc opa1 antisense oligomers for the treatment of conditions and diseases
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WO2024002045A1 (en) * 2022-06-27 2024-01-04 Aoan Biosciences Oligonucleotide delivery agents, pharmaceutical compositions and methods using the same
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Also Published As

Publication number Publication date
SG11202012161RA (en) 2021-01-28
JP2024056820A (ja) 2024-04-23
KR20210027384A (ko) 2021-03-10
EP3810776A1 (en) 2021-04-28
US12116576B2 (en) 2024-10-15
ZA202007542B (en) 2022-06-29
WO2019243430A1 (en) 2019-12-26
IL279024A (en) 2021-01-31
JP2021527437A (ja) 2021-10-14
AU2019291050A1 (en) 2020-12-24
BR112020026169A2 (pt) 2021-04-06
US20210238608A1 (en) 2021-08-05
CL2020003304A1 (es) 2021-06-04
CN112513273A (zh) 2021-03-16
PE20210314A1 (es) 2021-02-12
CA3103756A1 (en) 2019-12-26
MX2020013930A (es) 2021-03-09

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