CL2020003304A1 - Oligonucleótidos para modular la expresión de scn9a - Google Patents

Oligonucleótidos para modular la expresión de scn9a

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Publication number
CL2020003304A1
CL2020003304A1 CL2020003304A CL2020003304A CL2020003304A1 CL 2020003304 A1 CL2020003304 A1 CL 2020003304A1 CL 2020003304 A CL2020003304 A CL 2020003304A CL 2020003304 A CL2020003304 A CL 2020003304A CL 2020003304 A1 CL2020003304 A1 CL 2020003304A1
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CL
Chile
Prior art keywords
oligonucleotides
modulate
voltage
scn9a
expression
Prior art date
Application number
CL2020003304A
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English (en)
Inventor
Lykke Pedersen
Søren Rasmussen
Gianluigi Lichinchi
Christoffer Sondergaard
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Hoffmann La Roche
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Publication date
Application filed by Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of CL2020003304A1 publication Critical patent/CL2020003304A1/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
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    • C12N2310/11—Antisense
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    • 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
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
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    • 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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    • C12N2310/30—Chemical structure
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2320/00—Applications; Uses
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    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Pain & Pain Management (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

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.
CL2020003304A 2018-06-22 2020-12-18 Oligonucleótidos para modular la expresión de scn9a CL2020003304A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP18179339 2018-06-22
EP18194982 2018-09-18

Publications (1)

Publication Number Publication Date
CL2020003304A1 true CL2020003304A1 (es) 2021-06-04

Family

ID=66867163

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020003304A CL2020003304A1 (es) 2018-06-22 2020-12-18 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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WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
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EP4077672A1 (en) * 2019-12-20 2022-10-26 F. Hoffmann-La Roche AG Enhanced oligonucleotides for inhibiting scn9a expression
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
JP2023542889A (ja) * 2020-09-17 2023-10-12 キュー－ステート バイオサイエンシーズ， インコーポレイテッド 複数の標的を介して疼痛を処置するための治療用組成物
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WO2025153066A1 (zh) * 2024-01-19 2025-07-24 云合智药（苏州）生物科技有限公司 用于抑制钠电压门控通道α亚基10(SCN10A)基因表达的核酸及其用途
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CA3103756A1 (en) 2019-12-26
ZA202007542B (en) 2022-06-29
MX2020013930A (es) 2021-03-09
CR20200626A (es) 2021-02-22
JP2024056820A (ja) 2024-04-23
SG11202012161RA (en) 2021-01-28
WO2019243430A1 (en) 2019-12-26
PE20210314A1 (es) 2021-02-12
US12116576B2 (en) 2024-10-15
KR20210027384A (ko) 2021-03-10
EP3810776A1 (en) 2021-04-28
BR112020026169A2 (pt) 2021-04-06
IL279024A (en) 2021-01-31
AU2019291050A1 (en) 2020-12-24
CN112513273A (zh) 2021-03-16
US20210238608A1 (en) 2021-08-05
JP2021527437A (ja) 2021-10-14

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