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SG11202012161RA - Oligonucleotides for modulating scn9a expression - Google Patents

Oligonucleotides for modulating scn9a expression

Info

Publication number
SG11202012161RA
SG11202012161RA SG11202012161RA SG11202012161RA SG11202012161RA SG 11202012161R A SG11202012161R A SG 11202012161RA SG 11202012161R A SG11202012161R A SG 11202012161RA SG 11202012161R A SG11202012161R A SG 11202012161RA SG 11202012161R A SG11202012161R A SG 11202012161RA
Authority
SG
Singapore
Prior art keywords
oligonucleotides
modulating
scn9a expression
scn9a
expression
Prior art date
Application number
SG11202012161RA
Inventor
Lykke Pedersen
Søren Rasmussen
Gianluigi Lichinchi
Christoffer Sondergaard
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 SG11202012161RA publication Critical patent/SG11202012161RA/en

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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
    • C12N15/1138Non-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
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
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    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy

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  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • 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)
SG11202012161RA 2018-06-22 2019-06-19 Oligonucleotides for modulating scn9a expression SG11202012161RA (en)

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
SG11202012161RA true SG11202012161RA (en) 2021-01-28

Family

ID=66867163

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202012161RA SG11202012161RA (en) 2018-06-22 2019-06-19 Oligonucleotides for modulating scn9a expression

Country Status (16)

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

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KR102422625B1 (en) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 Regulation of gene expression and screening of deregulated protein expression
ES2882500T3 (en) 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of Dravet syndrome
AU2018322319B2 (en) 2017-08-25 2021-08-05 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
AR120817A1 (en) * 2019-12-20 2022-03-23 Hoffmann La Roche ENHANCED OLIGONUCLEOTIDES TO INHIBIT SCN9A EXPRESSION
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc opa1 antisense oligomers for the treatment of conditions and diseases
WO2022061108A2 (en) * 2020-09-17 2022-03-24 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
WO2022147541A1 (en) * 2021-01-04 2022-07-07 Exicure Operating Company Compounds for modulating scn9a expression
WO2022232395A2 (en) * 2021-04-28 2022-11-03 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets
WO2024002045A1 (en) * 2022-06-27 2024-01-04 Aoan Biosciences Oligonucleotide delivery agents, pharmaceutical compositions and methods using the same
WO2024169770A1 (en) * 2023-02-17 2024-08-22 苏州时安生物技术有限公司 Sirna inhibiting scn9a gene expression, pharmaceutical composition and use thereof
WO2024244000A1 (en) * 2023-06-02 2024-12-05 宜明(麻省)细胞生物科技有限公司 Engineered sgrna design and preparation method

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Also Published As

Publication number Publication date
EP3810776A1 (en) 2021-04-28
US12116576B2 (en) 2024-10-15
CR20200626A (en) 2021-02-22
ZA202007542B (en) 2022-06-29
CL2020003304A1 (en) 2021-06-04
JP2021527437A (en) 2021-10-14
KR20210027384A (en) 2021-03-10
JP2024056820A (en) 2024-04-23
IL279024A (en) 2021-01-31
US20210238608A1 (en) 2021-08-05
WO2019243430A1 (en) 2019-12-26
CN112513273A (en) 2021-03-16
MX2020013930A (en) 2021-03-09
CA3103756A1 (en) 2019-12-26
AU2019291050A1 (en) 2020-12-24
BR112020026169A2 (en) 2021-04-06
PE20210314A1 (en) 2021-02-12

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