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SG11202110896WA - Compositions and methods for inhibiting gene expression in the central nervous system - Google Patents

Compositions and methods for inhibiting gene expression in the central nervous system

Info

Publication number
SG11202110896WA
SG11202110896WA SG11202110896WA SG11202110896WA SG11202110896WA SG 11202110896W A SG11202110896W A SG 11202110896WA SG 11202110896W A SG11202110896W A SG 11202110896WA SG 11202110896W A SG11202110896W A SG 11202110896WA SG 11202110896W A SG11202110896W A SG 11202110896WA
Authority
SG
Singapore
Prior art keywords
compositions
methods
gene expression
nervous system
central nervous
Prior art date
Application number
SG11202110896WA
Inventor
Bob Dale Brown
Maire Osborn
Weimin Wang
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 SG11202110896WA publication Critical patent/SG11202110896WA/en

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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
    • C12N15/1137—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 enzymes
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • A61P25/22—Anxiolytics
    • 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
    • A61P25/28—Drugs 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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    • C12Y102/00—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2)
    • C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
    • C12Y102/01003—Aldehyde dehydrogenase (NAD+) (1.2.1.3)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3521—Methyl
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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SG11202110896WA 2019-04-04 2020-04-03 Compositions and methods for inhibiting gene expression in the central nervous system SG11202110896WA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962829595P 2019-04-04 2019-04-04
PCT/US2020/026717 WO2020206350A1 (en) 2019-04-04 2020-04-03 Compositions and methods for inhibiting gene expression in the central nervous system

Publications (1)

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SG11202110896WA true SG11202110896WA (en) 2021-10-28

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SG11202110896WA SG11202110896WA (en) 2019-04-04 2020-04-03 Compositions and methods for inhibiting gene expression in the central nervous system

Country Status (13)

Country Link
US (1) US20220170025A1 (en)
EP (1) EP3947683A1 (en)
JP (1) JP2022526419A (en)
KR (1) KR20210148264A (en)
CN (1) CN113748208A (en)
AU (1) AU2020252560A1 (en)
BR (1) BR112021019793A2 (en)
CA (1) CA3135402A1 (en)
CL (1) CL2021002585A1 (en)
IL (1) IL286868A (en)
MX (1) MX2021012126A (en)
SG (1) SG11202110896WA (en)
WO (1) WO2020206350A1 (en)

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CA3135402A1 (en) 2020-10-08
AU2020252560A1 (en) 2021-10-28
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