TW202400787A - 改良siRNA生物可利用性之GalNAc組合物 - Google Patents

改良siRNA生物可利用性之GalNAc組合物 Download PDF

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TW202400787A
TW202400787A TW112109623A TW112109623A TW202400787A TW 202400787 A TW202400787 A TW 202400787A TW 112109623 A TW112109623 A TW 112109623A TW 112109623 A TW112109623 A TW 112109623A TW 202400787 A TW202400787 A TW 202400787A
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compound
alkyl
salt
independently selected
oligonucleotide
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TW112109623A
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Chinese (zh)
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達倫 Ｈ 瓦克菲
大衛 羅茲馬
歐莫瑞 戈特斯曼
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美商安彼瑞可股份有限公司
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/18—Acyclic radicals, substituted by carbocyclic rings
    • 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
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    • 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
    • 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
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • 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
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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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    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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TW112109623A 2022-03-16 2023-03-15 改良siRNA生物可利用性之GalNAc組合物 TW202400787A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202263320431P 2022-03-16 2022-03-16
US63/320,431 2022-03-16
US202263354359P 2022-06-22 2022-06-22
US63/354,359 2022-06-22
US202263430542P 2022-12-06 2022-12-06
US63/430,542 2022-12-06

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TW202400787A true TW202400787A (zh) 2024-01-01

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US (3) US11879125B2 (https=)
EP (1) EP4493570A2 (https=)
JP (1) JP2025509698A (https=)
KR (1) KR20250006359A (https=)
CN (1) CN119213005A (https=)
AU (1) AU2023234536A1 (https=)
CA (1) CA3254474A1 (https=)
IL (1) IL315546A (https=)
MX (1) MX2024011241A (https=)
TW (1) TW202400787A (https=)
WO (1) WO2023178144A2 (https=)

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