TWI727009B - 治療性化合物之標靶性配體 - Google Patents

治療性化合物之標靶性配體 Download PDF

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Publication number
TWI727009B
TWI727009B TW106107454A TW106107454A TWI727009B TW I727009 B TWI727009 B TW I727009B TW 106107454 A TW106107454 A TW 106107454A TW 106107454 A TW106107454 A TW 106107454A TW I727009 B TWI727009 B TW I727009B
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Taiwan
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oligomeric compound
inhibitory
pharmaceutically acceptable
compound
acceptable salt
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TW106107454A
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Chinese (zh)
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TW201735953A (zh
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大衛Ｂ 羅茲馬
戴倫Ｈ 維克菲爾德
安卓Ｖ 布洛克希
強納森Ｄ 班森
李
濤 裴
佛瑞德 弗立茲
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美商愛羅海德製藥公司
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • 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
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    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
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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
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Bioinformatics & Cheminformatics (AREA)
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  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Crystallography & Structural Chemistry (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 Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
TW106107454A 2016-03-07 2017-03-07 治療性化合物之標靶性配體 TWI727009B (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201662304652P 2016-03-07 2016-03-07
US62/304,652 2016-03-07
US201662370754P 2016-08-04 2016-08-04
US62/370,754 2016-08-04
US201662426916P 2016-11-28 2016-11-28
US62/426,916 2016-11-28

Publications (2)

Publication Number Publication Date
TW201735953A TW201735953A (zh) 2017-10-16
TWI727009B true TWI727009B (zh) 2021-05-11

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TW106107454A TWI727009B (zh) 2016-03-07 2017-03-07 治療性化合物之標靶性配體
TW111137872A TWI842117B (zh) 2016-03-07 2017-03-07 治療性化合物之標靶性配體
TW110112971A TWI783434B (zh) 2016-03-07 2017-03-07 治療性化合物之標靶性配體

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TW111137872A TWI842117B (zh) 2016-03-07 2017-03-07 治療性化合物之標靶性配體
TW110112971A TWI783434B (zh) 2016-03-07 2017-03-07 治療性化合物之標靶性配體

Country Status (14)

Country Link
US (4) US10246709B2 (https=)
EP (1) EP3426261A4 (https=)
JP (4) JP6983797B2 (https=)
KR (3) KR102830443B1 (https=)
CN (2) CN113797348A (https=)
AU (3) AU2017229469B2 (https=)
BR (1) BR112018068230B1 (https=)
CA (1) CA3011946A1 (https=)
IL (3) IL296377A (https=)
JO (2) JOP20170057B1 (https=)
MX (3) MX2018009854A (https=)
TW (3) TWI727009B (https=)
UY (1) UY37146A (https=)
WO (1) WO2017156012A1 (https=)

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TWI842117B (zh) * 2016-03-07 2024-05-11 美商愛羅海德製藥公司 治療性化合物之標靶性配體

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