TW202307208A - 反義寡聚物之組合 - Google Patents

反義寡聚物之組合 Download PDF

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TW202307208A
TW202307208A TW111123470A TW111123470A TW202307208A TW 202307208 A TW202307208 A TW 202307208A TW 111123470 A TW111123470 A TW 111123470A TW 111123470 A TW111123470 A TW 111123470A TW 202307208 A TW202307208 A TW 202307208A
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oligomer comprises
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TW111123470A
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戸根悠一郎
青木吉嗣
本橋紀夫
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日商日本新藥股份有限公司
國立研究開發法人國立精神 神經醫療研究中心
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Publication of TW202307208A publication Critical patent/TW202307208A/zh

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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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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    • C12N2310/00—Structure or type of the nucleic acid
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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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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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TW111123470A 2021-06-23 2022-06-23 反義寡聚物之組合 TW202307208A (zh)

Applications Claiming Priority (2)

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JP2021-104145 2021-06-23
JP2021104145 2021-06-23

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TW202307208A true TW202307208A (zh) 2023-02-16

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US (1) US20240301416A1 (cg-RX-API-DMAC7.html)
EP (1) EP4361269A4 (cg-RX-API-DMAC7.html)
JP (1) JPWO2022270585A1 (cg-RX-API-DMAC7.html)
KR (1) KR20240024176A (cg-RX-API-DMAC7.html)
CN (1) CN117940564A (cg-RX-API-DMAC7.html)
AU (1) AU2022298028A1 (cg-RX-API-DMAC7.html)
BR (1) BR112023027298A2 (cg-RX-API-DMAC7.html)
CA (1) CA3224782A1 (cg-RX-API-DMAC7.html)
CO (1) CO2023016805A2 (cg-RX-API-DMAC7.html)
IL (1) IL309626A (cg-RX-API-DMAC7.html)
MX (1) MX2023014417A (cg-RX-API-DMAC7.html)
TW (1) TW202307208A (cg-RX-API-DMAC7.html)
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US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US20230111212A1 (en) * 2020-01-10 2023-04-13 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
WO2025076006A2 (en) * 2023-10-02 2025-04-10 Avidity Biosciences, Inc. Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 45 skipping

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IL309626A (en) 2024-02-01
JPWO2022270585A1 (cg-RX-API-DMAC7.html) 2022-12-29
WO2022270585A1 (ja) 2022-12-29
EP4361269A4 (en) 2026-01-14
CO2023016805A2 (es) 2023-12-29
CN117940564A (zh) 2024-04-26
MX2023014417A (es) 2023-12-15
BR112023027298A2 (pt) 2024-03-12
AU2022298028A1 (en) 2023-12-21
CA3224782A1 (en) 2022-12-29
KR20240024176A (ko) 2024-02-23
US20240301416A1 (en) 2024-09-12
EP4361269A1 (en) 2024-05-01

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