JP7672786B2 - 合成メッセンジャーｒｎａによる原発性繊毛機能不全症の治療 - Google Patents

合成メッセンジャーｒｎａによる原発性繊毛機能不全症の治療 Download PDF

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JP7672786B2
JP7672786B2 JP2019514195A JP2019514195A JP7672786B2 JP 7672786 B2 JP7672786 B2 JP 7672786B2 JP 2019514195 A JP2019514195 A JP 2019514195A JP 2019514195 A JP2019514195 A JP 2019514195A JP 7672786 B2 JP7672786 B2 JP 7672786B2
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ロックハート、デイビッド・ジェー
ウストマン、ブランドン
ヘニッヒ、ミルコ
イシマル、ダニエラ
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トランスクリプティクス・インコーポレイテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K48/0008—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
    • 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
    • A61K48/0041—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 polymeric
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    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
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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
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2800/00—Nucleic acids vectors
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2019514195A 2016-05-27 2017-05-26 合成メッセンジャーｒｎａによる原発性繊毛機能不全症の治療 Active JP7672786B2 (ja)

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JP2022098210A JP2022120180A (ja) 2016-05-27 2022-06-17 合成メッセンジャーｒｎａによる原発性繊毛機能不全症の治療
JP2025030716A JP2025090635A (ja) 2016-05-27 2025-02-27 合成メッセンジャーｒｎａによる原発性繊毛機能不全症の治療

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US201662342784P 2016-05-27 2016-05-27
US62/342,784 2016-05-27
PCT/US2017/034723 WO2017205767A1 (en) 2016-05-27 2017-05-26 Treatment of primary ciliary dyskinesia with synthetic messenger rna

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EP (2) EP3463483B1 (https=)
JP (3) JP7672786B2 (https=)
CN (2) CN117018228A (https=)
AU (3) AU2017271665B2 (https=)
CA (1) CA3025626A1 (https=)
DK (1) DK3463483T3 (https=)
ES (1) ES2972405T3 (https=)
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CN111500697A (zh) * 2020-03-20 2020-08-07 四川省人民医院 不动纤毛综合症筛查试剂盒
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WO2022204215A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
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JP2024527541A (ja) * 2021-07-01 2024-07-25 トランスレイト バイオ， インコーポレイテッド Ｍｒｎａの送達のための組成物
EP4379055A4 (en) * 2021-07-27 2025-08-06 Sk Bioscience Co Ltd MRNA FOR PROTEIN EXPRESSION AND TEMPLATE FOR IT
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CN115287326B (zh) * 2022-08-05 2025-05-16 中国科学院深圳先进技术研究院 一种评估流感嗜血杆菌感染性的方法
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