JP7712270B2 - 未成熟終止コドン媒介障害を処置するための方法および組成物 - Google Patents

未成熟終止コドン媒介障害を処置するための方法および組成物

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JP7712270B2
JP7712270B2 JP2022526004A JP2022526004A JP7712270B2 JP 7712270 B2 JP7712270 B2 JP 7712270B2 JP 2022526004 A JP2022526004 A JP 2022526004A JP 2022526004 A JP2022526004 A JP 2022526004A JP 7712270 B2 JP7712270 B2 JP 7712270B2
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ピーター エム． エイモン
ショーン マクファーランド
イン－シン チェン
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テバード バイオサイエンシズ インコーポレイテッド
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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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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JP2022526004A 2019-11-01 2020-10-30 未成熟終止コドン媒介障害を処置するための方法および組成物 Active JP7712270B2 (ja)

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US201962929428P 2019-11-01 2019-11-01
US62/929,428 2019-11-01
PCT/US2020/058415 WO2021087401A1 (en) 2019-11-01 2020-10-30 Methods and compositions for treating a premature termination codon-mediated disorder

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CN (2) CN119955780A (enExample)
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WO2025119204A1 (en) * 2023-12-04 2025-06-12 Modit Therapeutics Beijing Limited Methods and compositions for improved readthrough of pseudouridine-modified codons
WO2026030209A1 (en) * 2024-07-29 2026-02-05 University Of Rochester Use of decoding trnas to boost protein expression or function
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JP2025148444A (ja) 2025-10-07
US20240148772A1 (en) 2024-05-09
CN115209902B (zh) 2025-01-07
CN119955780A (zh) 2025-05-09
AU2020375040A1 (en) 2022-05-19
CN115209902A (zh) 2022-10-18
JP2022554357A (ja) 2022-12-28
WO2021087401A1 (en) 2021-05-06
EP4051291A1 (en) 2022-09-07
KR20220155981A (ko) 2022-11-24
US12697350B2 (en) 2026-08-04
CA3156791A1 (en) 2021-05-06
EP4051291A4 (en) 2024-07-03

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