KR20220155981A - 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물 - Google Patents

미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물 Download PDF

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KR20220155981A
KR20220155981A KR1020227018429A KR20227018429A KR20220155981A KR 20220155981 A KR20220155981 A KR 20220155981A KR 1020227018429 A KR1020227018429 A KR 1020227018429A KR 20227018429 A KR20227018429 A KR 20227018429A KR 20220155981 A KR20220155981 A KR 20220155981A
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trna
seq
artificial sequence
gene
dna
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피터 엠. 에이몬
션 맥팔랜드
잉신 첸
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테바드 바이오사이언시즈, 인크.
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
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    • 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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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KR1020227018429A 2019-11-01 2020-10-30 미성숙 종결 코돈-매개 장애를 치료하기 위한 방법 및 조성물 Ceased KR20220155981A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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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KR20220155981A true KR20220155981A (ko) 2022-11-24

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US (1) US12697350B2 (enExample)
EP (1) EP4051291A4 (enExample)
JP (2) JP7712270B2 (enExample)
KR (1) KR20220155981A (enExample)
CN (2) CN119955780A (enExample)
AU (1) AU2020375040A1 (enExample)
CA (1) CA3156791A1 (enExample)
WO (1) WO2021087401A1 (enExample)

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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
JP7712270B2 (ja) 2025-07-23
WO2021087401A1 (en) 2021-05-06
EP4051291A1 (en) 2022-09-07
US12697350B2 (en) 2026-08-04
CA3156791A1 (en) 2021-05-06
EP4051291A4 (en) 2024-07-03

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