KR20240014477A - 변이체 캡시드를 갖는 재조합 아데노-연관 바이러스 및 이의 응용 - Google Patents

변이체 캡시드를 갖는 재조합 아데노-연관 바이러스 및 이의 응용 Download PDF

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KR20240014477A
KR20240014477A KR1020237043802A KR20237043802A KR20240014477A KR 20240014477 A KR20240014477 A KR 20240014477A KR 1020237043802 A KR1020237043802 A KR 1020237043802A KR 20237043802 A KR20237043802 A KR 20237043802A KR 20240014477 A KR20240014477 A KR 20240014477A
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웬타오 장
쳉 리아오
웨이 닝
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상하이 레제네리드 테라피즈 컴퍼니 리미티드
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    • 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
    • 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
    • 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
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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
    • 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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020237043802A 2021-05-28 2022-05-27 변이체 캡시드를 갖는 재조합 아데노-연관 바이러스 및 이의 응용 Pending KR20240014477A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
CN202110594986 2021-05-28
CN202110594986.X 2021-05-28
PCT/CN2022/095422 WO2022247917A1 (zh) 2021-05-28 2022-05-27 衣壳变异的重组腺相关病毒及其应用

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US (1) US20240252679A1 (https=)
EP (1) EP4349852A4 (https=)
JP (1) JP2024519888A (https=)
KR (1) KR20240014477A (https=)
CN (1) CN117157309A (https=)
AU (1) AU2022281825A1 (https=)
BR (1) BR112023024375A2 (https=)
CA (1) CA3219898A1 (https=)
MX (1) MX2023014041A (https=)
TW (1) TW202313096A (https=)
WO (1) WO2022247917A1 (https=)

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CN119569833A (zh) * 2023-09-06 2025-03-07 北京锦篮基因科技有限公司 靶向骨骼肌的aav衣壳蛋白变体及其用途
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CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用
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CN119039401B (zh) * 2024-08-19 2025-04-11 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN118754952B (zh) * 2024-09-06 2025-01-10 上海玮美基因科技有限责任公司 一种新型腺相关病毒及其应用
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