KR20230003477A - 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도 - Google Patents

비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도 Download PDF

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KR20230003477A
KR20230003477A KR1020227036899A KR20227036899A KR20230003477A KR 20230003477 A KR20230003477 A KR 20230003477A KR 1020227036899 A KR1020227036899 A KR 1020227036899A KR 20227036899 A KR20227036899 A KR 20227036899A KR 20230003477 A KR20230003477 A KR 20230003477A
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cedna
itr
cedna vector
itrs
fix
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더글라스 앤소니 커
필립 사마요아
나다니엘 실버
세바스찬 아귀레
데브라 클라테
엘리 헤르첼 파사코우
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제너레이션 바이오 컴퍼니
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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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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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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    • C12N2800/00—Nucleic acids vectors
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    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
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KR1020227036899A 2020-03-24 2021-03-24 비-바이러스성 dna 벡터 및 인자 ix 치료제 발현을 위한 이의 용도 Withdrawn KR20230003477A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062993857P 2020-03-24 2020-03-24
US62/993,857 2020-03-24
PCT/US2021/023891 WO2021195214A1 (en) 2020-03-24 2021-03-24 Non-viral dna vectors and uses thereof for expressing factor ix therapeutics

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KR20230003477A true KR20230003477A (ko) 2023-01-06

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US (1) US20230138409A1 (https=)
EP (1) EP4127187A1 (https=)
JP (2) JP2023520764A (https=)
KR (1) KR20230003477A (https=)
CN (1) CN115667530A (https=)
AU (1) AU2021244555A1 (https=)
CA (1) CA3172572A1 (https=)
IL (1) IL296660A (https=)
MX (1) MX2022011805A (https=)
WO (1) WO2021195214A1 (https=)

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CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
AU2024257493A1 (en) * 2023-04-18 2025-12-04 Siren Biotechnology, Inc. Adeno-associated viral vector compositions and methods of use

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