KR20210068014A - 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법 - Google Patents

레버 유전성 시신경병증의 치료를 위한 조성물 및 방법 Download PDF

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KR20210068014A
KR20210068014A KR1020217007727A KR20217007727A KR20210068014A KR 20210068014 A KR20210068014 A KR 20210068014A KR 1020217007727 A KR1020217007727 A KR 1020217007727A KR 20217007727 A KR20217007727 A KR 20217007727A KR 20210068014 A KR20210068014 A KR 20210068014A
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nucleic acid
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빈 리
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우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니
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Priority claimed from CN201810948193.1A external-priority patent/CN110846392A/zh
Priority claimed from PCT/CN2018/103937 external-priority patent/WO2020000641A1/zh
Priority claimed from CN201811221305.XA external-priority patent/CN111073899B/zh
Priority claimed from CN201811230856.2A external-priority patent/CN111068071A/zh
Application filed by 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 filed Critical 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니
Priority claimed from PCT/CN2019/101538 external-priority patent/WO2020038352A1/en
Publication of KR20210068014A publication Critical patent/KR20210068014A/ko
Ceased legal-status Critical Current

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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • C12Y106/99003—NADH dehydrogenase (1.6.99.3)
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    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
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    • G01N33/502—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects
    • G01N33/5023—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing non-proliferative effects on expression patterns
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C12N2750/14011—Parvoviridae
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KR1020217007727A 2018-08-20 2019-08-20 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법 Ceased KR20210068014A (ko)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
CN201810948193.1A CN110846392A (zh) 2018-08-20 2018-08-20 一种重组腺相关病毒或含其的试剂盒及其应用
CN201810948193.1 2018-08-20
CNPCT/CN2018/103937 2018-09-04
PCT/CN2018/103937 WO2020000641A1 (zh) 2018-06-29 2018-09-04 编码人nadh脱氢酶亚单位蛋白的核酸及其应用
CN201811221305.X 2018-10-19
CN201811221305.XA CN111073899B (zh) 2018-10-19 2018-10-19 一种编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变
CN201811230856.2 2018-10-22
CNPCT/CN2018/113799 2018-11-02
PCT/CN2018/113799 WO2020082417A1 (zh) 2018-10-22 2018-11-02 基因治疗Leber遗传性视神经病变
PCT/CN2018/118662 WO2020077756A1 (zh) 2018-10-19 2018-11-30 Nd4蛋白的编码序列及其应用
CNPCT/CN2018/118662 2018-11-30
PCT/CN2019/070461 WO2020037938A1 (zh) 2018-08-20 2019-01-04 一种重组腺相关病毒或含其的试剂盒及其应用
CNPCT/CN2019/070461 2019-01-04
PCT/CN2019/101538 WO2020038352A1 (en) 2018-08-20 2019-08-20 Compositions and methods for treating leber's hereditary optic neuropathy

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Publication Number Publication Date
KR20210068014A true KR20210068014A (ko) 2021-06-08

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KR1020217007727A Ceased KR20210068014A (ko) 2018-08-20 2019-08-20 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법

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US (2) US11352645B2 (https=)
EP (1) EP3840785A4 (https=)
JP (2) JP7403852B2 (https=)
KR (1) KR20210068014A (https=)
CN (1) CN112584874A (https=)
AU (1) AU2019323434A1 (https=)
CA (1) CA3109432A1 (https=)
SG (1) SG11202101032VA (https=)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
WO2024144330A1 (ko) * 2022-12-29 2024-07-04 주식회사 엣진 레베르 유전성 시신경병증의 미토콘드리아 염기 변이 교정 시스템

Families Citing this family (7)

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Publication number Priority date Publication date Assignee Title
KR20240014102A (ko) 2018-06-29 2024-01-31 우한 뉴로프스 바이오테크놀로지 리미티드 컴퍼니 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CA3109432A1 (en) 2018-08-20 2020-02-27 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
US20210100856A1 (en) * 2019-09-11 2021-04-08 Adverum Biotechnologies, Inc. Methods of treating ocular neovascular diseases using aav2 variants encoding aflibercept
CN113025633B (zh) * 2019-12-09 2024-08-27 武汉纽福斯生物科技有限公司 编码人nadh脱氢酶亚单位1蛋白的核酸及其应用
CA3228344A1 (en) * 2021-08-06 2023-02-09 Wuhan Neurophth Biotechnology Limited Company Composition and method for treating leber's hereditary optic neuropathy caused by nd4 mutation
CN116789772A (zh) * 2022-03-18 2023-09-22 北京安龙生物医药有限公司 Aav5扩容衣壳突变体及其扩容检测方法
CN118320132A (zh) * 2024-04-26 2024-07-12 首都医科大学附属北京友谊医院 重组腺相关病毒rAAV2/Rec2作为胰腺靶向性载体的应用

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