CN111068071A - 基因治疗Leber遗传学视神经病变 - Google Patents

基因治疗Leber遗传学视神经病变 Download PDF

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Publication number
CN111068071A
CN111068071A CN201811230856.2A CN201811230856A CN111068071A CN 111068071 A CN111068071 A CN 111068071A CN 201811230856 A CN201811230856 A CN 201811230856A CN 111068071 A CN111068071 A CN 111068071A
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associated virus
recombinant adeno
polypeptide
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Chinese (zh)
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李斌
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Wuhan Niufusi Biological Technology Co ltd
Wuhan Neurophth Biotechnology Ltd Co
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Wuhan Niufusi Biological Technology Co ltd
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Priority to CN201811230856.2A priority Critical patent/CN111068071A/zh
Priority to PCT/CN2018/113799 priority patent/WO2020082417A1/fr
Priority to MX2020013772A priority patent/MX2020013772A/es
Priority to SG11202012044QA priority patent/SG11202012044QA/en
Priority to KR1020217001385A priority patent/KR102627561B1/ko
Priority to CN202110786772.2A priority patent/CN113528510A/zh
Priority to JP2021521870A priority patent/JP2021529001A/ja
Priority to CN201980003485.0A priority patent/CN110876269B/zh
Priority to KR1020247001775A priority patent/KR20240014102A/ko
Priority to CA3103740A priority patent/CA3103740A1/fr
Priority to EP19826653.8A priority patent/EP3814492A4/fr
Priority to PCT/CN2019/094136 priority patent/WO2020001657A1/fr
Priority to CN202110786630.6A priority patent/CN113476484A/zh
Priority to BR112020026361-3A priority patent/BR112020026361A2/pt
Priority to AU2019296451A priority patent/AU2019296451B2/en
Priority to SG11202101032VA priority patent/SG11202101032VA/en
Priority to AU2019323434A priority patent/AU2019323434A1/en
Priority to PCT/CN2019/101538 priority patent/WO2020038352A1/fr
Priority to KR1020217007727A priority patent/KR20210068014A/ko
Priority to JP2021509893A priority patent/JP7403852B2/ja
Priority to EP19853225.1A priority patent/EP3840785A4/fr
Priority to CN201980054770.5A priority patent/CN112584874A/zh
Priority to CA3109432A priority patent/CA3109432A1/fr
Priority to US16/836,644 priority patent/US11034954B2/en
Publication of CN111068071A publication Critical patent/CN111068071A/zh
Priority to US17/181,849 priority patent/US11352645B2/en
Priority to US17/317,295 priority patent/US20220340895A1/en
Priority to US17/320,388 priority patent/US11332741B1/en
Priority to AU2021204690A priority patent/AU2021204690A1/en
Priority to US17/726,833 priority patent/US20220259619A1/en
Priority to JP2023029170A priority patent/JP2023078173A/ja
Priority to JP2023205807A priority patent/JP2024028861A/ja
Priority to AU2023285773A priority patent/AU2023285773A1/en
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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
    • A61K48/0033—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 non-polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • 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
    • A61K48/005—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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN201811230856.2A 2018-06-29 2018-10-22 基因治疗Leber遗传学视神经病变 Pending CN111068071A (zh)

Priority Applications (32)

Application Number Priority Date Filing Date Title
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变
PCT/CN2018/113799 WO2020082417A1 (fr) 2018-10-22 2018-11-02 Thérapie génique pour neuropathie optique héréditaire de leber
MX2020013772A MX2020013772A (es) 2018-06-29 2019-07-01 Composiciones y métodos para el tratamiento de la neuropatía óptica hereditaria de leber.
SG11202012044QA SG11202012044QA (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy
KR1020217001385A KR102627561B1 (ko) 2018-06-29 2019-07-01 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CN202110786772.2A CN113528510A (zh) 2018-06-29 2019-07-01 治疗遗传性视神经病变的组合物和方法
JP2021521870A JP2021529001A (ja) 2018-06-29 2019-07-01 レーベル遺伝性視神経症を治療するための組成物及び方法
CN201980003485.0A CN110876269B (zh) 2018-06-29 2019-07-01 治疗遗传性视神经病变的组合物和方法
KR1020247001775A KR20240014102A (ko) 2018-06-29 2019-07-01 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
CA3103740A CA3103740A1 (fr) 2018-06-29 2019-07-01 Compositions et methodes de traitement de la neuropathie optique hereditaire de leber
EP19826653.8A EP3814492A4 (fr) 2018-06-29 2019-07-01 Compositions et méthodes de traitement de la neuropathie optique héréditaire de leber
PCT/CN2019/094136 WO2020001657A1 (fr) 2018-06-29 2019-07-01 Compositions et méthodes de traitement de la neuropathie optique héréditaire de leber
CN202110786630.6A CN113476484A (zh) 2018-06-29 2019-07-01 治疗遗传性视神经病变的组合物和方法
BR112020026361-3A BR112020026361A2 (pt) 2018-06-29 2019-07-01 Composições e métodos para tratar neuropatia óptica hereditária de leber
AU2019296451A AU2019296451B2 (en) 2018-06-29 2019-07-01 Compositions and methods for treating leber's hereditary optic neuropathy
SG11202101032VA SG11202101032VA (en) 2018-08-20 2019-08-20 Compositions and methods for treating leber's hereditary optic neuropathy
CA3109432A CA3109432A1 (fr) 2018-08-20 2019-08-20 Compositions et methodes de traitement de la neuropathie optique hereditaire de leber
AU2019323434A AU2019323434A1 (en) 2018-08-20 2019-08-20 Compositions and methods for treating leber's hereditary optic neuropathy
PCT/CN2019/101538 WO2020038352A1 (fr) 2018-08-20 2019-08-20 Compositions et méthodes de traitement de la neuropathie optique héréditaire de leber
KR1020217007727A KR20210068014A (ko) 2018-08-20 2019-08-20 레버 유전성 시신경병증의 치료를 위한 조성물 및 방법
JP2021509893A JP7403852B2 (ja) 2018-08-20 2019-08-20 レーベル遺伝性視神経症を治療するための組成物及び方法
EP19853225.1A EP3840785A4 (fr) 2018-08-20 2019-08-20 Compositions et méthodes de traitement de la neuropathie optique héréditaire de leber
CN201980054770.5A CN112584874A (zh) 2018-08-20 2019-08-20 用于治疗莱伯氏遗传性视神经病变的组合物和方法
US16/836,644 US11034954B2 (en) 2018-06-29 2020-03-31 Compositions and methods for treating leber's hereditary optic neuropathy
US17/181,849 US11352645B2 (en) 2018-08-20 2021-02-22 Compositions and methods for treating Leber's hereditary optic neuropathy
US17/317,295 US20220340895A1 (en) 2018-06-29 2021-05-11 Compositions and methods for treating leber's hereditary optic neuropathy
US17/320,388 US11332741B1 (en) 2018-06-29 2021-05-14 Compositions and methods for treating leber's hereditary optic neuropathy
AU2021204690A AU2021204690A1 (en) 2018-06-29 2021-07-05 Compositions and methods for treating Leber's hereditary optic neuropathy
US17/726,833 US20220259619A1 (en) 2018-08-20 2022-04-22 Compositions and methods for treating leber's hereditary optic neuropathy
JP2023029170A JP2023078173A (ja) 2018-06-29 2023-02-28 レーベル遺伝性視神経症を治療するための組成物及び方法
JP2023205807A JP2024028861A (ja) 2018-08-20 2023-12-06 レーベル遺伝性視神経症を治療するための組成物及び方法
AU2023285773A AU2023285773A1 (en) 2018-06-29 2023-12-20 Compositions and methods for treating Leber's hereditary optic neuropathy

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
CN201811230856.2A CN111068071A (zh) 2018-10-22 2018-10-22 基因治疗Leber遗传学视神经病变

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CN (1) CN111068071A (fr)
WO (1) WO2020082417A1 (fr)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110857440A (zh) * 2018-08-23 2020-03-03 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
WO2023011632A1 (fr) * 2021-08-06 2023-02-09 武汉纽福斯生物科技有限公司 Composition et méthode de traitement de la neuropathie optique héréditaire de leber provoquée par la mutation nd4

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202012044QA (en) 2018-06-29 2021-01-28 Wuhan Neurophth Biological Tech Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
AU2019323434A1 (en) 2018-08-20 2021-02-25 Wuhan Neurophth Biotechnology Limited Company Compositions and methods for treating leber's hereditary optic neuropathy
CN113025633A (zh) 2019-12-09 2021-06-25 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

Citations (4)

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Publication number Priority date Publication date Assignee Title
CN101553235A (zh) * 2006-06-01 2009-10-07 诺瓦加利制药公司 甾族化合物前药在治疗眼后段疾病中的用途
CN102634527A (zh) * 2012-04-11 2012-08-15 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
CN103897034A (zh) * 2012-12-27 2014-07-02 上海市第一人民医院 一种预防和/或治疗炎症反应的小分子多肽及其应用
CN104450747A (zh) * 2014-09-23 2015-03-25 李斌 用于治疗Leber遗传性视神经病变的重组腺相关病毒-NADH脱氢酶亚单位4基因全长以及药剂

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101553235A (zh) * 2006-06-01 2009-10-07 诺瓦加利制药公司 甾族化合物前药在治疗眼后段疾病中的用途
CN102634527A (zh) * 2012-04-11 2012-08-15 华中科技大学同济医学院附属同济医院 重组人nadh脱氢酶亚单位4基因及其表达载体构建方法
CN103897034A (zh) * 2012-12-27 2014-07-02 上海市第一人民医院 一种预防和/或治疗炎症反应的小分子多肽及其应用
CN104450747A (zh) * 2014-09-23 2015-03-25 李斌 用于治疗Leber遗传性视神经病变的重组腺相关病毒-NADH脱氢酶亚单位4基因全长以及药剂

Non-Patent Citations (1)

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Title
孙莉等: "磷酸肌酸钠治疗Leber遗传性视神经病变的临床观察及护理", 《实用临床护理学杂志》 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110857440A (zh) * 2018-08-23 2020-03-03 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
WO2023011632A1 (fr) * 2021-08-06 2023-02-09 武汉纽福斯生物科技有限公司 Composition et méthode de traitement de la neuropathie optique héréditaire de leber provoquée par la mutation nd4

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