CN109069668B - 用于眼病的基因疗法 - Google Patents
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- CN109069668B CN109069668B CN201680081801.2A CN201680081801A CN109069668B CN 109069668 B CN109069668 B CN 109069668B CN 201680081801 A CN201680081801 A CN 201680081801A CN 109069668 B CN109069668 B CN 109069668B
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
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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
- A61K48/005—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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/04—Uses of viruses as vector in vivo
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/002—Vectors comprising a special translation-regulating system controllable or inducible
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- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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- Engineering & Computer Science (AREA)
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- General Health & Medical Sciences (AREA)
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- Public Health (AREA)
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- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Ophthalmology & Optometry (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266789P | 2015-12-14 | 2015-12-14 | |
| US62/266789 | 2015-12-14 | ||
| PCT/US2016/066402 WO2017106202A2 (en) | 2015-12-14 | 2016-12-13 | Gene therapy for ocular disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN109069668A CN109069668A (zh) | 2018-12-21 |
| CN109069668B true CN109069668B (zh) | 2023-04-18 |
Family
ID=58455636
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201680081801.2A Active CN109069668B (zh) | 2015-12-14 | 2016-12-13 | 用于眼病的基因疗法 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US11090392B2 (enExample) |
| EP (2) | EP3389724B1 (enExample) |
| JP (2) | JP7057281B2 (enExample) |
| KR (1) | KR20180099719A (enExample) |
| CN (1) | CN109069668B (enExample) |
| AU (1) | AU2016370487C1 (enExample) |
| BR (1) | BR112018011838A2 (enExample) |
| CA (1) | CA3008264A1 (enExample) |
| ES (1) | ES2826384T3 (enExample) |
| MX (2) | MX2018007230A (enExample) |
| RU (1) | RU2762747C2 (enExample) |
| WO (1) | WO2017106202A2 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3389724B1 (en) * | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| EP3377637B1 (en) * | 2016-04-08 | 2020-03-18 | Krystal Biotech, LLC | Compositions for use in methods for the treatment of wounds, disorders, and diseases of the skin |
| SG10202100632WA (en) * | 2016-07-26 | 2021-03-30 | Univ Cornell | Gene therapy for the treatment of aldehyde dehydrogenase deficiency |
| EP3638316A4 (en) * | 2017-06-14 | 2021-03-24 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR EYE DISEASES |
| CN112041434A (zh) | 2018-04-27 | 2020-12-04 | 克里斯托生物技术股份有限公司 | 用于美容性应用的编码一种或多种美容蛋白的重组核酸 |
| US20220143217A1 (en) * | 2019-03-04 | 2022-05-12 | The Trustees Of The University Of Pennsylvania | Neuroprotective gene therapy targeting the akt pathway |
| CN110889859A (zh) * | 2019-11-11 | 2020-03-17 | 珠海上工医信科技有限公司 | 一种用于眼底图像血管分割的u型网络 |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| CN116806158A (zh) * | 2020-09-02 | 2023-09-26 | 4D分子治疗有限公司 | 密码子优化的rep1基因及其用途 |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| KR20230160829A (ko) * | 2021-02-26 | 2023-11-24 | 로직바이오 테라퓨틱스, 인크. | 재조합 aav 벡터의 제조 및 사용 |
| JP2024513826A (ja) | 2021-04-02 | 2024-03-27 | クリスタル バイオテック インコーポレイテッド | がん治療のためのウイルスベクター |
| US20240335562A1 (en) * | 2021-08-04 | 2024-10-10 | Alpine Biotherapeutics Corporation | Sustainable ocular cell-mediated intraocular delivery of cellular therapeutics for treatment of ocular diseases or disorders |
Citations (4)
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| WO2011034947A2 (en) * | 2009-09-15 | 2011-03-24 | University Of Washington | Reagents and methods for modulating cone photoreceptor activity |
| WO2012114090A1 (en) * | 2011-02-22 | 2012-08-30 | Isis Innovation Limited | Aav -vectors for use in gene therapy of choroideremia |
| CN103608455A (zh) * | 2011-01-07 | 2014-02-26 | 应用遗传科技公司 | 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法 |
| WO2015160893A1 (en) * | 2014-04-15 | 2015-10-22 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr) |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
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| EP1381276A4 (en) | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
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| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
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| RU2444977C2 (ru) | 2009-08-03 | 2012-03-20 | Федеральное государственное учреждение "МОСКОВСКИЙ НАУЧНО-ИССЛЕДОВАТЕЛЬСКИЙ ИНСТИТУТ ГЛАЗНЫХ БОЛЕЗНЕЙ ИМЕНИ ГЕЛЬМГОЛЬЦА ФЕДЕРАЛЬНОГО АГЕНТСТВА ПО ВЫСОКОТЕХНОЛОГИЧНОЙ МЕДИЦИНСКОЙ ПОМОЩИ" | Способ диагностики ахроматопсии |
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| US20160206704A1 (en) | 2015-01-16 | 2016-07-21 | Isis Innovation Limited | Method |
| EP3389724B1 (en) * | 2015-12-14 | 2020-07-01 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
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2016
- 2016-12-13 EP EP16852859.4A patent/EP3389724B1/en active Active
- 2016-12-13 ES ES16852859T patent/ES2826384T3/es active Active
- 2016-12-13 AU AU2016370487A patent/AU2016370487C1/en active Active
- 2016-12-13 WO PCT/US2016/066402 patent/WO2017106202A2/en not_active Ceased
- 2016-12-13 RU RU2018125468A patent/RU2762747C2/ru active
- 2016-12-13 CN CN201680081801.2A patent/CN109069668B/zh active Active
- 2016-12-13 CA CA3008264A patent/CA3008264A1/en active Pending
- 2016-12-13 JP JP2018531202A patent/JP7057281B2/ja active Active
- 2016-12-13 KR KR1020187019811A patent/KR20180099719A/ko not_active Ceased
- 2016-12-13 BR BR112018011838-9A patent/BR112018011838A2/pt active Search and Examination
- 2016-12-13 US US16/061,530 patent/US11090392B2/en active Active
- 2016-12-13 MX MX2018007230A patent/MX2018007230A/es unknown
- 2016-12-13 EP EP20183330.8A patent/EP3795180B1/en active Active
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2018
- 2018-06-13 MX MX2022010959A patent/MX2022010959A/es unknown
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2021
- 2021-07-07 US US17/369,525 patent/US12403204B2/en active Active
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2022
- 2022-04-07 JP JP2022063749A patent/JP2022088656A/ja active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011034947A2 (en) * | 2009-09-15 | 2011-03-24 | University Of Washington | Reagents and methods for modulating cone photoreceptor activity |
| CN103608455A (zh) * | 2011-01-07 | 2014-02-26 | 应用遗传科技公司 | 用于治疗色盲和其它疾病的启动子、表达盒、载体、药盒和方法 |
| WO2012114090A1 (en) * | 2011-02-22 | 2012-08-30 | Isis Innovation Limited | Aav -vectors for use in gene therapy of choroideremia |
| WO2015160893A1 (en) * | 2014-04-15 | 2015-10-22 | Applied Genetic Technologies Corporation | Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr) |
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| BR112018011838A2 (pt) | 2018-12-04 |
| JP2022088656A (ja) | 2022-06-14 |
| EP3795180A1 (en) | 2021-03-24 |
| JP2019502378A (ja) | 2019-01-31 |
| WO2017106202A2 (en) | 2017-06-22 |
| JP7057281B2 (ja) | 2022-04-19 |
| ES2826384T3 (es) | 2021-05-18 |
| HK1258518A1 (zh) | 2019-11-15 |
| US20200061209A1 (en) | 2020-02-27 |
| RU2762747C2 (ru) | 2021-12-22 |
| US11090392B2 (en) | 2021-08-17 |
| MX2022010959A (es) | 2022-10-07 |
| WO2017106202A3 (en) | 2017-08-31 |
| EP3389724A2 (en) | 2018-10-24 |
| RU2018125468A (ru) | 2020-01-16 |
| KR20180099719A (ko) | 2018-09-05 |
| CN109069668A (zh) | 2018-12-21 |
| MX2018007230A (es) | 2018-11-09 |
| EP3795180B1 (en) | 2025-11-05 |
| AU2016370487A1 (en) | 2018-07-05 |
| EP3389724B1 (en) | 2020-07-01 |
| US12403204B2 (en) | 2025-09-02 |
| AU2016370487B2 (en) | 2021-08-05 |
| AU2016370487C1 (en) | 2022-03-03 |
| RU2018125468A3 (enExample) | 2020-10-22 |
| CA3008264A1 (en) | 2017-06-22 |
| US20210330816A1 (en) | 2021-10-28 |
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