KR20170137730A - 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 - Google Patents
망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 Download PDFInfo
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- KR20170137730A KR20170137730A KR1020177027451A KR20177027451A KR20170137730A KR 20170137730 A KR20170137730 A KR 20170137730A KR 1020177027451 A KR1020177027451 A KR 1020177027451A KR 20177027451 A KR20177027451 A KR 20177027451A KR 20170137730 A KR20170137730 A KR 20170137730A
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
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- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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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
- A61K48/0075—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
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- 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
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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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- 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
- C12N15/861—Adenoviral vectors
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- Biotechnology (AREA)
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- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
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- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Virology (AREA)
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- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Ophthalmology & Optometry (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
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- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562127194P | 2015-03-02 | 2015-03-02 | |
| US62/127,194 | 2015-03-02 | ||
| US201562134466P | 2015-03-17 | 2015-03-17 | |
| US62/134,466 | 2015-03-17 | ||
| PCT/US2016/020482 WO2016141078A1 (en) | 2015-03-02 | 2016-03-02 | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20170137730A true KR20170137730A (ko) | 2017-12-13 |
Family
ID=56848956
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020177027451A Ceased KR20170137730A (ko) | 2015-03-02 | 2016-03-02 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US11021519B2 (https=) |
| EP (1) | EP3265178B1 (https=) |
| JP (3) | JP7095994B2 (https=) |
| KR (1) | KR20170137730A (https=) |
| CN (2) | CN107405507B (https=) |
| AU (2) | AU2016226289B2 (https=) |
| BR (1) | BR112017018846A2 (https=) |
| CA (1) | CA2978068C (https=) |
| EA (1) | EA201791939A1 (https=) |
| HK (1) | HK1249067A1 (https=) |
| IL (1) | IL254148B (https=) |
| MX (1) | MX2017011041A (https=) |
| MY (1) | MY187898A (https=) |
| SG (1) | SG11201707063TA (https=) |
| WO (1) | WO2016141078A1 (https=) |
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| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CN105755044A (zh) | 2011-04-22 | 2016-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| TWI698240B (zh) | 2012-05-15 | 2020-07-11 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| EP3003391B1 (en) | 2013-05-31 | 2021-09-22 | The Regents of The University of California | Adeno-associated virus variants and methods of use thereof |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US11021519B2 (en) | 2015-03-02 | 2021-06-01 | Adverum Biotechnologies, Inc. | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| JP6836999B2 (ja) | 2015-03-24 | 2021-03-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California | アデノ随伴ウイルス変異体及びその使用方法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| ES2840059T3 (es) * | 2016-06-16 | 2021-07-06 | Adverum Biotechnologies Inc | Tratamiento de la DMRE mediante el uso de la variante AAV2 con aflibercept |
| AU2017302013B2 (en) | 2016-07-29 | 2022-05-26 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) * | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| AU2017390276A1 (en) * | 2017-01-03 | 2019-06-27 | Vitrean, Inc. | Methods and devices for treating a retinal detachment |
| WO2018134168A1 (en) * | 2017-01-17 | 2018-07-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors |
| EP4653536A3 (en) | 2017-03-17 | 2026-02-25 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression |
| CA3053154A1 (en) * | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
| JP2020534788A (ja) | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| RU2770922C2 (ru) | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| US12043848B2 (en) | 2017-10-16 | 2024-07-23 | Vigeneron Gmbh | AAV vectors |
| CN111867635A (zh) * | 2017-10-20 | 2020-10-30 | 法国国家卫生及研究医学协会 | 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法 |
| GB201800546D0 (en) | 2018-01-12 | 2018-02-28 | Ucl Business Plc | Treatment |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
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| JP7371083B2 (ja) * | 2018-07-20 | 2023-10-30 | ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 遺伝子治療発現ベクターの眼内送達 |
| CN111118016B (zh) | 2018-10-30 | 2022-04-15 | 上海朗昇生物科技有限公司 | 治疗视网膜色素变性疾病的基因治疗载体 |
| EP3897679A1 (en) * | 2018-12-21 | 2021-10-27 | Wade Wei-De Chien | Adeno-associated viruses and their uses for inner ear therapy |
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| JP7597380B2 (ja) * | 2019-03-04 | 2024-12-10 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | 対側眼へのaav遺伝子治療の逐次的硝子体内投与 |
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| TW202532428A (zh) * | 2019-04-26 | 2025-08-16 | 美商愛德維仁生物科技公司 | 用於玻璃體內遞送之變異體aav蛋白殼 |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ,インコーポレイテッド | ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター |
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| US12031147B2 (en) * | 2020-05-29 | 2024-07-09 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsids and methods of use thereof |
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| KR20190038536A (ko) | 2016-06-16 | 2019-04-08 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 안구 혈관신생을 저하시키기 위한 조성물 및 방법 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| JP2020510424A (ja) | 2017-02-28 | 2020-04-09 | アドヴェラム バイオテクノロジーズ, インコーポレイテッド | 改変aavカプシドおよびその使用 |
| EP4653536A3 (en) | 2017-03-17 | 2026-02-25 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression |
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| HK1249067A1 (zh) | 2018-10-26 |
| IL254148B (en) | 2022-09-01 |
| CN107405507A (zh) | 2017-11-28 |
| CN114480502A (zh) | 2022-05-13 |
| AU2016226289A1 (en) | 2017-09-21 |
| US20210388030A1 (en) | 2021-12-16 |
| SG11201707063TA (en) | 2017-09-28 |
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| AU2021209206A1 (en) | 2021-08-19 |
| JP7095994B2 (ja) | 2022-07-05 |
| CA2978068A1 (en) | 2016-09-09 |
| BR112017018846A2 (pt) | 2018-07-31 |
| AU2016226289B2 (en) | 2021-04-29 |
| US11021519B2 (en) | 2021-06-01 |
| WO2016141078A1 (en) | 2016-09-09 |
| EA201791939A1 (ru) | 2018-01-31 |
| EP3265178A4 (en) | 2018-09-05 |
| JP2020122008A (ja) | 2020-08-13 |
| EP3265178B1 (en) | 2026-05-06 |
| IL254148A0 (en) | 2017-10-31 |
| MX2017011041A (es) | 2018-03-02 |
| JP2018508519A (ja) | 2018-03-29 |
| EP3265178A1 (en) | 2018-01-10 |
| MY187898A (en) | 2021-10-27 |
| US20180066022A1 (en) | 2018-03-08 |
| CN107405507B (zh) | 2022-05-03 |
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