KR20230122076A - 아데노-연관 바이러스 벡터를 함유하는 개선된 제약조성물 - Google Patents
아데노-연관 바이러스 벡터를 함유하는 개선된 제약조성물 Download PDFInfo
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- KR20230122076A KR20230122076A KR1020237023859A KR20237023859A KR20230122076A KR 20230122076 A KR20230122076 A KR 20230122076A KR 1020237023859 A KR1020237023859 A KR 1020237023859A KR 20237023859 A KR20237023859 A KR 20237023859A KR 20230122076 A KR20230122076 A KR 20230122076A
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- chloride
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- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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- 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
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- 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
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- 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
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| US202063127826P | 2020-12-18 | 2020-12-18 | |
| US63/127,826 | 2020-12-18 | ||
| PCT/US2021/064222 WO2022133324A1 (en) | 2020-12-18 | 2021-12-17 | Improved pharmaceutical compositions containing adeno-associated viral vector |
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| US20240307553A1 (en) * | 2021-04-01 | 2024-09-19 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| EP4651883A1 (en) * | 2023-01-19 | 2025-11-26 | uniQure biopharma B.V. | Pharmaceutical formulations of gene delivery vehicles |
| WO2025045083A1 (zh) * | 2023-09-01 | 2025-03-06 | 康霖生物科技（杭州）有限公司 | 一种液体制剂及其用途 |
| WO2025076729A1 (zh) * | 2023-10-11 | 2025-04-17 | 朗信启昇(苏州)生物制药有限公司 | 包含aav的药物组合物及其制备方法 |
| WO2025108407A2 (en) * | 2023-11-23 | 2025-05-30 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Gene therapy compositions and methods for treating glioma |
| WO2025113676A1 (en) * | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
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| ME03008B (me) * | 2011-09-19 | 2018-10-20 | Axon Neuroscience Se | Terapija na osnovu proteina i dijagnoza patologije u којој posreduje tau u alcнajмerovoj bolesti |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| MY189674A (en) | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| CN112770812A (zh) * | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| US12497632B2 (en) * | 2018-08-03 | 2025-12-16 | Sangamo Therapeutics, Inc. | Clinical parameters by expression of factor VIII |
| US12478553B2 (en) * | 2018-11-13 | 2025-11-25 | Sio2 Medical Products, Llc | Polymer vials with substantially flat bottoms and injection stretch blow molding methods for making the same |
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
| AU2019428629A1 (en) | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| WO2021071835A1 (en) * | 2019-10-07 | 2021-04-15 | Regenxbio Inc. | Adeno-associated virus vector pharmaceutical composition and methods |
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