EA038288B1 - Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a - Google Patents
Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a Download PDFInfo
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- EA038288B1 EA038288B1 EA201891137A EA201891137A EA038288B1 EA 038288 B1 EA038288 B1 EA 038288B1 EA 201891137 A EA201891137 A EA 201891137A EA 201891137 A EA201891137 A EA 201891137A EA 038288 B1 EA038288 B1 EA 038288B1
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- 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
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- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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| US201562255317P | 2015-11-13 | 2015-11-13 | |
| PCT/US2016/061684 WO2017083762A1 (en) | 2015-11-13 | 2016-11-11 | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
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| EA201891137A1 EA201891137A1 (ru) | 2018-12-28 |
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| EA201891137A EA038288B1 (ru) | 2015-11-13 | 2016-11-11 | Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a |
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| AU2016343887B2 (en) | 2015-10-28 | 2023-04-06 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| SG11201804064WA (en) * | 2015-11-13 | 2018-06-28 | Baxalta Inc | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
| US10189888B2 (en) | 2015-11-13 | 2019-01-29 | Baxalta Incorporated | Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A |
| US11191847B2 (en) | 2016-04-15 | 2021-12-07 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia B |
| PE20250736A1 (es) * | 2016-04-15 | 2025-03-10 | Univ Pennsylvania | Terapia genica para tratar la hemofilia a |
| ES2981519T3 (es) | 2016-11-04 | 2024-10-09 | Takeda Pharmaceuticals Co | Formulaciones de virus adeno-asociados |
| CA3077380A1 (en) * | 2017-09-27 | 2019-04-04 | Sigilon Therapeutics, Inc. | Methods, compositions, and implantable elements comprising active cells |
| EP3668997A4 (en) * | 2017-10-02 | 2021-05-19 | Research Institute at Nationwide Children's Hospital | SYSTEM FOR MIRNA DETARGETING FOR TISSUE-SPECIFIC INTERFERENCE |
| KR102105145B1 (ko) * | 2017-10-13 | 2020-05-13 | 영남대학교 산학협력단 | 촬영 장치 및 용기 |
| KR101952102B1 (ko) * | 2017-12-07 | 2019-02-26 | 주식회사 지앤피바이오사이언스 | 단백질 발현량이 증대된 인자 ⅷ 변이체 발현벡터 |
| CA3106590A1 (en) * | 2018-07-16 | 2020-01-23 | Baxalta Incorporated | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
| AU2019314513A1 (en) * | 2018-08-03 | 2021-01-28 | Sangamo Therapeutics, Inc. | Improved clinical parameters by expression of factor viii |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| TWI851647B (zh) * | 2019-01-16 | 2024-08-11 | 日商武田藥品工業股份有限公司 | 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體 |
| EP3923992A1 (en) | 2019-02-15 | 2021-12-22 | CRISPR Therapeutics AG | Gene editing for hemophilia a with improved factor viii expression |
| BR112021015776A2 (pt) * | 2019-06-05 | 2021-11-09 | Bayer Healthcare Llc | Edição de gene para hemofilia a com expressão de fator viii melhorada |
| US20200405883A1 (en) * | 2019-06-20 | 2020-12-31 | Baxalta Incorporated | Method of treatment with viral-based gene therapy |
| JP2023501262A (ja) * | 2019-11-01 | 2023-01-18 | フリーライン セラピューティクス リミテッド | 転写調節エレメント |
| EP4073106A2 (en) * | 2019-12-12 | 2022-10-19 | Takeda Pharmaceutical Company Limited | Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression |
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