KR20230128001A - 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도 - Google Patents
엔젤만 증후군의 치료를 위한 조성물 및 이의 용도 Download PDFInfo
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- KR20230128001A KR20230128001A KR1020237021519A KR20237021519A KR20230128001A KR 20230128001 A KR20230128001 A KR 20230128001A KR 1020237021519 A KR1020237021519 A KR 1020237021519A KR 20237021519 A KR20237021519 A KR 20237021519A KR 20230128001 A KR20230128001 A KR 20230128001A
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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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- 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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- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
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- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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Applications Claiming Priority (5)
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| US202063119860P | 2020-12-01 | 2020-12-01 | |
| US63/119,860 | 2020-12-01 | ||
| US202163179807P | 2021-04-26 | 2021-04-26 | |
| US63/179,807 | 2021-04-26 | ||
| PCT/US2021/061346 WO2022119890A1 (en) | 2020-12-01 | 2021-12-01 | Compositions and uses thereof for treatment of angelman syndrome |
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| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
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| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| ES2829199T3 (es) | 2010-11-22 | 2021-05-31 | Amicus Therapeutics Inc | Nuevas secuencias de señalización para mejorar la expresión y segregación de proteínas de enzimas recombinantes y otras proteínas |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
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| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
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| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
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| JP2021505135A (ja) | 2017-11-30 | 2021-02-18 | アミカス セラピューティックス インコーポレイテッド | Ｃｄｋｌ５発現変異体及びｃｄｋｌ５融合タンパク質 |
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| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN114206393A (zh) * | 2019-03-21 | 2022-03-18 | Ptc医疗公司 | 用于治疗天使综合征的载体和方法 |
| SG11202111375RA (en) * | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| US12465655B2 (en) * | 2019-05-22 | 2025-11-11 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| IT201900008877A1 (it) | 2019-06-13 | 2020-12-13 | Univ Bologna Alma Mater Studiorum | Nuovi costrutti per terapia genica |
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| US20230043046A1 (en) | 2019-10-30 | 2023-02-09 | Amicus Therapeutics, Inc. | Recombinant CDKL5 Proteins, Gene Therapy and Production Methods |
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2021
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| CA3200192A1 (en) | 2022-06-09 |
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| EP4255500A1 (en) | 2023-10-11 |
| AU2021392642A1 (en) | 2023-06-22 |
| US20230414785A1 (en) | 2023-12-28 |
| JP2023551911A (ja) | 2023-12-13 |
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