IL305440A - Preparation and methods for treating Fabry disease - Google Patents
Preparation and methods for treating Fabry diseaseInfo
- Publication number
- IL305440A IL305440A IL305440A IL30544023A IL305440A IL 305440 A IL305440 A IL 305440A IL 305440 A IL305440 A IL 305440A IL 30544023 A IL30544023 A IL 30544023A IL 305440 A IL305440 A IL 305440A
- Authority
- IL
- Israel
- Prior art keywords
- vector
- itr
- seq
- raav
- gal
- Prior art date
Links
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- 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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- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
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- Organic Chemistry (AREA)
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- Biotechnology (AREA)
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- General Engineering & Computer Science (AREA)
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- Public Health (AREA)
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- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
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- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
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- Diabetes (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
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| US202163154485P | 2021-02-26 | 2021-02-26 | |
| PCT/US2022/017998 WO2022183052A1 (en) | 2021-02-26 | 2022-02-25 | Composition and methods for the treatment of fabry disease |
Publications (1)
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| IL305440A true IL305440A (en) | 2023-10-01 |
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL305440A IL305440A (en) | 2021-02-26 | 2022-02-25 | Preparation and methods for treating Fabry disease |
Country Status (17)
| Country | Link |
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| US (1) | US20240175054A1 (https=) |
| EP (1) | EP4298227A1 (https=) |
| JP (1) | JP2024509792A (https=) |
| KR (1) | KR20230150836A (https=) |
| CN (1) | CN117321212A (https=) |
| AR (1) | AR124981A1 (https=) |
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| BR (1) | BR112023016983A2 (https=) |
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| PE (1) | PE20240806A1 (https=) |
| TW (1) | TW202302855A (https=) |
| WO (1) | WO2022183052A1 (https=) |
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| AU2023331251A1 (en) * | 2022-08-25 | 2025-02-27 | Takeda Pharmaceutical Company Limited | Composition for use in the treatment of fabry disease |
| AU2024257162A1 (en) * | 2023-04-17 | 2025-11-06 | Exegenesis Bio Singapore Pte. Ltd. | Compositions and methods for gene therapies |
| WO2024236544A1 (en) | 2023-05-17 | 2024-11-21 | Takeda Pharmaceutical Company Limited | Pet tracer compounds for tracing alpha-galactosidase a and reaction intermediates, and methods of use |
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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 |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The 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 |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| 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 |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| 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 |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| WO2019222314A1 (en) * | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a |
| CN113631182B (zh) * | 2018-10-10 | 2025-03-21 | 阿米库斯治疗学公司 | 二硫键稳定的多肽组合物和使用方法 |
| KR20220022107A (ko) * | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| AU2019403323B2 (en) * | 2018-12-20 | 2026-02-26 | Codexis, Inc. | Human alpha-galactosidase variants |
| CN113423419A (zh) * | 2019-01-04 | 2021-09-21 | 桑格摩生物治疗股份有限公司 | 用于治疗法布里病的方法和组合物 |
| AU2021356684A1 (en) * | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
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| PE20240806A1 (es) | 2024-04-18 |
| BR112023016983A2 (pt) | 2023-11-07 |
| KR20230150836A (ko) | 2023-10-31 |
| CL2023002530A1 (es) | 2024-04-19 |
| CA3209673A1 (en) | 2022-09-01 |
| MX2023009978A (es) | 2023-09-06 |
| TW202302855A (zh) | 2023-01-16 |
| AR124981A1 (es) | 2023-05-24 |
| CO2023011012A2 (es) | 2023-08-28 |
| EP4298227A1 (en) | 2024-01-03 |
| JP2024509792A (ja) | 2024-03-05 |
| CN117321212A (zh) | 2023-12-29 |
| WO2022183052A1 (en) | 2022-09-01 |
| ECSP23072174A (es) | 2023-10-31 |
| US20240175054A1 (en) | 2024-05-30 |
| AU2022227017A1 (en) | 2023-09-07 |
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