JP2023545433A5 - - Google Patents
Info
- Publication number
- JP2023545433A5 JP2023545433A5 JP2023521706A JP2023521706A JP2023545433A5 JP 2023545433 A5 JP2023545433 A5 JP 2023545433A5 JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023521706 A JP2023521706 A JP 2023521706A JP 2023545433 A5 JP2023545433 A5 JP 2023545433A5
- Authority
- JP
- Japan
- Prior art keywords
- expression cassette
- sequence
- hgla
- raav
- seq
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063089850P | 2020-10-09 | 2020-10-09 | |
| US63/089,850 | 2020-10-09 | ||
| US202163146286P | 2021-02-05 | 2021-02-05 | |
| US63/146,286 | 2021-02-05 | ||
| US202163186092P | 2021-05-08 | 2021-05-08 | |
| US63/186,092 | 2021-05-08 | ||
| PCT/US2021/054145 WO2022076803A1 (en) | 2020-10-09 | 2021-10-08 | Compositions and methods for treatment of fabry disease |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023545433A JP2023545433A (ja) | 2023-10-30 |
| JP2023545433A5 true JP2023545433A5 (https=) | 2024-10-10 |
Family
ID=81125461
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023521706A Pending JP2023545433A (ja) | 2020-10-09 | 2021-10-08 | ファブリー病の治療のための組成物及び方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230365955A1 (https=) |
| EP (1) | EP4225906A1 (https=) |
| JP (1) | JP2023545433A (https=) |
| KR (1) | KR20230118075A (https=) |
| AU (1) | AU2021356684A1 (https=) |
| CA (1) | CA3193833A1 (https=) |
| TW (1) | TW202229560A (https=) |
| WO (1) | WO2022076803A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR124981A1 (es) * | 2021-02-26 | 2023-05-24 | Takeda Pharmaceuticals Co | Composición y métodos para el tratamiento de la enfermedad de fabry |
| US20240408157A1 (en) * | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| EP4551711A1 (en) * | 2022-07-08 | 2025-05-14 | Cornell University | Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
| AU2023331251A1 (en) * | 2022-08-25 | 2025-02-27 | Takeda Pharmaceutical Company Limited | Composition for use in the treatment of fabry disease |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5786464C1 (en) | 1994-09-19 | 2012-04-24 | Gen Hospital Corp | Overexpression of mammalian and viral proteins |
| US6136597A (en) | 1997-09-18 | 2000-10-24 | The Salk Institute For Biological Studies | RNA export element |
| CA2379166C (en) | 1999-08-09 | 2013-03-26 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs |
| US7419829B2 (en) | 2000-10-06 | 2008-09-02 | Oxford Biomedica (Uk) Limited | Vector system |
| 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 |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| 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 |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| ES2528494T3 (es) | 2009-09-03 | 2015-02-10 | Evonik Röhm Gmbh | Forma de dosificación oral que comprende al menos un agente biológicamente activo, sustancias auxiliares de formulación y partículas magnetizables |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US20170051267A1 (en) * | 2015-08-20 | 2017-02-23 | Research Foundation Of The City University Of New York | Novel alpha-galactosidase a derivatives |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| WO2018046774A1 (en) | 2016-09-12 | 2018-03-15 | Genethon | Acid-alpha glucosidase variants and uses thereof |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| US12070702B2 (en) | 2018-06-14 | 2024-08-27 | Regenxbio Inc. | Anion exchange chromatography for recombinant AAV production |
| 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 |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| 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 |
| WO2021158915A1 (en) | 2020-02-06 | 2021-08-12 | Precision Biosciences, Inc. | Recombinant adeno-associated virus compositions and methods for producing and using the same |
| EP3868886A1 (en) | 2020-02-21 | 2021-08-25 | Bia Separations D.O.O. | A method for separation or depletion of empty aav capsids from full aav capsids |
| EP4103211A4 (en) * | 2020-04-27 | 2023-08-23 | 4D Molecular Therapeutics Inc. | CODON-OPTIMISED GLA GENES AND THEIR USES |
-
2021
- 2021-10-08 AU AU2021356684A patent/AU2021356684A1/en not_active Abandoned
- 2021-10-08 JP JP2023521706A patent/JP2023545433A/ja active Pending
- 2021-10-08 TW TW110137528A patent/TW202229560A/zh unknown
- 2021-10-08 CA CA3193833A patent/CA3193833A1/en active Pending
- 2021-10-08 EP EP21805710.7A patent/EP4225906A1/en active Pending
- 2021-10-08 US US18/246,872 patent/US20230365955A1/en active Pending
- 2021-10-08 WO PCT/US2021/054145 patent/WO2022076803A1/en not_active Ceased
- 2021-10-08 KR KR1020237015206A patent/KR20230118075A/ko not_active Withdrawn
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