JP2022512589A5 - - Google Patents
Info
- Publication number
- JP2022512589A5 JP2022512589A5 JP2021518190A JP2021518190A JP2022512589A5 JP 2022512589 A5 JP2022512589 A5 JP 2022512589A5 JP 2021518190 A JP2021518190 A JP 2021518190A JP 2021518190 A JP2021518190 A JP 2021518190A JP 2022512589 A5 JP2022512589 A5 JP 2022512589A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- seq
- pharmaceutical composition
- acid sequence
- amino acid
- 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.)
- Granted
Links
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024125570A JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862739811P | 2018-10-01 | 2018-10-01 | |
| US62/739,811 | 2018-10-01 | ||
| US201962835178P | 2019-04-17 | 2019-04-17 | |
| US62/835,178 | 2019-04-17 | ||
| PCT/US2019/053797 WO2020072354A1 (en) | 2018-10-01 | 2019-09-30 | Compositions useful for treating gm1 gangliosidosis |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024125570A Division JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022512589A JP2022512589A (ja) | 2022-02-07 |
| JP2022512589A5 true JP2022512589A5 (https=) | 2022-10-06 |
| JP7534290B2 JP7534290B2 (ja) | 2024-08-14 |
Family
ID=69165478
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021518190A Active JP7534290B2 (ja) | 2018-10-01 | 2019-09-30 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
| JP2024125570A Pending JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024125570A Pending JP2024161415A (ja) | 2018-10-01 | 2024-08-01 | Ｇｍ１ガングリオシドーシスの治療に有用な組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US12594327B2 (https=) |
| EP (1) | EP3860659A1 (https=) |
| JP (2) | JP7534290B2 (https=) |
| KR (1) | KR20210071017A (https=) |
| CN (1) | CN113438954A (https=) |
| AU (1) | AU2019351815A1 (https=) |
| BR (1) | BR112021006060A2 (https=) |
| CA (1) | CA3114175A1 (https=) |
| CL (1) | CL2021000798A1 (https=) |
| CO (1) | CO2021005443A2 (https=) |
| IL (1) | IL281923A (https=) |
| MX (1) | MX2021003869A (https=) |
| PE (1) | PE20211596A1 (https=) |
| SA (1) | SA521421639B1 (https=) |
| SG (1) | SG11202102942VA (https=) |
| WO (1) | WO2020072354A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| KR20220145838A (ko) * | 2020-02-02 | 2022-10-31 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오사이드증을 치료하는 데 유용한 조성물 |
| CA3177869A1 (en) * | 2020-05-05 | 2021-11-11 | Duke University | Cross-species compatible adeno-associated virus compositions and methods of use thereof |
| CN116926047A (zh) * | 2022-03-31 | 2023-10-24 | 北京锦篮基因科技有限公司 | 功能性β-半乳糖苷酶变体、AAV介导的人β-半乳糖苷酶表达载体及其用途 |
| WO2024040258A2 (en) * | 2022-08-19 | 2024-02-22 | Codexis, Inc. | Engineered beta- galactosidase polypeptides |
| CN120035662A (zh) * | 2022-10-12 | 2025-05-23 | 新型医药公司 | 用于治疗、预防或改善GM1神经节苷脂沉积症或Morquio综合征B的药物组合物及其施用方法 |
Family Cites Families (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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. |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | 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 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| ES2635726T3 (es) * | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| 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 |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| WO2013151665A2 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins associated with human disease |
| 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 |
| CN115120746A (zh) | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| AU2015320694B2 (en) | 2014-09-24 | 2021-11-11 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3285788B1 (en) | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| MX2018004755A (es) | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| US20190060359A1 (en) | 2015-11-02 | 2019-02-28 | Neuraltus Pharmaceuticals, Inc. | Treatment of neurodegenerative disease with sodium chlorite |
| 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 |
| EP3411484B1 (en) | 2016-02-05 | 2023-10-04 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| JP7610923B2 (ja) | 2016-09-02 | 2025-01-09 | スパーク セラピューティクス インコーポレイテッド | Ｃｎｓ障害を治療するための方法及びベクター |
| JP2020500928A (ja) | 2016-11-15 | 2020-01-16 | リジェネクスバイオ インコーポレイテッド | Ｍｐｓｉおよびｍｐｓｉｉならびに他の神経障害において神経機能を改善するための方法 |
| PT3589730T (pt) * | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| WO2019222314A1 (en) | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a |
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| KR20220022107A (ko) | 2018-12-05 | 2022-02-24 | 아베오나 테라퓨틱스 인코퍼레이티드 | 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터 |
| KR20220145838A (ko) | 2020-02-02 | 2022-10-31 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오사이드증을 치료하는 데 유용한 조성물 |
-
2019
- 2019-09-30 CA CA3114175A patent/CA3114175A1/en active Pending
- 2019-09-30 US US17/281,378 patent/US12594327B2/en active Active
- 2019-09-30 AU AU2019351815A patent/AU2019351815A1/en active Pending
- 2019-09-30 BR BR112021006060-0A patent/BR112021006060A2/pt unknown
- 2019-09-30 SG SG11202102942VA patent/SG11202102942VA/en unknown
- 2019-09-30 CN CN201980079543.8A patent/CN113438954A/zh active Pending
- 2019-09-30 WO PCT/US2019/053797 patent/WO2020072354A1/en not_active Ceased
- 2019-09-30 KR KR1020217012705A patent/KR20210071017A/ko active Pending
- 2019-09-30 EP EP19836625.4A patent/EP3860659A1/en active Pending
- 2019-09-30 PE PE2021000431A patent/PE20211596A1/es unknown
- 2019-09-30 MX MX2021003869A patent/MX2021003869A/es unknown
- 2019-09-30 JP JP2021518190A patent/JP7534290B2/ja active Active
-
2021
- 2021-03-29 CL CL2021000798A patent/CL2021000798A1/es unknown
- 2021-03-31 IL IL281923A patent/IL281923A/en unknown
- 2021-04-01 SA SA521421639A patent/SA521421639B1/ar unknown
- 2021-04-27 CO CONC2021/0005443A patent/CO2021005443A2/es unknown
-
2024
- 2024-08-01 JP JP2024125570A patent/JP2024161415A/ja active Pending
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