JPWO2020142653A5 - - Google Patents
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- JPWO2020142653A5 JPWO2020142653A5 JP2021538754A JP2021538754A JPWO2020142653A5 JP WO2020142653 A5 JPWO2020142653 A5 JP WO2020142653A5 JP 2021538754 A JP2021538754 A JP 2021538754A JP 2021538754 A JP2021538754 A JP 2021538754A JP WO2020142653 A5 JPWO2020142653 A5 JP WO2020142653A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence
- raav
- item
- aav
- nucleic 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.)
- Pending
Links
- 150000007523 nucleic acids Chemical class 0.000 claims description 55
- 108020004707 nucleic acids Proteins 0.000 claims description 41
- 239000003623 enhancer Substances 0.000 claims description 33
- 230000002708 enhancing Effects 0.000 claims description 33
- 229920002459 Intron Polymers 0.000 claims description 30
- 230000001402 polyadenylating Effects 0.000 claims description 27
- 229920001850 Nucleic acid sequence Polymers 0.000 claims description 14
- 101700032974 ATP7B Proteins 0.000 claims description 12
- 102100019961 ATP7B Human genes 0.000 claims description 12
- 102000007584 Prealbumin Human genes 0.000 claims description 11
- 108010071690 Prealbumin Proteins 0.000 claims description 11
- 102000034451 ATPases Human genes 0.000 claims description 10
- 108091006096 ATPases Proteins 0.000 claims description 10
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 10
- 210000000234 Capsid Anatomy 0.000 claims description 10
- 108010006025 bovine growth hormone Proteins 0.000 claims description 10
- RYGMFSIKBFXOCR-UHFFFAOYSA-N copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 claims description 10
- 229910052802 copper Inorganic materials 0.000 claims description 10
- 239000010949 copper Substances 0.000 claims description 10
- 101700033661 ACTB Proteins 0.000 claims description 6
- 102100011550 ACTB Human genes 0.000 claims description 6
- 101710032514 ACTI Proteins 0.000 claims description 6
- 102000007592 Apolipoproteins Human genes 0.000 claims description 6
- 108010071619 Apolipoproteins Proteins 0.000 claims description 6
- 241000701022 Cytomegalovirus Species 0.000 claims description 6
- 241000287828 Gallus gallus Species 0.000 claims description 6
- 102100019126 HBB Human genes 0.000 claims description 6
- 108091005902 Hemoglobin subunit beta Proteins 0.000 claims description 6
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 6
- 102100011516 SERPINA1 Human genes 0.000 claims description 6
- 102100005424 SERPINA7 Human genes 0.000 claims description 6
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims description 6
- MTCFGRXMJLQNBG-UWTATZPHSA-N D-serine Chemical compound OC[C@@H](N)C(O)=O MTCFGRXMJLQNBG-UWTATZPHSA-N 0.000 claims description 4
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims description 4
- 102100008692 MBD3 Human genes 0.000 claims description 4
- 101700079069 MBD3 Proteins 0.000 claims description 4
- 102100008690 MBD4 Human genes 0.000 claims description 4
- 101700060039 MBD4 Proteins 0.000 claims description 4
- 201000001203 Wilson disease Diseases 0.000 claims description 4
- 239000002184 metal Substances 0.000 claims description 4
- 229910052751 metal Inorganic materials 0.000 claims description 4
- 125000003616 serine group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 claims description 4
- 241000432074 Adeno-associated virus Species 0.000 claims description 3
- 102000006734 Beta-Globulins Human genes 0.000 claims description 3
- 108010087504 Beta-Globulins Proteins 0.000 claims description 3
- 101710009148 SERPINA1 Proteins 0.000 claims description 3
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims description 3
- 229940024142 alpha 1-Antitrypsin Drugs 0.000 claims description 3
- 108091006028 chimera Proteins 0.000 claims description 3
- 238000011144 upstream manufacturing Methods 0.000 claims description 3
- 101000059197 ATP7B Proteins 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 102000031095 human ATP7B protein Human genes 0.000 claims description 2
- 230000032258 transport Effects 0.000 claims 2
- 238000004113 cell culture Methods 0.000 description 2
- 210000004027 cells Anatomy 0.000 description 2
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 238000003306 harvesting Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000006011 modification reaction Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024031460A JP2024054426A (ja) | 2019-01-04 | 2024-03-01 | ウイルソン病を処置するための遺伝子治療構築物 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962788324P | 2019-01-04 | 2019-01-04 | |
| US62/788,324 | 2019-01-04 | ||
| US201962834830P | 2019-04-16 | 2019-04-16 | |
| US62/834,830 | 2019-04-16 | ||
| PCT/US2020/012131 WO2020142653A1 (en) | 2019-01-04 | 2020-01-03 | Gene therapy constructs for treating wilson disease |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024031460A Division JP2024054426A (ja) | 2019-01-04 | 2024-03-01 | ウイルソン病を処置するための遺伝子治療構築物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022516283A JP2022516283A (ja) | 2022-02-25 |
| JPWO2020142653A5 true JPWO2020142653A5 (es) | 2023-01-19 |
Family
ID=69528954
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538754A Pending JP2022516283A (ja) | 2019-01-04 | 2020-01-03 | ウイルソン病を処置するための遺伝子治療構築物 |
| JP2024031460A Pending JP2024054426A (ja) | 2019-01-04 | 2024-03-01 | ウイルソン病を処置するための遺伝子治療構築物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024031460A Pending JP2024054426A (ja) | 2019-01-04 | 2024-03-01 | ウイルソン病を処置するための遺伝子治療構築物 |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20220090131A1 (es) |
| EP (2) | EP4269579A3 (es) |
| JP (2) | JP2022516283A (es) |
| KR (1) | KR20210112339A (es) |
| CN (1) | CN113518628A (es) |
| AU (1) | AU2020204679A1 (es) |
| BR (1) | BR112021013140A2 (es) |
| CA (1) | CA3124880A1 (es) |
| CO (1) | CO2021010088A2 (es) |
| DK (1) | DK3906066T3 (es) |
| ES (1) | ES2970216T3 (es) |
| FI (1) | FI3906066T3 (es) |
| HR (1) | HRP20231750T1 (es) |
| IL (1) | IL284554A (es) |
| MX (1) | MX2021008135A (es) |
| PT (1) | PT3906066T (es) |
| SG (1) | SG11202106855YA (es) |
| TW (1) | TW202043479A (es) |
| WO (1) | WO2020142653A1 (es) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11473106B2 (en) | 2016-12-30 | 2022-10-18 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating Wilson's disease |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
| AU2020274200A1 (en) * | 2019-05-14 | 2021-11-25 | Duke University | Compositions and methods for the treatment of ATPase-mediated diseases |
| BR112022006530A2 (pt) | 2019-10-17 | 2022-07-05 | Stridebio Inc | Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c |
| CN115216481A (zh) * | 2021-04-21 | 2022-10-21 | 四川至善唯新生物科技有限公司 | 一种表达量提高的截短型atp7b基因及其应用 |
| CN113694218A (zh) * | 2021-08-30 | 2021-11-26 | 昆明理工大学 | 一种atp7b基因p992l突变的基因修复治疗载体 |
| WO2023108159A1 (en) * | 2021-12-10 | 2023-06-15 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector |
| WO2023108130A2 (en) * | 2021-12-10 | 2023-06-15 | Aavantibio, Inc. | Compositions comprising kozak sequences selected for enhanced expression |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6759050B1 (en) | 1998-12-03 | 2004-07-06 | Avigen, Inc. | Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith |
| NZ600121A (en) | 2001-11-13 | 2013-12-20 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| AU2002360291A1 (en) | 2001-12-17 | 2003-06-30 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| ES2521682T3 (es) | 2003-05-21 | 2014-11-13 | Genzyme Corporation | Procedimientos para producir preparaciones de viriones de AAV recombinantes sustancialmente exentas de cápsidas vacías |
| JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
| JP5702519B2 (ja) | 2005-04-07 | 2015-04-15 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ａａｖベクターの機能を上げる方法 |
| CA2713338C (en) | 2008-01-29 | 2021-10-26 | Applied Genetic Technologies Corporation | Recombinant virus production using mammalian cells in suspension |
| WO2010114948A2 (en) | 2009-04-02 | 2010-10-07 | University Of Florida Research Foundation, Inc. | An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| MX340102B (es) | 2010-01-28 | 2016-06-24 | The Children's Hospital Of Philadelphia * | Una plataforma de fabricacion ajustable en escala, para la purificacion de vector viral y vectores virales asi purificados para el uso en terapia de genes. |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| IL292951B1 (en) * | 2014-05-02 | 2024-06-01 | Genzyme Corp | AAV vectors for gene therapy in the central nervous system and retina |
| PT3198018T (pt) | 2014-09-24 | 2021-02-24 | Hope City | Variantes dos vetores de vírus adenoassociados para edição do genoma de alta eficácia e métodos da mesma |
| KR102526616B1 (ko) * | 2014-12-17 | 2023-04-27 | 푼다시온 파라 라 인베스티가시온 메디카 아플리카다 | 윌슨병 및 기타 병태의 치료에 사용되기 위한 핵산 구조물 및 유전자 치료용 벡터 |
| US11473106B2 (en) * | 2016-12-30 | 2022-10-18 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating Wilson's disease |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
-
2020
- 2020-01-03 EP EP23196931.2A patent/EP4269579A3/en active Pending
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 US US17/417,619 patent/US20220090131A1/en active Pending
- 2020-01-03 CA CA3124880A patent/CA3124880A1/en active Pending
- 2020-01-03 DK DK20704633.5T patent/DK3906066T3/da active
- 2020-01-03 CN CN202080007863.5A patent/CN113518628A/zh active Pending
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko unknown
- 2020-01-03 WO PCT/US2020/012131 patent/WO2020142653A1/en unknown
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 JP JP2021538754A patent/JP2022516283A/ja active Pending
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
- 2020-01-03 AU AU2020204679A patent/AU2020204679A1/en active Pending
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/en active Active
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 TW TW109100147A patent/TW202043479A/zh unknown
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
-
2021
- 2021-07-01 IL IL284554A patent/IL284554A/en unknown
- 2021-07-29 CO CONC2021/0010088A patent/CO2021010088A2/es unknown
-
2024
- 2024-03-01 JP JP2024031460A patent/JP2024054426A/ja active Pending
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