JPWO2020142653A5 - - Google Patents
Download PDFInfo
- Publication number
- 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 (fr) | 2019-01-04 | 2020-01-03 | Constructions de thérapie génique pour le traitement de la maladie de wilson |
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 (fr) | 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 (18)
| Country | Link |
|---|---|
| US (1) | US20220090131A1 (fr) |
| EP (2) | EP3906066B1 (fr) |
| JP (2) | JP2022516283A (fr) |
| KR (1) | KR20210112339A (fr) |
| CN (1) | CN113518628A (fr) |
| AU (1) | AU2020204679A1 (fr) |
| BR (1) | BR112021013140A2 (fr) |
| CA (1) | CA3124880A1 (fr) |
| CO (1) | CO2021010088A2 (fr) |
| DK (1) | DK3906066T3 (fr) |
| ES (1) | ES2970216T3 (fr) |
| FI (1) | FI3906066T3 (fr) |
| HR (1) | HRP20231750T1 (fr) |
| IL (1) | IL284554A (fr) |
| MX (1) | MX2021008135A (fr) |
| PT (1) | PT3906066T (fr) |
| SG (1) | SG11202106855YA (fr) |
| WO (1) | WO2020142653A1 (fr) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112019013245A2 (pt) | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| WO2019195449A1 (fr) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Vecteurs de virus évitant les anticorps |
| MX2021011468A (es) | 2019-03-21 | 2021-12-15 | Vectores de virus adenoasociados recombinantes. | |
| CA3140049A1 (fr) * | 2019-05-14 | 2020-11-19 | Duke University | Compositions et procedes pour le traitement de maladies mediees par l'atpase |
| WO2021076925A1 (fr) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Vecteurs de virus adéno-associés pour le traitement de la maladie de niemann-pick de type 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 |
| WO2023108130A2 (fr) * | 2021-12-10 | 2023-06-15 | Aavantibio, Inc. | Compositions comprenant des séquences kozak sélectionnées pour produire une expression améliorée |
| AU2022407655A1 (en) * | 2021-12-10 | 2024-06-20 | Aavantibio, Inc. | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector |
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 |
| SG157224A1 (en) | 2001-11-13 | 2009-12-29 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP1453547B1 (fr) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Sequences du serotype 8 du virus associe a l'adenovirus (aav), vecteurs les contenant et utilisations correspondantes |
| EP2281877A3 (fr) | 2003-05-21 | 2011-06-01 | Genzyme Corporation | Procedes de production de preparations de virions aav recombinants sensiblement exemptes de capsides vides |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| DK2359867T3 (en) | 2005-04-07 | 2015-01-05 | Univ Pennsylvania | A method for increasing an AAV vector function |
| EP2242840B1 (fr) | 2008-01-29 | 2019-07-24 | Applied Genetic Technologies Corporation | Production de virus adeno associe recombinants à l'aide de cellules bhk en suspension |
| WO2010114948A2 (fr) | 2009-04-02 | 2010-10-07 | University Of Florida Research Foundation, Inc. | Système inductible de production à haut rendement de vecteurs recombinants dérivés de virus adéno-associés (raav) |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| IN2012DN06629A (fr) | 2010-01-28 | 2015-10-23 | Philadelphia Children Hospital | |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| DK3137497T3 (da) * | 2014-05-02 | 2021-07-12 | Genzyme Corp | Aav-vektorer til retinal- og cns-genterapi |
| WO2016049230A1 (fr) | 2014-09-24 | 2016-03-31 | City Of Hope | Variants de vecteur de virus adénoassocié pour une édition de haute efficacité du génome et procédés correspondants |
| PL3233130T3 (pl) * | 2014-12-17 | 2021-11-22 | Fundacion Para La Investigacion Medica Aplicada | Konstrukty kwasów nukleinowych i wektory do terapii genowej do zastosowania w leczeniu choroby wilsona |
| BR112019013245A2 (pt) * | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| WO2023230466A1 (fr) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions et méthodes pour le traitement de la maladie de wilson |
-
2020
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/fr active Active
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 AU AU2020204679A patent/AU2020204679A1/en active Pending
- 2020-01-03 US US17/417,619 patent/US20220090131A1/en active Pending
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko unknown
- 2020-01-03 JP JP2021538754A patent/JP2022516283A/ja active Pending
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
- 2020-01-03 EP EP23196931.2A patent/EP4269579A3/fr active Pending
- 2020-01-03 DK DK20704633.5T patent/DK3906066T3/da active
- 2020-01-03 CA CA3124880A patent/CA3124880A1/fr active Pending
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 CN CN202080007863.5A patent/CN113518628A/zh active Pending
- 2020-01-03 WO PCT/US2020/012131 patent/WO2020142653A1/fr unknown
-
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
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| HRP20231750T1 (hr) | Konstrukti genske terapije za liječenje wilsonove bolesti | |
| EP3313991B1 (fr) | Facteur ix modifié, et compositions, méthodes et utilisations pour un transfert de gènes dans des cellules, des organes et des tissus | |
| JP2021087431A5 (fr) | ||
| JP2020514286A5 (fr) | ||
| JP2020519292A5 (fr) | ||
| JP2023179414A (ja) | アフェレーシスの方法及び使用 | |
| JPWO2020142653A5 (fr) | ||
| RU2019139555A (ru) | Генная терапия нейрональных цероидных липофусцинозов | |
| JP2023076698A (ja) | ２１－ヒドロキシラーゼ欠損症のためのアデノ随伴ウイルス遺伝子療法 | |
| US20230175015A1 (en) | Immunosuppressive agents and viral delivery re-dosing methods for gene therapy | |
| JPWO2020106916A5 (fr) | ||
| KR20210153069A (ko) | 섬유아세포 성장 인자 23 관련 저인산혈증성 질환의 유전자 요법 | |
| JPWO2020069461A5 (fr) | ||
| RU2021123108A (ru) | Генотерапевтические конструкции для лечения болезни вильсона | |
| JPWO2020072451A5 (fr) | ||
| AU2022407655A1 (en) | Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector | |
| JPWO2021163322A5 (fr) | ||
| JPWO2020033473A5 (fr) | ||
| US20230043051A1 (en) | Adeno-associated virus vectors based gene therapy for hereditary angioedema | |
| JPWO2021021661A5 (fr) | ||
| JPWO2021202532A5 (fr) | ||
| RU2815545C2 (ru) | Генная терапия гипофосфатемических заболеваний, связанных с фактором роста фибробластов 23 | |
| JPWO2020172490A5 (fr) | ||
| JPWO2021108809A5 (fr) | ||
| JPWO2021076925A5 (fr) |