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.)
- Granted
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- 150000007523 nucleic acids Chemical class 0.000 claims description 55
- 108020004707 nucleic acids Proteins 0.000 claims description 41
- 102000039446 nucleic acids Human genes 0.000 claims description 41
- 108010076504 Protein Sorting Signals Proteins 0.000 claims description 27
- 230000008488 polyadenylation Effects 0.000 claims description 27
- 239000003623 enhancer Substances 0.000 claims description 21
- 239000013598 vector Substances 0.000 claims description 21
- 241000702421 Dependoparvovirus Species 0.000 claims description 15
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 claims description 14
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 14
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 claims description 12
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 12
- 108010071690 Prealbumin Proteins 0.000 claims description 11
- 102000009190 Transthyretin Human genes 0.000 claims description 11
- 108091006112 ATPases Proteins 0.000 claims description 10
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 10
- 102000057290 Adenosine Triphosphatases Human genes 0.000 claims description 10
- 108010006025 bovine growth hormone Proteins 0.000 claims description 10
- 210000000234 capsid Anatomy 0.000 claims description 10
- 101000727865 Homo sapiens Copper transport protein ATOX1 Proteins 0.000 claims description 8
- 102000051131 human ATOX1 Human genes 0.000 claims description 8
- 102100022712 Alpha-1-antitrypsin Human genes 0.000 claims description 6
- 108010071619 Apolipoproteins Proteins 0.000 claims description 6
- 102000007592 Apolipoproteins Human genes 0.000 claims description 6
- 241000701022 Cytomegalovirus Species 0.000 claims description 6
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 claims description 6
- 108700002232 Immediate-Early Genes Proteins 0.000 claims description 6
- 241000283973 Oryctolagus cuniculus Species 0.000 claims description 6
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims description 6
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims description 6
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims description 6
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims description 4
- 101000615495 Homo sapiens Methyl-CpG-binding domain protein 3 Proteins 0.000 claims description 4
- 101000615492 Homo sapiens Methyl-CpG-binding domain protein 4 Proteins 0.000 claims description 4
- 102100021291 Methyl-CpG-binding domain protein 3 Human genes 0.000 claims description 4
- 102100021290 Methyl-CpG-binding domain protein 4 Human genes 0.000 claims description 4
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims description 4
- 208000018839 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
- 125000003607 serino group Chemical group [H]N([H])[C@]([H])(C(=O)[*])C(O[H])([H])[H] 0.000 claims description 4
- 102000006734 Beta-Globulins Human genes 0.000 claims description 3
- 108010087504 Beta-Globulins Proteins 0.000 claims description 3
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 3
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 3
- 101000899111 Homo sapiens Hemoglobin subunit beta 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
- 238000011144 upstream manufacturing Methods 0.000 claims description 3
- RYGMFSIKBFXOCR-UHFFFAOYSA-N Copper Chemical compound [Cu] RYGMFSIKBFXOCR-UHFFFAOYSA-N 0.000 claims description 2
- 229910052802 copper Inorganic materials 0.000 claims description 2
- 239000010949 copper Substances 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 102000050677 human ATP7B Human genes 0.000 claims description 2
- 230000032258 transport Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 14
- 241000649044 Adeno-associated virus 9 Species 0.000 description 4
- 210000004027 cell Anatomy 0.000 description 2
- 238000004113 cell culture Methods 0.000 description 2
- 239000013607 AAV vector Substances 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 210000003527 eukaryotic cell Anatomy 0.000 description 1
- 238000003306 harvesting Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024031460A JP7746437B2 (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 JP7746437B2 (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 (4)
| Publication Number | Publication Date |
|---|---|
| JP2022516283A JP2022516283A (ja) | 2022-02-25 |
| JP2022516283A5 JP2022516283A5 (https=) | 2023-01-19 |
| JPWO2020142653A5 true JPWO2020142653A5 (https=) | 2023-01-19 |
| JP7531495B2 JP7531495B2 (ja) | 2024-08-09 |
Family
ID=69528954
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538754A Active JP7531495B2 (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 Active JP7746437B2 (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 Active JP7746437B2 (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 (22)
| Country | Link |
|---|---|
| US (2) | US12338450B2 (https=) |
| EP (2) | EP4269579B1 (https=) |
| JP (2) | JP7531495B2 (https=) |
| KR (1) | KR20210112339A (https=) |
| CN (1) | CN113518628B (https=) |
| AU (1) | AU2020204679B2 (https=) |
| BR (1) | BR112021013140A2 (https=) |
| CA (1) | CA3124880A1 (https=) |
| CO (1) | CO2021010088A2 (https=) |
| DK (1) | DK3906066T5 (https=) |
| ES (1) | ES2970216T3 (https=) |
| FI (1) | FI3906066T3 (https=) |
| HR (1) | HRP20231750T1 (https=) |
| HU (1) | HUE065782T2 (https=) |
| IL (1) | IL284554A (https=) |
| MX (1) | MX2021008135A (https=) |
| PL (1) | PL3906066T3 (https=) |
| PT (1) | PT3906066T (https=) |
| SG (1) | SG11202106855YA (https=) |
| TW (1) | TWI848038B (https=) |
| WO (1) | WO2020142653A1 (https=) |
| ZA (1) | ZA202104515B (https=) |
Families Citing this family (17)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| CN114072514A (zh) | 2019-05-14 | 2022-02-18 | 杜克大学 | 用于治疗atp酶介导的疾病的组合物和方法 |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| 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 |
| CA3240197A1 (en) * | 2021-12-10 | 2025-02-26 | Aavantibio, Inc. | Compositions comprising kozak sequences selected for enhanced expression |
| KR20240118855A (ko) * | 2021-12-10 | 2024-08-05 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물 |
| WO2024183769A1 (en) * | 2023-03-06 | 2024-09-12 | Lingyi Biotech Co., Ltd. | A truncated atp7b and the use thereof |
| JP2026508707A (ja) * | 2023-03-22 | 2026-03-11 | スカイライン、セラピューティクス、リミテッド | ウィルソン病の遺伝子療法のための組換えａａｖ |
| WO2025002226A1 (zh) * | 2023-06-28 | 2025-01-02 | 迈威(上海)生物科技股份有限公司 | 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用 |
| WO2025026198A1 (en) * | 2023-07-28 | 2025-02-06 | Lingyi Biotech Co., Ltd. | Polynucleotides for the treatment of wilson disease |
Family Cites Families (30)
| 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 |
| US7985553B2 (en) * | 2001-10-29 | 2011-07-26 | Nathaniel Heintz | Method for isolating cell type-specific mRNAs |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DE10156121A1 (de) | 2001-11-16 | 2003-05-28 | Wolfgang Stremmel | Somatische Gentherapie mit Vektoren, die einen verkürzten metallsensitiven Promotor besitzen, zur Behandlung des Morbus Wilson sowie anderer Erkrankungen, die mit Veränderungen im Metallstoffwechsel einhergehen, wie z.B. Alzheimer-Erkrankung |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| DE602004030327D1 (de) | 2003-05-21 | 2011-01-13 | Genzyme Corp | Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| DK2242840T3 (da) | 2008-01-29 | 2019-10-21 | Applied Genetic Tech Corporation | Produktion af rekombinant adeno-associeret virus under anvendelse af bhk-celler i suspension |
| US8679837B2 (en) | 2009-04-02 | 2014-03-25 | University Of Florida Research Foundation, Inc. | 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 |
| DK2529020T3 (en) | 2010-01-28 | 2018-08-06 | Childrens Hospital Philadelphia | SCALABLE PREPARATION PLATF FOR CLEANING VIRAL VECTORS AND CLEANED VIRAL VECTORS FOR USE IN GENTHERAPY |
| US20130023033A1 (en) | 2010-03-29 | 2013-01-24 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | 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 |
| AU2013221212B2 (en) * | 2012-02-17 | 2018-08-09 | The Children's Hospital Of Philadelphia | AAV vector compositions and methods for gene transfer to cells, organs and tissues |
| AU2013243948A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins associated with human disease |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| IL248102B (en) * | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| BR112017005892A2 (pt) | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| ES2876412T3 (es) | 2014-12-17 | 2021-11-12 | Fundacion Para La Investig Medica Aplicada | Construcciones de ácido nucleico y vectores de terapia génica para su uso en el tratamiento de la enfermedad de Wilson |
| EP3233129B1 (en) | 2014-12-17 | 2020-01-15 | Fundacion para la Investigacion Medica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of wilson's disease and other conditions |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| WO2017100676A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| TW202039854A (zh) | 2018-11-16 | 2020-11-01 | 美商編碼製藥公司 | 治療威爾遜氏病的組合物和方法 |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| WO2023230466A1 (en) * | 2022-05-23 | 2023-11-30 | Lutsenka Svetlana | Compositions and methods for treating wilson's disease |
-
2020
- 2020-01-03 KR KR1020217024152A patent/KR20210112339A/ko active Pending
- 2020-01-03 EP EP23196931.2A patent/EP4269579B1/en active Active
- 2020-01-03 CN CN202080007863.5A patent/CN113518628B/zh active Active
- 2020-01-03 PL PL20704633.5T patent/PL3906066T3/pl unknown
- 2020-01-03 CA CA3124880A patent/CA3124880A1/en active Pending
- 2020-01-03 HU HUE20704633A patent/HUE065782T2/hu unknown
- 2020-01-03 WO PCT/US2020/012131 patent/WO2020142653A1/en not_active Ceased
- 2020-01-03 MX MX2021008135A patent/MX2021008135A/es unknown
- 2020-01-03 AU AU2020204679A patent/AU2020204679B2/en active Active
- 2020-01-03 SG SG11202106855YA patent/SG11202106855YA/en unknown
- 2020-01-03 PT PT207046335T patent/PT3906066T/pt unknown
- 2020-01-03 EP EP20704633.5A patent/EP3906066B1/en active Active
- 2020-01-03 US US17/417,619 patent/US12338450B2/en active Active
- 2020-01-03 ES ES20704633T patent/ES2970216T3/es active Active
- 2020-01-03 FI FIEP20704633.5T patent/FI3906066T3/fi active
- 2020-01-03 JP JP2021538754A patent/JP7531495B2/ja active Active
- 2020-01-03 TW TW109100147A patent/TWI848038B/zh active
- 2020-01-03 BR BR112021013140A patent/BR112021013140A2/pt unknown
- 2020-01-03 DK DK20704633.5T patent/DK3906066T5/da active
- 2020-01-03 HR HRP20231750TT patent/HRP20231750T1/hr unknown
-
2021
- 2021-06-29 ZA ZA2021/04515A patent/ZA202104515B/en unknown
- 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/JP7746437B2/ja active Active
-
2025
- 2025-05-20 US US19/213,623 patent/US20250333763A1/en active Pending
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