JPWO2020102723A5 - - Google Patents
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- JPWO2020102723A5 JPWO2020102723A5 JP2021526562A JP2021526562A JPWO2020102723A5 JP WO2020102723 A5 JPWO2020102723 A5 JP WO2020102723A5 JP 2021526562 A JP2021526562 A JP 2021526562A JP 2021526562 A JP2021526562 A JP 2021526562A JP WO2020102723 A5 JPWO2020102723 A5 JP WO2020102723A5
- Authority
- JP
- Japan
- Prior art keywords
- item
- nucleic acid
- acid molecule
- items
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- 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.)
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- 108020004707 nucleic acids Proteins 0.000 claims description 183
- 102000039446 nucleic acids Human genes 0.000 claims description 183
- 150000007523 nucleic acids Chemical class 0.000 claims description 183
- 239000013604 expression vector Substances 0.000 claims description 59
- 230000001105 regulatory effect Effects 0.000 claims description 50
- 239000002245 particle Substances 0.000 claims description 47
- 102100027591 Copper-transporting ATPase 2 Human genes 0.000 claims description 46
- 101000936280 Homo sapiens Copper-transporting ATPase 2 Proteins 0.000 claims description 46
- 239000002773 nucleotide Substances 0.000 claims description 38
- 125000003729 nucleotide group Chemical group 0.000 claims description 38
- 241000700605 Viruses Species 0.000 claims description 32
- 238000000034 method Methods 0.000 claims description 30
- 239000008194 pharmaceutical composition Substances 0.000 claims description 29
- 230000003612 virological effect Effects 0.000 claims description 25
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 20
- 108700019146 Transgenes Proteins 0.000 claims description 20
- 210000004027 cell Anatomy 0.000 claims description 20
- 230000001225 therapeutic effect Effects 0.000 claims description 20
- 210000004185 liver Anatomy 0.000 claims description 18
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 10
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 239000013603 viral vector Substances 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 8
- 239000012634 fragment Substances 0.000 claims description 8
- 210000004962 mammalian cell Anatomy 0.000 claims description 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 108090000565 Capsid Proteins Proteins 0.000 claims description 5
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 238000004113 cell culture Methods 0.000 claims description 5
- 239000012228 culture supernatant Substances 0.000 claims description 5
- 238000012258 culturing Methods 0.000 claims description 5
- 239000001963 growth medium Substances 0.000 claims description 5
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 5
- 108700028146 Genetic Enhancer Elements Proteins 0.000 claims description 4
- 108010054218 Factor VIII Proteins 0.000 claims description 3
- 102000001690 Factor VIII Human genes 0.000 claims description 3
- 229960000301 factor viii Drugs 0.000 claims description 3
- 102100028282 Bile salt export pump Human genes 0.000 claims description 2
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 2
- 102100023804 Coagulation factor VII Human genes 0.000 claims description 2
- 102100040499 Contactin-associated protein-like 2 Human genes 0.000 claims description 2
- 108010022637 Copper-Transporting ATPases Proteins 0.000 claims description 2
- 102100027587 Copper-transporting ATPase 1 Human genes 0.000 claims description 2
- 102100034746 Cyclin-dependent kinase-like 5 Human genes 0.000 claims description 2
- 108010076282 Factor IX Proteins 0.000 claims description 2
- 108010014172 Factor V Proteins 0.000 claims description 2
- 108010023321 Factor VII Proteins 0.000 claims description 2
- 108010014173 Factor X Proteins 0.000 claims description 2
- 108010074864 Factor XI Proteins 0.000 claims description 2
- 108010080865 Factor XII Proteins 0.000 claims description 2
- 101000749877 Homo sapiens Contactin-associated protein-like 2 Proteins 0.000 claims description 2
- 101000945692 Homo sapiens Cyclin-dependent kinase-like 5 Proteins 0.000 claims description 2
- 101000955481 Homo sapiens Phosphatidylcholine translocator ABCB4 Proteins 0.000 claims description 2
- 101000701363 Homo sapiens Phospholipid-transporting ATPase IC Proteins 0.000 claims description 2
- 101000723833 Homo sapiens Zinc finger E-box-binding homeobox 2 Proteins 0.000 claims description 2
- 108010093662 Member 11 Subfamily B ATP Binding Cassette Transporter Proteins 0.000 claims description 2
- 102100039032 Phosphatidylcholine translocator ABCB4 Human genes 0.000 claims description 2
- 102100030448 Phospholipid-transporting ATPase IC Human genes 0.000 claims description 2
- 102100028458 Zinc finger E-box-binding homeobox 2 Human genes 0.000 claims description 2
- 229960004222 factor ix Drugs 0.000 claims description 2
- 229940012413 factor vii Drugs 0.000 claims description 2
- 229940012426 factor x Drugs 0.000 claims description 2
- 239000008177 pharmaceutical agent Substances 0.000 claims 1
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 description 8
- 208000018839 Wilson disease Diseases 0.000 description 8
- 230000007812 deficiency Effects 0.000 description 8
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 8
- 208000035475 disorder Diseases 0.000 description 8
- 230000003190 augmentative effect Effects 0.000 description 2
- 230000002708 enhancing effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862768744P | 2018-11-16 | 2018-11-16 | |
| US62/768,744 | 2018-11-16 | ||
| PCT/US2019/061808 WO2020102723A1 (en) | 2018-11-16 | 2019-11-15 | Compositions and methods for treating wilson's disease |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022507534A JP2022507534A (ja) | 2022-01-18 |
| JPWO2020102723A5 true JPWO2020102723A5 (https=) | 2023-06-27 |
| JP2022507534A5 JP2022507534A5 (https=) | 2023-06-27 |
| JP7532360B2 JP7532360B2 (ja) | 2024-08-13 |
Family
ID=70731690
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021526562A Active JP7532360B2 (ja) | 2018-11-16 | 2019-11-15 | ウィルソン病を処置するための組成物および方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210369870A1 (https=) |
| EP (2) | EP4257155A3 (https=) |
| JP (1) | JP7532360B2 (https=) |
| CN (1) | CN113302290A (https=) |
| CA (1) | CA3118936A1 (https=) |
| TW (1) | TW202039854A (https=) |
| WO (1) | WO2020102723A1 (https=) |
Families Citing this family (18)
| 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. |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| KR20210112339A (ko) | 2019-01-04 | 2021-09-14 | 울트라제닉스 파마수티컬 인코포레이티드 | 윌슨병을 치료하기 위한 유전자 요법 구축물 |
| WO2022208342A1 (en) * | 2021-04-01 | 2022-10-06 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| 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 |
| CN113699211A (zh) * | 2021-07-20 | 2021-11-26 | 深圳市儿童医院 | 一种用于儿童威尔森氏症快速筛查的参数组合和试剂盒 |
| EP4398896A4 (en) * | 2021-09-07 | 2025-07-02 | Alexion Pharma Inc | METHODS FOR TREATING DISEASES OR DISORDERS ASSOCIATED WITH COPPER METABOLISM |
| US20250144239A1 (en) * | 2021-10-18 | 2025-05-08 | Logicbio Therapeutics, Inc. | Gene therapy for the treatment of wilson's disease |
| KR20250102134A (ko) * | 2022-10-11 | 2025-07-04 | 메이라지티엑스 유케이 Ii 리미티드 | Atp7b 유전자 요법 |
| 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 |
| CN117757797B (zh) * | 2023-09-25 | 2024-08-16 | 北京圭特细胞科技有限公司 | 一种编码人III型胶原蛋白α链的核酸及其应用 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
| CN121265551A (zh) * | 2024-07-02 | 2026-01-06 | 苏州星核迪赛生物技术有限公司 | 一种靶向肝实质细胞的核酸递送载体及其应用 |
| CN121319202B (zh) * | 2025-12-15 | 2026-04-07 | 凌意(杭州)生物科技有限公司 | Atp7b抗体及其应用 |
Family Cites Families (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6995006B2 (en) | 1997-09-05 | 2006-02-07 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| ES2284545T3 (es) * | 1999-11-16 | 2007-11-16 | Genzyme Corporation | Vectores y transgenes con elementos reguladores para la administracion de genes del higado. |
| EP1103610A1 (en) | 1999-11-26 | 2001-05-30 | Introgene B.V. | Production of vaccines from immortalised mammalian cell lines |
| FR2808803B1 (fr) | 2000-05-11 | 2004-12-10 | Agronomique Inst Nat Rech | Cellules es modifiees et gene specifique de cellules es |
| FR2836924B1 (fr) | 2002-03-08 | 2005-01-14 | Vivalis | Lignees de cellules aviaires utiles pour la production de substances d'interet |
| 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 |
| EP1528101A1 (en) | 2003-11-03 | 2005-05-04 | ProBioGen AG | Immortalized avian cell lines for virus production |
| FR2884255B1 (fr) | 2005-04-11 | 2010-11-05 | Vivalis | Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe |
| BRPI0619794B8 (pt) | 2005-12-13 | 2022-06-14 | Univ Kyoto | Uso de um fator de reprogramação, agente para a preparação de uma célula-tronco pluripotente induzida a partir de uma célula somática e métodos para preparar uma célula- tronco pluripotente induzida método e para preparar uma célula somática e uso de células-tronco pluripotentes induzidas |
| DK3399025T3 (da) | 2007-03-23 | 2025-06-16 | Wisconsin Alumni Res Found | Omprogrammering af somatiske celler |
| EP1985305A1 (en) | 2007-04-24 | 2008-10-29 | Vivalis | Duck embryonic derived stem cell lines for the production of viral vaccines |
| EP1995309A1 (en) | 2007-05-21 | 2008-11-26 | Vivalis | Recombinant protein production in avian EBx® cells |
| HUE028341T2 (en) | 2009-06-16 | 2016-12-28 | Genzyme Corp | Improved methods for purification of recombinant aav vectors |
| US20150045284A1 (en) | 2011-01-21 | 2015-02-12 | Aarhus Universitet | CRYSTAL STRUCTURE OF A TYPE IB P-TYPE ATPase |
| WO2015121793A1 (en) * | 2014-02-11 | 2015-08-20 | Friedrich Miescher Institute For Biomedical Research | Müller cell-specific promoter |
| 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 |
| KR102580696B1 (ko) * | 2014-12-30 | 2023-09-19 | 큐어백 에스이 | 신규 인공 핵산 분자 |
| EP3270944B1 (en) | 2015-03-17 | 2019-10-23 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for fviii and fix |
| GB201522416D0 (en) | 2015-12-18 | 2016-02-03 | Ucl Business Plc | Wilson's disease gene therapy |
| MX2019007873A (es) | 2016-12-30 | 2019-11-18 | Univ Pennsylvania | Terapia génica para tratar la enfermedad de wilson. |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
-
2019
- 2019-11-15 TW TW108141687A patent/TW202039854A/zh unknown
- 2019-11-15 CN CN201980089235.3A patent/CN113302290A/zh active Pending
- 2019-11-15 EP EP23185569.3A patent/EP4257155A3/en active Pending
- 2019-11-15 US US17/294,325 patent/US20210369870A1/en not_active Abandoned
- 2019-11-15 JP JP2021526562A patent/JP7532360B2/ja active Active
- 2019-11-15 WO PCT/US2019/061808 patent/WO2020102723A1/en not_active Ceased
- 2019-11-15 EP EP19884231.2A patent/EP3880809A4/en not_active Withdrawn
- 2019-11-15 CA CA3118936A patent/CA3118936A1/en active Pending
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