JP2020510424A5 - - Google Patents
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- Publication number
- JP2020510424A5 JP2020510424A5 JP2019546817A JP2019546817A JP2020510424A5 JP 2020510424 A5 JP2020510424 A5 JP 2020510424A5 JP 2019546817 A JP2019546817 A JP 2019546817A JP 2019546817 A JP2019546817 A JP 2019546817A JP 2020510424 A5 JP2020510424 A5 JP 2020510424A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- modified
- pharmaceutical composition
- amino acid
- recombinant virus
- 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
- 108090000565 Capsid Proteins Proteins 0.000 claims description 54
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 54
- 241000700605 Viruses Species 0.000 claims description 34
- 239000013603 viral vector Substances 0.000 claims description 32
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 30
- 239000008194 pharmaceutical composition Substances 0.000 claims description 30
- 108090000623 proteins and genes Proteins 0.000 claims description 23
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 20
- 230000001225 therapeutic effect Effects 0.000 claims description 20
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 19
- 125000000539 amino acid group Chemical group 0.000 claims description 18
- 238000000034 method Methods 0.000 claims description 17
- 208000035475 disorder Diseases 0.000 claims description 16
- 238000003780 insertion Methods 0.000 claims description 16
- 230000037431 insertion Effects 0.000 claims description 16
- 108091033319 polynucleotide Proteins 0.000 claims description 16
- 102000040430 polynucleotide Human genes 0.000 claims description 16
- 239000002157 polynucleotide Substances 0.000 claims description 16
- 201000010099 disease Diseases 0.000 claims description 14
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 10
- 210000004027 cell Anatomy 0.000 claims description 9
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 8
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 7
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 6
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 6
- 208000030533 eye disease Diseases 0.000 claims description 6
- 230000002265 prevention Effects 0.000 claims description 5
- 102000004169 proteins and genes Human genes 0.000 claims description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 4
- 108010041308 Endothelial Growth Factors Proteins 0.000 claims description 4
- 108010076282 Factor IX Proteins 0.000 claims description 4
- 108010054218 Factor VIII Proteins 0.000 claims description 4
- 102000001690 Factor VIII Human genes 0.000 claims description 4
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims description 4
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims description 4
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims description 4
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 4
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims description 4
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims description 4
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims description 4
- 230000002137 anti-vascular effect Effects 0.000 claims description 4
- 229940009550 c1 esterase inhibitor Drugs 0.000 claims description 4
- 239000003795 chemical substances by application Substances 0.000 claims description 4
- 239000013604 expression vector Substances 0.000 claims description 4
- 229960004222 factor ix Drugs 0.000 claims description 4
- 229960000301 factor viii Drugs 0.000 claims description 4
- 150000007523 nucleic acids Chemical group 0.000 claims description 4
- 208000017442 Retinal disease Diseases 0.000 claims description 3
- 206010038923 Retinopathy Diseases 0.000 claims description 3
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 3
- 230000002207 retinal effect Effects 0.000 claims description 3
- 208000004644 retinal vein occlusion Diseases 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 208000009292 Hemophilia A Diseases 0.000 claims description 2
- 229920002971 Heparan sulfate Polymers 0.000 claims description 2
- 206010019860 Hereditary angioedema Diseases 0.000 claims description 2
- 241000124008 Mammalia Species 0.000 claims description 2
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 2
- 208000005980 beta thalassemia Diseases 0.000 claims description 2
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 2
- 208000009429 hemophilia B Diseases 0.000 claims description 2
- 210000003494 hepatocyte Anatomy 0.000 claims description 2
- 238000002347 injection Methods 0.000 claims description 2
- 239000007924 injection Substances 0.000 claims description 2
- 230000000302 ischemic effect Effects 0.000 claims description 2
- 208000019423 liver disease Diseases 0.000 claims description 2
- 239000012528 membrane Substances 0.000 claims description 2
- 201000007914 proliferative diabetic retinopathy Diseases 0.000 claims description 2
- 150000003839 salts Chemical class 0.000 claims description 2
- 239000000047 product Substances 0.000 claims 10
- 206010012601 diabetes mellitus Diseases 0.000 claims 3
- 208000031220 Hemophilia Diseases 0.000 claims 1
- 206010030113 Oedema Diseases 0.000 claims 1
- 201000010183 Papilledema Diseases 0.000 claims 1
- 206010038886 Retinal oedema Diseases 0.000 claims 1
- 230000033115 angiogenesis Effects 0.000 claims 1
- 210000000234 capsid Anatomy 0.000 claims 1
- 201000005667 central retinal vein occlusion Diseases 0.000 claims 1
- 239000013066 combination product Substances 0.000 claims 1
- 229940127555 combination product Drugs 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 230000000938 luteal effect Effects 0.000 claims 1
- 201000011195 retinal edema Diseases 0.000 claims 1
- 208000032253 retinal ischemia Diseases 0.000 claims 1
- 210000001210 retinal vessel Anatomy 0.000 claims 1
- 239000000126 substance Substances 0.000 claims 1
- 239000002753 trypsin inhibitor Substances 0.000 claims 1
- 239000013598 vector Substances 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 description 2
- 210000001957 retinal vein Anatomy 0.000 description 2
- 102100026735 Coagulation factor VIII Human genes 0.000 description 1
- 206010012688 Diabetic retinal oedema Diseases 0.000 description 1
- 201000003542 Factor VIII deficiency Diseases 0.000 description 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 description 1
- 206010029113 Neovascularisation Diseases 0.000 description 1
- 208000007135 Retinal Neovascularization Diseases 0.000 description 1
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 description 1
- 201000011190 diabetic macular edema Diseases 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 230000003612 virological effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762464878P | 2017-02-28 | 2017-02-28 | |
| US62/464,878 | 2017-02-28 | ||
| PCT/US2018/020215 WO2018160686A1 (en) | 2017-02-28 | 2018-02-28 | Modified aav capsids and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020510424A JP2020510424A (ja) | 2020-04-09 |
| JP2020510424A5 true JP2020510424A5 (https=) | 2021-04-08 |
Family
ID=63370228
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019546817A Pending JP2020510424A (ja) | 2017-02-28 | 2018-02-28 | 改変ａａｖカプシドおよびその使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210130413A1 (https=) |
| EP (1) | EP3589277A4 (https=) |
| JP (1) | JP2020510424A (https=) |
| CN (1) | CN110650733A (https=) |
| AU (1) | AU2018227483A1 (https=) |
| CA (1) | CA3054687A1 (https=) |
| WO (1) | WO2018160686A1 (https=) |
Families Citing this family (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| MY187898A (en) | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CN110546257B (zh) | 2017-03-17 | 2024-03-01 | 阿德夫拉姆生物技术股份有限公司 | 用于增强基因表达的组合物和方法 |
| AU2018350990A1 (en) * | 2017-10-18 | 2020-05-21 | Regenxbio Inc. | Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| WO2020068990A1 (en) * | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| WO2020180951A1 (en) * | 2019-03-04 | 2020-09-10 | Adverum Biotechnologies, Inc. | Sequential intravitreal administration of aav gene therapy to contralateral eyes |
| TW202102526A (zh) * | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| KR20220012231A (ko) * | 2019-04-24 | 2022-02-03 | 리젠엑스바이오 인크. | 완전-인간 번역 후 변형된 항체 치료제 |
| TW202532428A (zh) * | 2019-04-26 | 2025-08-16 | 美商愛德維仁生物科技公司 | 用於玻璃體內遞送之變異體aav蛋白殼 |
| AR122013A1 (es) * | 2020-05-05 | 2022-08-03 | Univ Duke | Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso |
| CN116121274B (zh) * | 2020-07-29 | 2024-11-19 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| BR112023003548A2 (pt) * | 2020-09-03 | 2023-04-04 | Univ Massachusetts | Vírus adenoassociado para liberação de kh902 (conbercept) e usos do mesmo |
| CN116528892A (zh) * | 2020-10-28 | 2023-08-01 | 再生生物股份有限公司 | 用于眼部适应症的载体化抗TNF-α抗体 |
| TW202242124A (zh) * | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | 靶向ｔ細胞之ａａｖ載體 |
| CN114686448A (zh) * | 2022-03-31 | 2022-07-01 | 上海勉亦生物科技有限公司 | 具有肝脏特异靶向性的纯化的腺相关病毒及其应用 |
| CN112961220B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体 |
| CN113121652B (zh) * | 2021-04-19 | 2022-10-11 | 上海信致医药科技有限公司 | 视网膜和肌肉高亲和性腺相关病毒衣壳蛋白及相关应用 |
| MX2023014041A (es) | 2021-05-28 | 2023-12-15 | Shanghai Regenelead Therapies Co Ltd | Virus adenoasociado recombinante que tiene una capsida variante y su aplicacion. |
| CN113564187B (zh) * | 2021-07-30 | 2026-03-27 | 上海信致医药科技有限公司 | 基于aav的抗血管生成基因递送系统及其用途 |
| CN117230117A (zh) * | 2023-08-04 | 2023-12-15 | 中国科学院深圳先进技术研究院 | 重组腺相关病毒包装质粒、突变体及其制备方法和应用 |
| CN117143920A (zh) * | 2023-08-30 | 2023-12-01 | 呈诺再生医学科技(北京)有限公司 | 一种治疗新生血管性眼部疾病的基因药物 |
| CN117368466B (zh) * | 2023-12-08 | 2024-03-12 | 安徽惠邦生物工程有限公司 | 用生化分析仪测定尿结合珠蛋白的试剂盒及其制备方法 |
| WO2025214359A1 (en) * | 2024-04-09 | 2025-10-16 | Hangzhou Jiayin Biotech Ltd. | A novel aav variant |
| GB202414499D0 (en) | 2024-10-02 | 2024-11-13 | Purespring Therapeutics Ltd | Methods |
| WO2025246365A2 (en) * | 2025-06-20 | 2025-12-04 | Hangzhou Jiayin Biotech Ltd. | Aav variants and uses thereof |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2349838C (en) * | 1998-11-05 | 2011-06-07 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| US8663624B2 (en) * | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| HRP20171334T1 (hr) * | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| GB201403260D0 (en) * | 2014-02-25 | 2014-04-09 | Univ Manchester | Treatment of retinal degeneration using gene therapy |
| AU2015229464B2 (en) * | 2014-03-11 | 2021-07-22 | Wayne State University | A modified mGluR6 promoter and methods of use |
-
2018
- 2018-02-28 AU AU2018227483A patent/AU2018227483A1/en not_active Abandoned
- 2018-02-28 CN CN201880025324.7A patent/CN110650733A/zh active Pending
- 2018-02-28 US US16/488,689 patent/US20210130413A1/en not_active Abandoned
- 2018-02-28 JP JP2019546817A patent/JP2020510424A/ja active Pending
- 2018-02-28 EP EP18760397.2A patent/EP3589277A4/en not_active Withdrawn
- 2018-02-28 WO PCT/US2018/020215 patent/WO2018160686A1/en not_active Ceased
- 2018-02-28 CA CA3054687A patent/CA3054687A1/en active Pending
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