JPWO2020219933A5 - - Google Patents
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- Publication number
- JPWO2020219933A5 JPWO2020219933A5 JP2021561683A JP2021561683A JPWO2020219933A5 JP WO2020219933 A5 JPWO2020219933 A5 JP WO2020219933A5 JP 2021561683 A JP2021561683 A JP 2021561683A JP 2021561683 A JP2021561683 A JP 2021561683A JP WO2020219933 A5 JPWO2020219933 A5 JP WO2020219933A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- capsid protein
- retinal
- virion
- diabetic
- 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
Links
- 108090000565 Capsid Proteins Proteins 0.000 claims description 67
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 67
- 210000002845 virion Anatomy 0.000 claims description 56
- 108090000623 proteins and genes Proteins 0.000 claims description 33
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims description 25
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 24
- 210000004027 cell Anatomy 0.000 claims description 23
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 22
- 230000001225 therapeutic effect Effects 0.000 claims description 21
- 208000017442 Retinal disease Diseases 0.000 claims description 20
- 238000000034 method Methods 0.000 claims description 16
- 208000004644 retinal vein occlusion Diseases 0.000 claims description 16
- 208000002780 macular degeneration Diseases 0.000 claims description 15
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 14
- 150000007523 nucleic acids Chemical class 0.000 claims description 13
- 108091033319 polynucleotide Proteins 0.000 claims description 13
- 102000040430 polynucleotide Human genes 0.000 claims description 13
- 239000002157 polynucleotide Substances 0.000 claims description 13
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 12
- 125000000539 amino acid group Chemical group 0.000 claims description 12
- 239000008194 pharmaceutical composition Substances 0.000 claims description 12
- 201000010099 disease Diseases 0.000 claims description 11
- 206010038923 Retinopathy Diseases 0.000 claims description 10
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 10
- 208000035475 disorder Diseases 0.000 claims description 10
- 206010012601 diabetes mellitus Diseases 0.000 claims description 9
- 108020004707 nucleic acids Proteins 0.000 claims description 9
- 102000039446 nucleic acids Human genes 0.000 claims description 9
- 241000288906 Primates Species 0.000 claims description 8
- 210000000234 capsid Anatomy 0.000 claims description 8
- 102000004169 proteins and genes Human genes 0.000 claims description 8
- 210000001525 retina Anatomy 0.000 claims description 8
- 208000005590 Choroidal Neovascularization Diseases 0.000 claims description 7
- 206010060823 Choroidal neovascularisation Diseases 0.000 claims description 7
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 7
- 230000000302 ischemic effect Effects 0.000 claims description 7
- 208000032253 retinal ischemia Diseases 0.000 claims description 7
- 206010012688 Diabetic retinal oedema Diseases 0.000 claims description 6
- 241000124008 Mammalia Species 0.000 claims description 6
- 201000005667 central retinal vein occlusion Diseases 0.000 claims description 6
- 239000013604 expression vector Substances 0.000 claims description 6
- 238000002347 injection Methods 0.000 claims description 6
- 239000007924 injection Substances 0.000 claims description 6
- 238000006467 substitution reaction Methods 0.000 claims description 6
- 208000002158 Proliferative Vitreoretinopathy Diseases 0.000 claims description 5
- 206010038934 Retinopathy proliferative Diseases 0.000 claims description 5
- 208000021971 neovascular inflammatory vitreoretinopathy Diseases 0.000 claims description 5
- 230000006785 proliferative vitreoretinopathy Effects 0.000 claims description 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 4
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 4
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 4
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 4
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 4
- 206010003694 Atrophy Diseases 0.000 claims description 4
- 241000271566 Aves Species 0.000 claims description 4
- 201000001321 Bardet-Biedl syndrome Diseases 0.000 claims description 4
- 241000283690 Bos taurus Species 0.000 claims description 4
- 241000282465 Canis Species 0.000 claims description 4
- 206010010356 Congenital anomaly Diseases 0.000 claims description 4
- 206010012692 Diabetic uveitis Diseases 0.000 claims description 4
- 208000001351 Epiretinal Membrane Diseases 0.000 claims description 4
- 241000283073 Equus caballus Species 0.000 claims description 4
- 208000010412 Glaucoma Diseases 0.000 claims description 4
- 208000032087 Hereditary Leber Optic Atrophy Diseases 0.000 claims description 4
- 201000002563 Histoplasmosis Diseases 0.000 claims description 4
- 206010056715 Laurence-Moon-Bardet-Biedl syndrome Diseases 0.000 claims description 4
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims description 4
- 201000000639 Leber hereditary optic neuropathy Diseases 0.000 claims description 4
- 208000031471 Macular fibrosis Diseases 0.000 claims description 4
- 208000010164 Multifocal Choroiditis Diseases 0.000 claims description 4
- 208000001140 Night Blindness Diseases 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 4
- 206010030113 Oedema Diseases 0.000 claims description 4
- 206010038848 Retinal detachment Diseases 0.000 claims description 4
- 108020004459 Small interfering RNA Proteins 0.000 claims description 4
- 208000027073 Stargardt disease Diseases 0.000 claims description 4
- 206010042742 Sympathetic ophthalmia Diseases 0.000 claims description 4
- 208000014769 Usher Syndromes Diseases 0.000 claims description 4
- 230000001154 acute effect Effects 0.000 claims description 4
- 230000002137 anti-vascular effect Effects 0.000 claims description 4
- 230000037444 atrophy Effects 0.000 claims description 4
- 230000004456 color vision Effects 0.000 claims description 4
- 238000009792 diffusion process Methods 0.000 claims description 4
- 208000024519 eye neoplasm Diseases 0.000 claims description 4
- 208000014674 injury Diseases 0.000 claims description 4
- 108091070501 miRNA Proteins 0.000 claims description 4
- 239000002679 microRNA Substances 0.000 claims description 4
- 238000002428 photodynamic therapy Methods 0.000 claims description 4
- 210000001927 retinal artery Anatomy 0.000 claims description 4
- 230000004264 retinal detachment Effects 0.000 claims description 4
- 230000004283 retinal dysfunction Effects 0.000 claims description 4
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 claims description 4
- 201000007714 retinoschisis Diseases 0.000 claims description 4
- 208000011580 syndromic disease Diseases 0.000 claims description 4
- 230000002463 transducing effect Effects 0.000 claims description 4
- 230000008733 trauma Effects 0.000 claims description 4
- 208000036443 AIPL1-related retinopathy Diseases 0.000 claims description 3
- 208000009137 Behcet syndrome Diseases 0.000 claims description 3
- 108010041308 Endothelial Growth Factors Proteins 0.000 claims description 3
- 208000010038 Ischemic Optic Neuropathy Diseases 0.000 claims description 3
- 206010030924 Optic ischaemic neuropathy Diseases 0.000 claims description 3
- 208000007135 Retinal Neovascularization Diseases 0.000 claims description 3
- 206010038933 Retinopathy of prematurity Diseases 0.000 claims description 3
- 206010043189 Telangiectasia Diseases 0.000 claims description 3
- 201000007058 anterior ischemic optic neuropathy Diseases 0.000 claims description 3
- 201000004709 chorioretinitis Diseases 0.000 claims description 3
- 201000006754 cone-rod dystrophy Diseases 0.000 claims description 3
- 201000011190 diabetic macular edema Diseases 0.000 claims description 3
- 239000003814 drug Substances 0.000 claims description 3
- 208000011325 dry age related macular degeneration Diseases 0.000 claims description 3
- 230000001771 impaired effect Effects 0.000 claims description 3
- 230000000649 photocoagulation Effects 0.000 claims description 3
- 201000007914 proliferative diabetic retinopathy Diseases 0.000 claims description 3
- 208000009056 telangiectasis Diseases 0.000 claims description 3
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 208000003098 Ganglion Cysts Diseases 0.000 claims description 2
- 208000005400 Synovial Cyst Diseases 0.000 claims description 2
- 208000000208 Wet Macular Degeneration Diseases 0.000 claims description 2
- 210000000411 amacrine cell Anatomy 0.000 claims description 2
- 210000003986 cell retinal photoreceptor Anatomy 0.000 claims description 2
- 230000005101 cell tropism Effects 0.000 claims description 2
- 238000012258 culturing Methods 0.000 claims description 2
- 210000002287 horizontal cell Anatomy 0.000 claims description 2
- 238000004519 manufacturing process Methods 0.000 claims description 2
- 238000002360 preparation method Methods 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 7
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 2
- 201000008106 ocular cancer Diseases 0.000 claims 2
- 230000003085 retinopathic effect Effects 0.000 claims 2
- 208000027496 Behcet disease Diseases 0.000 claims 1
- 206010061323 Optic neuropathy Diseases 0.000 claims 1
- 230000006378 damage Effects 0.000 claims 1
- 230000006735 deficit Effects 0.000 claims 1
- 230000007850 degeneration Effects 0.000 claims 1
- 230000003511 endothelial effect Effects 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 208000020911 optic nerve disease Diseases 0.000 claims 1
- 230000002062 proliferating effect Effects 0.000 claims 1
- 230000035755 proliferation Effects 0.000 claims 1
- 208000031816 Pathologic Dilatation Diseases 0.000 description 1
- 206010064714 Radiation retinopathy Diseases 0.000 description 1
- 201000007737 Retinal degeneration Diseases 0.000 description 1
- 230000015271 coagulation Effects 0.000 description 1
- 238000005345 coagulation Methods 0.000 description 1
- 229940124597 therapeutic agent Drugs 0.000 description 1
- 210000003462 vein Anatomy 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962839548P | 2019-04-26 | 2019-04-26 | |
| US62/839,548 | 2019-04-26 | ||
| US201962923924P | 2019-10-21 | 2019-10-21 | |
| US62/923,924 | 2019-10-21 | ||
| PCT/US2020/029895 WO2020219933A1 (en) | 2019-04-26 | 2020-04-24 | Variant aav capsids for intravitreal delivery |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022529775A JP2022529775A (ja) | 2022-06-24 |
| JP2022529775A5 JP2022529775A5 (https=) | 2023-05-11 |
| JPWO2020219933A5 true JPWO2020219933A5 (https=) | 2023-05-11 |
| JP7623703B2 JP7623703B2 (ja) | 2025-01-29 |
Family
ID=70740759
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021561683A Active JP7623703B2 (ja) | 2019-04-26 | 2020-04-24 | 硝子体内送達のためのバリアントａａｖキャプシド |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US11510950B2 (https=) |
| EP (1) | EP3958911A1 (https=) |
| JP (1) | JP7623703B2 (https=) |
| KR (1) | KR20220004114A (https=) |
| CN (2) | CN113766934B (https=) |
| AU (1) | AU2020262948A1 (https=) |
| BR (1) | BR112021020957A2 (https=) |
| CA (1) | CA3130217A1 (https=) |
| IL (1) | IL287438A (https=) |
| MX (2) | MX2021012311A (https=) |
| SG (1) | SG11202108928XA (https=) |
| TW (2) | TW202532428A (https=) |
| WO (1) | WO2020219933A1 (https=) |
| ZA (1) | ZA202105975B (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3841109A4 (en) * | 2018-08-21 | 2022-09-21 | Massachusetts Eye and Ear Infirmary | COMPOSITIONS AND METHODS TO MODULATE THE TRANSDUCTION EFFICIENCY OF ADENO-ASSOCIATED VIRUSES |
| EP3861010A1 (en) | 2018-10-02 | 2021-08-11 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| CN116096734A (zh) | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| CN115044614B (zh) * | 2021-03-09 | 2023-10-20 | 上海目镜生物医药科技有限公司 | 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用 |
| US12385064B2 (en) * | 2021-03-26 | 2025-08-12 | Adverum Biotechnologies, Inc. | Intravitreal dosing for delivery of polynucleotides to retinal cones |
| CN113648432B (zh) * | 2021-03-31 | 2024-01-19 | 中山大学中山眼科中心 | rAAV2/Retro作为针对视网膜感光细胞的递送系统及其在制备治疗视网膜疾病药物中的应用 |
| MX2024004936A (es) | 2021-11-02 | 2024-05-08 | Voyager Therapeutics Inc | Variantes de capsides de aav y sus usos. |
| AU2024242380A1 (en) * | 2023-03-31 | 2025-11-06 | Exegenesis Bio Singapore Pte. Ltd. | Variant aav9 capsid proteins and uses thereof |
| WO2024211831A1 (en) | 2023-04-05 | 2024-10-10 | Adverum Biotechnologies, Inc. | Immune landscape signatures for ocular inflammation |
| AU2024297654A1 (en) | 2023-07-13 | 2026-01-22 | Kriya Therapeutics, Inc. | Complement pathway inhibitor fusions and methods of using the same |
| CN117247434B (zh) * | 2023-11-10 | 2024-02-02 | 上海朗昇生物科技有限公司 | 衣壳修饰型病毒载体及其制备和用途 |
| CN118955654B (zh) * | 2024-10-15 | 2025-05-16 | 朗信启昇(苏州)生物制药有限公司 | 具有变异衣壳的腺相关病毒病毒体及其用途 |
| WO2026084101A1 (ko) | 2024-10-18 | 2026-04-23 | 주식회사 아바타테라퓨틱스 | 아데노연관바이러스 변이체 |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| WO1994004679A1 (en) | 1991-06-14 | 1994-03-03 | Genentech, Inc. | Method for making humanized antibodies |
| AU684498B2 (en) | 1993-03-25 | 1997-12-18 | Merck & Co., Inc. | Inhibitor of vascular endothelial cell growth factor |
| JP3957765B2 (ja) | 1997-04-07 | 2007-08-15 | ジェネンテク・インコーポレイテッド | 抗ｖｅｇｆ抗体 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US7087411B2 (en) | 1999-06-08 | 2006-08-08 | Regeneron Pharmaceuticals, Inc. | Fusion protein capable of binding VEGF |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2004075861A2 (en) | 2003-02-26 | 2004-09-10 | Children's Hospital, Inc. | Recombinant adeno-associated virus production |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| EP1919497B1 (en) | 2005-07-22 | 2020-02-12 | The Board of Trustees of the Leland Stanford Junior University | Light-activated cation channel and uses thereof |
| EP2019588A4 (en) | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| EP2287323A1 (en) * | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
| 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 |
| TWI775096B (zh) | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| TWI738632B (zh) | 2014-11-07 | 2021-09-11 | 瑞士商諾華公司 | 穩定的含有高濃度抗vegf抗體之蛋白質溶液調配物 |
| 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 |
| CA3022667A1 (en) | 2016-05-03 | 2017-11-09 | Adverum Biotechnologies, Inc. | Organotypic pig retina culture system |
| FI3445773T3 (fi) * | 2016-05-13 | 2023-03-30 | 4D Molecular Therapeutics Inc | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä |
| EP3827812B1 (en) * | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| AU2018227483A1 (en) * | 2017-02-28 | 2019-09-12 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
-
2020
- 2020-04-23 TW TW113141337A patent/TW202532428A/zh unknown
- 2020-04-23 TW TW109113631A patent/TWI862583B/zh active
- 2020-04-24 CA CA3130217A patent/CA3130217A1/en active Pending
- 2020-04-24 CN CN202080031274.0A patent/CN113766934B/zh active Active
- 2020-04-24 AU AU2020262948A patent/AU2020262948A1/en active Pending
- 2020-04-24 WO PCT/US2020/029895 patent/WO2020219933A1/en not_active Ceased
- 2020-04-24 US US16/858,042 patent/US11510950B2/en active Active
- 2020-04-24 CN CN202411569264.9A patent/CN119708162A/zh active Pending
- 2020-04-24 SG SG11202108928XA patent/SG11202108928XA/en unknown
- 2020-04-24 JP JP2021561683A patent/JP7623703B2/ja active Active
- 2020-04-24 BR BR112021020957A patent/BR112021020957A2/pt unknown
- 2020-04-24 KR KR1020217038123A patent/KR20220004114A/ko active Pending
- 2020-04-24 MX MX2021012311A patent/MX2021012311A/es unknown
- 2020-04-24 EP EP20726600.8A patent/EP3958911A1/en active Pending
-
2021
- 2021-08-19 ZA ZA2021/05975A patent/ZA202105975B/en unknown
- 2021-10-07 MX MX2025015023A patent/MX2025015023A/es unknown
- 2021-10-20 IL IL287438A patent/IL287438A/en unknown
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