JPWO2020242984A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020242984A5 JPWO2020242984A5 JP2021569402A JP2021569402A JPWO2020242984A5 JP WO2020242984 A5 JPWO2020242984 A5 JP WO2020242984A5 JP 2021569402 A JP2021569402 A JP 2021569402A JP 2021569402 A JP2021569402 A JP 2021569402A JP WO2020242984 A5 JPWO2020242984 A5 JP WO2020242984A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- acid sequence
- seq
- amino acid
- protein
- 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 295
- 102100023321 Ceruloplasmin Human genes 0.000 claims description 295
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 288
- 108090000623 proteins and genes Proteins 0.000 claims description 144
- 102000004169 proteins and genes Human genes 0.000 claims description 136
- 241000288906 Primates Species 0.000 claims description 126
- 239000002245 particle Substances 0.000 claims description 122
- 150000007523 nucleic acids Chemical group 0.000 claims description 78
- 241000700605 Viruses Species 0.000 claims description 64
- 239000002773 nucleotide Substances 0.000 claims description 60
- 125000003729 nucleotide group Chemical group 0.000 claims description 60
- 108020004707 nucleic acids Proteins 0.000 claims description 46
- 102000039446 nucleic acids Human genes 0.000 claims description 46
- 210000000234 capsid Anatomy 0.000 claims description 39
- 210000004027 cell Anatomy 0.000 claims description 35
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 33
- 150000001413 amino acids Chemical class 0.000 claims description 29
- 230000035772 mutation Effects 0.000 claims description 29
- 101150044789 Cap gene Proteins 0.000 claims description 26
- 230000010415 tropism Effects 0.000 claims description 24
- 238000000034 method Methods 0.000 claims description 21
- 238000004806 packaging method and process Methods 0.000 claims description 20
- 230000003612 virological effect Effects 0.000 claims description 18
- 230000004048 modification Effects 0.000 claims description 16
- 238000012986 modification Methods 0.000 claims description 16
- 241000282320 Panthera leo Species 0.000 claims description 13
- 101150066583 rep gene Proteins 0.000 claims description 11
- 241000702421 Dependoparvovirus Species 0.000 claims description 7
- 239000003446 ligand Substances 0.000 claims description 7
- 230000008685 targeting Effects 0.000 claims description 7
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 241000271566 Aves Species 0.000 claims description 5
- 238000010453 CRISPR/Cas method Methods 0.000 claims description 5
- 102000053642 Catalytic RNA Human genes 0.000 claims description 5
- 108090000994 Catalytic RNA Proteins 0.000 claims description 5
- 101710135898 Myc proto-oncogene protein Proteins 0.000 claims description 5
- 102100038895 Myc proto-oncogene protein Human genes 0.000 claims description 5
- 108091034117 Oligonucleotide Proteins 0.000 claims description 5
- 108091030071 RNAI Proteins 0.000 claims description 5
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 5
- 101710150448 Transcriptional regulator Myc Proteins 0.000 claims description 5
- 230000004075 alteration Effects 0.000 claims description 5
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 5
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 5
- 239000012634 fragment Substances 0.000 claims description 5
- 230000009368 gene silencing by RNA Effects 0.000 claims description 5
- 239000000203 mixture Substances 0.000 claims description 5
- 108091092562 ribozyme Proteins 0.000 claims description 5
- 239000004055 small Interfering RNA Substances 0.000 claims description 5
- 230000001225 therapeutic effect Effects 0.000 claims description 5
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 4
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 claims description 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 3
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 3
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 3
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 3
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 3
- 210000005260 human cell Anatomy 0.000 claims description 3
- 238000012258 culturing Methods 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 2
- 241000282806 Rhinoceros Species 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 210000002845 virion Anatomy 0.000 claims 1
- 108091005957 yellow fluorescent proteins Proteins 0.000 description 6
- 101100524324 Adeno-associated virus 2 (isolate Srivastava/1982) Rep78 gene Proteins 0.000 description 4
- 108091005941 EBFP Proteins 0.000 description 4
- 108010043121 Green Fluorescent Proteins Proteins 0.000 description 4
- 102000004144 Green Fluorescent Proteins Human genes 0.000 description 4
- 108700008625 Reporter Genes Proteins 0.000 description 4
- 108091005948 blue fluorescent proteins Proteins 0.000 description 4
- 108010082025 cyan fluorescent protein Proteins 0.000 description 4
- 108010048367 enhanced green fluorescent protein Proteins 0.000 description 4
- 108010021843 fluorescent protein 583 Proteins 0.000 description 4
- 239000005090 green fluorescent protein Substances 0.000 description 4
- YMHOBZXQZVXHBM-UHFFFAOYSA-N 2,5-dimethoxy-4-bromophenethylamine Chemical compound COC1=CC(CCN)=C(OC)C=C1Br YMHOBZXQZVXHBM-UHFFFAOYSA-N 0.000 description 2
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 description 2
- 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 2
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 description 2
- 102000002260 Alkaline Phosphatase Human genes 0.000 description 2
- 108020004774 Alkaline Phosphatase Proteins 0.000 description 2
- 108091005944 Cerulean Proteins 0.000 description 2
- 241000579895 Chlorostilbon Species 0.000 description 2
- 108020004705 Codon Proteins 0.000 description 2
- 108091005943 CyPet Proteins 0.000 description 2
- 108060001084 Luciferase Proteins 0.000 description 2
- 239000005089 Luciferase Substances 0.000 description 2
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 2
- 241000545067 Venus Species 0.000 description 2
- 108010005774 beta-Galactosidase Proteins 0.000 description 2
- 102000005936 beta-Galactosidase Human genes 0.000 description 2
- 229910052876 emerald Inorganic materials 0.000 description 2
- 239000010976 emerald Substances 0.000 description 2
- 239000000463 material Substances 0.000 description 2
- 239000013612 plasmid Substances 0.000 description 2
- 108010054624 red fluorescent protein Proteins 0.000 description 2
- 229910052594 sapphire Inorganic materials 0.000 description 2
- 239000010980 sapphire Substances 0.000 description 2
- 108010008532 Deoxyribonuclease I Proteins 0.000 description 1
- 102000007260 Deoxyribonuclease I Human genes 0.000 description 1
- 108010000916 Fimbriae Proteins Proteins 0.000 description 1
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 1
- 241001143970 Pelina Species 0.000 description 1
- 108010034546 Serratia marcescens nuclease Proteins 0.000 description 1
- 239000012228 culture supernatant Substances 0.000 description 1
- 229910001629 magnesium chloride Inorganic materials 0.000 description 1
- 239000006228 supernatant Substances 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024196799A JP2025032110A (ja) | 2019-05-24 | 2024-11-11 | 改変ウイルス粒子およびその使用 |
| JP2025085876A JP2025119015A (ja) | 2019-05-24 | 2025-05-22 | 改変ウイルス粒子およびその使用 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962852791P | 2019-05-24 | 2019-05-24 | |
| US62/852,791 | 2019-05-24 | ||
| PCT/US2020/034328 WO2020242984A1 (en) | 2019-05-24 | 2020-05-22 | Modified viral particles and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024196799A Division JP2025032110A (ja) | 2019-05-24 | 2024-11-11 | 改変ウイルス粒子およびその使用 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022533438A JP2022533438A (ja) | 2022-07-22 |
| JPWO2020242984A5 true JPWO2020242984A5 (enExample) | 2023-05-30 |
Family
ID=71094822
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021569402A Pending JP2022533438A (ja) | 2019-05-24 | 2020-05-22 | 改変ウイルス粒子およびその使用 |
| JP2024196799A Pending JP2025032110A (ja) | 2019-05-24 | 2024-11-11 | 改変ウイルス粒子およびその使用 |
| JP2025085876A Pending JP2025119015A (ja) | 2019-05-24 | 2025-05-22 | 改変ウイルス粒子およびその使用 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024196799A Pending JP2025032110A (ja) | 2019-05-24 | 2024-11-11 | 改変ウイルス粒子およびその使用 |
| JP2025085876A Pending JP2025119015A (ja) | 2019-05-24 | 2025-05-22 | 改変ウイルス粒子およびその使用 |
Country Status (19)
| Country | Link |
|---|---|
| US (1) | US20220241430A1 (enExample) |
| EP (1) | EP3976631A1 (enExample) |
| JP (3) | JP2022533438A (enExample) |
| KR (1) | KR20220011664A (enExample) |
| CN (2) | CN114989267A (enExample) |
| AR (2) | AR118997A1 (enExample) |
| AU (1) | AU2020283537A1 (enExample) |
| BR (1) | BR112021023692A2 (enExample) |
| CA (1) | CA3140386A1 (enExample) |
| CL (2) | CL2021003096A1 (enExample) |
| CO (1) | CO2021017692A2 (enExample) |
| IL (1) | IL288233A (enExample) |
| MA (1) | MA56035A (enExample) |
| MX (1) | MX2021014338A (enExample) |
| PE (1) | PE20212357A1 (enExample) |
| PH (1) | PH12021552887A1 (enExample) |
| SG (1) | SG11202112917PA (enExample) |
| TW (1) | TW202110869A (enExample) |
| WO (1) | WO2020242984A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12421525B2 (en) | 2018-02-28 | 2025-09-23 | University of Pittsburgh—of the Commonwealth System of Higher Education | Conjugates comprising AAVs and CAS9 polypeptides |
| AR118997A1 (es) * | 2019-05-24 | 2021-11-17 | Regeneron Pharma | Partículas virales modificadas y usos de estas |
| WO2022187377A1 (en) * | 2021-03-02 | 2022-09-09 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Adeno-associated viruses and methods and materials for making and using adeno-associated viruses |
| WO2022232575A1 (en) * | 2021-04-30 | 2022-11-03 | Duke University | Compositions comprising adeno-associated virus chimera capsid proteins and methods of using the same |
| JP2024525205A (ja) * | 2021-06-23 | 2024-07-10 | ダイノ セラピューティクス インコーポレイテッド | カプシドバリアント及びそれを使用する方法 |
| AU2023269134A1 (en) | 2022-05-09 | 2024-12-12 | Regeneron Pharmaceuticals, Inc. | Vectors and methods for in vivo antibody production |
| EP4536836A1 (en) | 2022-06-07 | 2025-04-16 | Regeneron Pharmaceuticals, Inc. | Pseudotyped viral particles for targeting tcr-expressing cells |
| WO2025042462A2 (en) * | 2023-08-24 | 2025-02-27 | Ohio State Innovation Foundation | Assays and methods for quality control of genome packaging for gene therapy |
| WO2025054526A1 (en) * | 2023-09-07 | 2025-03-13 | Regeneron Pharmaceuticals, Inc. | Production and purification of covalently surface modified adeno-associated virus |
| WO2025111473A1 (en) * | 2023-11-21 | 2025-05-30 | Regeneron Pharmaceuticals, Inc. | Covalently surface modified adeno-associated virus production by in vitro conjugation, and purification of covalently surface modified adeno-associated virus |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1997005266A1 (en) | 1995-07-25 | 1997-02-13 | Introgene B.V. | Methods and means for targeted gene delivery |
| CA2265460A1 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| EP1082413B1 (en) * | 1998-05-28 | 2008-07-23 | THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav5 vector and uses thereof |
| AU768729B2 (en) | 1998-11-05 | 2004-01-08 | Trustees Of The University Of Pennsylvania, The | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ATE317916T1 (de) * | 2001-11-13 | 2006-03-15 | Univ Pennsylvania | Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode |
| ES2602610T3 (es) | 2007-05-31 | 2017-02-21 | Medigene Ag | Proteína estructural mutada de un parvovirus |
| CN104131035A (zh) | 2009-12-10 | 2014-11-05 | 瑞泽恩制药公司 | 生产重链抗体的小鼠 |
| GB201002362D0 (en) | 2010-02-11 | 2010-03-31 | Isis Innovation | Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds |
| CN110885854A (zh) * | 2013-03-15 | 2020-03-17 | 北卡罗来纳-查佩尔山大学 | 双重聚糖结合aav载体的方法和组合物 |
| MX385339B (es) * | 2014-03-10 | 2025-03-18 | Uniqure Ip Bv | Vectores aav mejorados adicionales producidos en células de insecto. |
| BR112016021572A2 (pt) | 2014-03-21 | 2017-10-03 | Regeneron Pharma | Métodos para preparar um animal não humano geneticamente modificado e para produzir proteínas de ligação, hibridoma, ácido nucleico, célula, e, proteína de ligação |
| US10577627B2 (en) * | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| JP2020525020A (ja) * | 2017-06-27 | 2020-08-27 | リジェネロン・ファーマシューティカルズ・インコーポレイテッドＲｅｇｅｎｅｒｏｎ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． | ヒト細胞への遺伝物質の標的化導入のための指向性改変組換えウイルス粒子およびそれらの使用 |
| RS65569B1 (sr) | 2017-06-27 | 2024-06-28 | Regeneron Pharma | Rekombinantni virusni vektori modifikovani tropizmom i njihove upotrebe za ciljano uvođenje genetičkog materijala u humane ćelije |
| AR118997A1 (es) * | 2019-05-24 | 2021-11-17 | Regeneron Pharma | Partículas virales modificadas y usos de estas |
-
2020
- 2020-05-22 AR ARP200101465A patent/AR118997A1/es unknown
- 2020-05-22 CN CN202210622571.3A patent/CN114989267A/zh active Pending
- 2020-05-22 JP JP2021569402A patent/JP2022533438A/ja active Pending
- 2020-05-22 CN CN202080038612.3A patent/CN113874386A/zh active Pending
- 2020-05-22 KR KR1020217041074A patent/KR20220011664A/ko active Pending
- 2020-05-22 SG SG11202112917PA patent/SG11202112917PA/en unknown
- 2020-05-22 CA CA3140386A patent/CA3140386A1/en active Pending
- 2020-05-22 BR BR112021023692A patent/BR112021023692A2/pt unknown
- 2020-05-22 PE PE2021001934A patent/PE20212357A1/es unknown
- 2020-05-22 US US17/612,669 patent/US20220241430A1/en active Pending
- 2020-05-22 MA MA056035A patent/MA56035A/fr unknown
- 2020-05-22 TW TW109117155A patent/TW202110869A/zh unknown
- 2020-05-22 MX MX2021014338A patent/MX2021014338A/es unknown
- 2020-05-22 EP EP20733099.4A patent/EP3976631A1/en active Pending
- 2020-05-22 WO PCT/US2020/034328 patent/WO2020242984A1/en not_active Ceased
- 2020-05-22 AU AU2020283537A patent/AU2020283537A1/en active Pending
- 2020-05-22 PH PH1/2021/552887A patent/PH12021552887A1/en unknown
-
2021
- 2021-11-18 IL IL288233A patent/IL288233A/en unknown
- 2021-11-19 AR ARP210103220A patent/AR124119A2/es unknown
- 2021-11-23 CL CL2021003096A patent/CL2021003096A1/es unknown
- 2021-12-22 CO CONC2021/0017692A patent/CO2021017692A2/es unknown
-
2023
- 2023-03-03 CL CL2023000629A patent/CL2023000629A1/es unknown
-
2024
- 2024-11-11 JP JP2024196799A patent/JP2025032110A/ja active Pending
-
2025
- 2025-05-22 JP JP2025085876A patent/JP2025119015A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP6994018B2 (ja) | 新規アデノ随伴ウイルスキャプシドタンパク質 | |
| JP2023171525A (ja) | Ａａｖキメラ | |
| US20240197918A1 (en) | Tissue-targeted modified aav capsids and methods of use thereof | |
| IL317938A (en) | Recombinant viral vectors with altered tropism and their uses for the targeted introduction of genetic material into human cells | |
| CN112041442A (zh) | 用于逃避抗体的病毒载体的方法和组合物 | |
| KR102612563B1 (ko) | 변형된 포스포리파제 도메인을 갖는 아데노-연관 바이러스 (aav) | |
| US20230020565A1 (en) | Novel compositions and methods for producing recombinant aav | |
| JPWO2020223231A5 (enExample) | ||
| JPWO2020242984A5 (enExample) | ||
| CN116249771A (zh) | 改进的腺相关病毒基因治疗载体 | |
| US20250353883A1 (en) | Novel recombinant aav vp2 fusion polypeptides | |
| Coura et al. | A role for adeno-associated viral vectors in gene therapy | |
| AR123948A1 (es) | ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES | |
| CN106701691A (zh) | 一种可高效感染免疫细胞的aav病毒及其制备方法与应用 | |
| US12312606B2 (en) | Nucleic acid constructs for VA RNA transcription | |
| JPWO2020168222A5 (enExample) | ||
| WO2024078584A1 (zh) | 腺相关病毒的衣壳蛋白编码基因改造方法 | |
| RU2021133286A (ru) | Модифицированные вирусные частицы и пути их применения | |
| US20210292373A1 (en) | Aav vp1u chimeras | |
| JPWO2020041498A5 (enExample) | ||
| JPWO2023081850A5 (enExample) | ||
| RU2024111148A (ru) | Вирусные частицы, перенацеленные на скелетные мышцы | |
| Xu et al. | Capsid modification of adeno-associated virus and tumor targeting gene therapy | |
| JPWO2020047472A5 (enExample) | ||
| RU2020101596A (ru) | Рекомбинантные вирусные частицы с модифицированным тропизмом и пути их применения для нацеленного введения генетического материала в клетки человека |