JPWO2021226008A5 - - Google Patents
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- Publication number
- JPWO2021226008A5 JPWO2021226008A5 JP2022567634A JP2022567634A JPWO2021226008A5 JP WO2021226008 A5 JPWO2021226008 A5 JP WO2021226008A5 JP 2022567634 A JP2022567634 A JP 2022567634A JP 2022567634 A JP2022567634 A JP 2022567634A JP WO2021226008 A5 JPWO2021226008 A5 JP WO2021226008A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- nucleic acid
- particle
- vector
- capsid 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
- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 claims 16
- 241000702421 Dependoparvovirus Species 0.000 claims 16
- 239000002245 particle Substances 0.000 claims 16
- 108020004707 nucleic acids Proteins 0.000 claims 15
- 102000039446 nucleic acids Human genes 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 108090000565 Capsid Proteins Proteins 0.000 claims 9
- 102100023321 Ceruloplasmin Human genes 0.000 claims 9
- 239000013607 AAV vector Substances 0.000 claims 6
- 239000013598 vector Substances 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 4
- 210000000234 capsid Anatomy 0.000 claims 3
- 210000004185 liver Anatomy 0.000 claims 3
- 230000035772 mutation Effects 0.000 claims 3
- 210000003494 hepatocyte Anatomy 0.000 claims 2
- 238000000338 in vitro Methods 0.000 claims 2
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 2
- 229920001184 polypeptide Polymers 0.000 claims 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims 2
- 108090000765 processed proteins & peptides Proteins 0.000 claims 2
- 239000013603 viral vector Substances 0.000 claims 2
- 230000003612 virological effect Effects 0.000 claims 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 102000009027 Albumins Human genes 0.000 claims 1
- 108010088751 Albumins Proteins 0.000 claims 1
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 102000009081 Apolipoprotein A-II Human genes 0.000 claims 1
- 108010087614 Apolipoprotein A-II Proteins 0.000 claims 1
- 102000004328 Cytochrome P-450 CYP3A Human genes 0.000 claims 1
- 108010081668 Cytochrome P-450 CYP3A Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 102000053602 DNA Human genes 0.000 claims 1
- 108091007780 MiR-122 Proteins 0.000 claims 1
- 108700011259 MicroRNAs Proteins 0.000 claims 1
- 102000009572 RNA Polymerase II Human genes 0.000 claims 1
- 108010009460 RNA Polymerase II Proteins 0.000 claims 1
- 102000014450 RNA Polymerase III Human genes 0.000 claims 1
- 108010078067 RNA Polymerase III Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 0.000 claims 1
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims 1
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims 1
- 241000700605 Viruses Species 0.000 claims 1
- 229940050528 albumin Drugs 0.000 claims 1
- 102000015395 alpha 1-Antitrypsin Human genes 0.000 claims 1
- 108010050122 alpha 1-Antitrypsin Proteins 0.000 claims 1
- 229940024142 alpha 1-antitrypsin Drugs 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 210000004436 artificial bacterial chromosome Anatomy 0.000 claims 1
- 210000001106 artificial yeast chromosome Anatomy 0.000 claims 1
- 150000001720 carbohydrates Chemical class 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 230000001939 inductive effect Effects 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 239000013612 plasmid Substances 0.000 claims 1
- 102000004169 proteins and genes Human genes 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 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 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 241000701447 unidentified baculovirus Species 0.000 claims 1
- 241001529453 unidentified herpesvirus Species 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063020139P | 2020-05-05 | 2020-05-05 | |
| US63/020,139 | 2020-05-05 | ||
| PCT/US2021/030547 WO2021226008A1 (en) | 2020-05-05 | 2021-05-04 | Modified adeno-associated virus 5 capsids and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023524577A JP2023524577A (ja) | 2023-06-12 |
| JPWO2021226008A5 true JPWO2021226008A5 (https=) | 2024-05-15 |
| JP2023524577A5 JP2023524577A5 (https=) | 2024-05-15 |
Family
ID=78412297
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022567634A Pending JP2023524577A (ja) | 2020-05-05 | 2021-05-04 | 改変アデノ随伴ウイルス５カプシドおよびその使用 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US12209251B2 (https=) |
| EP (1) | EP4146673A4 (https=) |
| JP (1) | JP2023524577A (https=) |
| KR (1) | KR20230034211A (https=) |
| CN (1) | CN115916986A (https=) |
| AU (1) | AU2021267856A1 (https=) |
| CA (1) | CA3177268A1 (https=) |
| MX (1) | MX2022013819A (https=) |
| WO (1) | WO2021226008A1 (https=) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CN113121651B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 低中和抗体腺相关病毒衣壳蛋白 |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| MX2024004936A (es) | 2021-11-02 | 2024-05-08 | Voyager Therapeutics Inc | Variantes de capsides de aav y sus usos. |
| US20250084129A1 (en) * | 2022-01-10 | 2025-03-13 | University Of Florida Research Foundation, Incorporated | Aav5 capsid variants |
| AR129843A1 (es) * | 2022-07-06 | 2024-10-02 | Voyager Therapeutics Inc | Variantes de la cápside de aav y usos de estas |
| CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| CN119570747A (zh) * | 2024-12-06 | 2025-03-07 | 中国科学院深圳先进技术研究院 | 一种用于基因与细胞治疗的新型腺相关病毒变体库及其构建和应用 |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4474893A (en) | 1981-07-01 | 1984-10-02 | The University of Texas System Cancer Center | Recombinant monoclonal antibodies |
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| US4816567A (en) | 1983-04-08 | 1989-03-28 | Genentech, Inc. | Recombinant immunoglobin preparations |
| US4676980A (en) | 1985-09-23 | 1987-06-30 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Target specific cross-linked heteroantibodies |
| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| WO1990005142A1 (en) | 1988-11-10 | 1990-05-17 | Imperial Cancer Research Technology Ltd. | Polypeptides |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| DE69636937T3 (de) | 1995-12-15 | 2011-01-05 | Virxsys Corp. | Durch trans-spaltung erhaltene therapeutische molekule |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| 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 |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| 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 |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| JP2000325085A (ja) | 1999-05-21 | 2000-11-28 | Masafumi Matsuo | デュシェンヌ型筋ジストロフィー治療剤 |
| US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ522840A (en) | 2000-06-01 | 2004-12-24 | Univ North Carolina | A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together |
| US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| WO2005047501A1 (en) | 2003-02-24 | 2005-05-26 | Johns Hopkins University | Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP6396988B2 (ja) * | 2013-03-15 | 2018-09-26 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二重グリカン結合性ａａｖベクターのための方法および組成物 |
| KR102234672B1 (ko) * | 2013-05-21 | 2021-04-06 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| WO2016081811A1 (en) * | 2014-11-21 | 2016-05-26 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| WO2016126857A1 (en) | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| US10081659B2 (en) * | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| JP2019529499A (ja) * | 2016-09-30 | 2019-10-17 | ベイラー カレッジ オブ メディスンＢａｙｌｏｒ Ｃｏｌｌｅｇｅ Ｏｆ Ｍｅｄｉｃｉｎｅ | 組織指向性発現を用いた抗体ベースの遺伝子治療 |
| JP2021523702A (ja) | 2018-05-04 | 2021-09-09 | オレゴン ヘルス アンド サイエンス ユニバーシティ | ヒト抗ａａｖ２カプシドポリクローナル抗体エピトープ |
| CN112566923B (zh) * | 2018-06-12 | 2024-10-29 | 北卡罗来纳大学教堂山分校 | 合成嗜肝性腺相关病毒衣壳及其用途 |
| EP4461814A3 (en) * | 2018-10-12 | 2025-02-26 | Genzyme Corporation | Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy |
-
2021
- 2021-05-04 US US17/307,117 patent/US12209251B2/en active Active
- 2021-05-04 AU AU2021267856A patent/AU2021267856A1/en not_active Abandoned
- 2021-05-04 CN CN202180047746.6A patent/CN115916986A/zh active Pending
- 2021-05-04 KR KR1020227042655A patent/KR20230034211A/ko active Pending
- 2021-05-04 WO PCT/US2021/030547 patent/WO2021226008A1/en not_active Ceased
- 2021-05-04 MX MX2022013819A patent/MX2022013819A/es unknown
- 2021-05-04 CA CA3177268A patent/CA3177268A1/en active Pending
- 2021-05-04 JP JP2022567634A patent/JP2023524577A/ja active Pending
- 2021-05-04 EP EP21800414.1A patent/EP4146673A4/en not_active Withdrawn
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