JP2017536116A5 - - Google Patents
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- Publication number
- JP2017536116A5 JP2017536116A5 JP2017525107A JP2017525107A JP2017536116A5 JP 2017536116 A5 JP2017536116 A5 JP 2017536116A5 JP 2017525107 A JP2017525107 A JP 2017525107A JP 2017525107 A JP2017525107 A JP 2017525107A JP 2017536116 A5 JP2017536116 A5 JP 2017536116A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- nucleic acid
- capsid
- aav capsid
- particles
- 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.)
- Withdrawn
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- 208000002267 Anti-neutrophil cytoplasmic antibody-associated vasculitis Diseases 0.000 claims 51
- 241000702421 Dependoparvovirus Species 0.000 claims 51
- 210000000234 capsid Anatomy 0.000 claims 35
- 108020004707 nucleic acids Proteins 0.000 claims 29
- 102000039446 nucleic acids Human genes 0.000 claims 29
- 150000007523 nucleic acids Chemical class 0.000 claims 29
- 239000002245 particle Substances 0.000 claims 29
- 238000000034 method Methods 0.000 claims 9
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 238000006467 substitution reaction Methods 0.000 claims 8
- 108091026890 Coding region Proteins 0.000 claims 7
- 210000004248 oligodendroglia Anatomy 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 6
- 239000013607 AAV vector Substances 0.000 claims 5
- 241000700605 Viruses Species 0.000 claims 5
- 125000003275 alpha amino acid group Chemical group 0.000 claims 5
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 3
- 210000004027 cell Anatomy 0.000 claims 3
- 238000000338 in vitro Methods 0.000 claims 3
- 108010072388 Methyl-CpG-Binding Protein 2 Proteins 0.000 claims 2
- 108020004414 DNA Proteins 0.000 claims 1
- 102000006890 Methyl-CpG-Binding Protein 2 Human genes 0.000 claims 1
- 208000006289 Rett Syndrome Diseases 0.000 claims 1
- 108700019146 Transgenes Proteins 0.000 claims 1
- 230000008499 blood brain barrier function Effects 0.000 claims 1
- 210000001218 blood-brain barrier Anatomy 0.000 claims 1
- 150000001720 carbohydrates Chemical class 0.000 claims 1
- 235000014633 carbohydrates Nutrition 0.000 claims 1
- 210000003169 central nervous system Anatomy 0.000 claims 1
- 150000001875 compounds Chemical class 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000004064 dysfunction Effects 0.000 claims 1
- 230000001771 impaired 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
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 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
- 230000003612 virological effect Effects 0.000 claims 1
Priority Applications (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021019477A JP2021097675A (ja) | 2014-11-21 | 2021-02-10 | 中枢神経系を標的化したａａｖベクター |
| JP2023108201A JP2023123765A (ja) | 2014-11-21 | 2023-06-30 | 中枢神経系を標的化したａａｖベクター |
| JP2025015920A JP2025072468A (ja) | 2014-11-21 | 2025-02-03 | 中枢神経系を標的化したａａｖベクター |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462082897P | 2014-11-21 | 2014-11-21 | |
| US62/082,897 | 2014-11-21 | ||
| US201562218857P | 2015-09-15 | 2015-09-15 | |
| US62/218,857 | 2015-09-15 | ||
| PCT/US2015/061788 WO2016081811A1 (en) | 2014-11-21 | 2015-11-20 | Aav vectors targeted to the central nervous system |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021019477A Division JP2021097675A (ja) | 2014-11-21 | 2021-02-10 | 中枢神経系を標的化したａａｖベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017536116A JP2017536116A (ja) | 2017-12-07 |
| JP2017536116A5 true JP2017536116A5 (enExample) | 2018-12-13 |
Family
ID=56014589
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017525107A Withdrawn JP2017536116A (ja) | 2014-11-21 | 2015-11-20 | 中枢神経系を標的化したａａｖベクター |
| JP2021019477A Pending JP2021097675A (ja) | 2014-11-21 | 2021-02-10 | 中枢神経系を標的化したａａｖベクター |
| JP2023108201A Pending JP2023123765A (ja) | 2014-11-21 | 2023-06-30 | 中枢神経系を標的化したａａｖベクター |
| JP2025015920A Pending JP2025072468A (ja) | 2014-11-21 | 2025-02-03 | 中枢神経系を標的化したａａｖベクター |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021019477A Pending JP2021097675A (ja) | 2014-11-21 | 2021-02-10 | 中枢神経系を標的化したａａｖベクター |
| JP2023108201A Pending JP2023123765A (ja) | 2014-11-21 | 2023-06-30 | 中枢神経系を標的化したａａｖベクター |
| JP2025015920A Pending JP2025072468A (ja) | 2014-11-21 | 2025-02-03 | 中枢神経系を標的化したａａｖベクター |
Country Status (12)
| Country | Link |
|---|---|
| US (4) | US10532110B2 (enExample) |
| EP (2) | EP4344741A3 (enExample) |
| JP (4) | JP2017536116A (enExample) |
| KR (2) | KR20240063169A (enExample) |
| CN (2) | CN107532173A (enExample) |
| AU (2) | AU2015349759B2 (enExample) |
| BR (1) | BR112017010447A2 (enExample) |
| CA (1) | CA2967393A1 (enExample) |
| IL (1) | IL252072B (enExample) |
| MX (1) | MX2017006652A (enExample) |
| RU (1) | RU2727015C2 (enExample) |
| WO (1) | WO2016081811A1 (enExample) |
Families Citing this family (68)
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| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG10202007103TA (en) | 2014-11-05 | 2020-09-29 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
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| IL284949B2 (en) | 2014-11-14 | 2024-06-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| EP4344741A3 (en) | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| JP6754361B2 (ja) | 2014-12-16 | 2020-09-09 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
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| ES2799515T3 (es) | 2015-05-22 | 2020-12-18 | Dmitry Dmitrievich Genkin | ADN extracelular como una diana terapéutica en la neurodegeneración |
| AU2016332821B2 (en) | 2015-09-28 | 2022-02-17 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
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| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
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| EP3458588B1 (en) | 2016-05-18 | 2025-10-29 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
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| US7807814B2 (en) | 2003-12-23 | 2010-10-05 | The Trustees Of The University Of Pennsylvania | Compositions and methods for combined therapy of disease |
| 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 |
| EP1865992A2 (en) | 2005-03-31 | 2007-12-19 | Mytogen Inc. | Treatment for heart disease |
| CN104293835B (zh) | 2005-04-07 | 2017-07-04 | 宾夕法尼亚大学托管会 | 增强腺相关病毒载体功能的方法 |
| US7867484B2 (en) * | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| WO2007148971A2 (en) * | 2006-06-21 | 2007-12-27 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells |
| EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| US8632764B2 (en) * | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| ES2605305T3 (es) * | 2010-04-23 | 2017-03-13 | University Of Massachusetts | Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos |
| 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 |
| WO2012057363A1 (ja) * | 2010-10-27 | 2012-05-03 | 学校法人自治医科大学 | 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン |
| KR101830651B1 (ko) | 2011-01-04 | 2018-02-21 | 엘지전자 주식회사 | 정보 표시 장치 및 그 방법 |
| EP3738974A1 (en) | 2012-09-28 | 2020-11-18 | The University of North Carolina at Chapel Hill | Aav vectors targeted to oligodendrocytes |
| EP4344741A3 (en) * | 2014-11-21 | 2024-08-28 | The University of North Carolina at Chapel Hill | Aav vectors targeted to the central nervous system |
-
2015
- 2015-11-20 EP EP23199459.1A patent/EP4344741A3/en active Pending
- 2015-11-20 JP JP2017525107A patent/JP2017536116A/ja not_active Withdrawn
- 2015-11-20 CA CA2967393A patent/CA2967393A1/en active Pending
- 2015-11-20 MX MX2017006652A patent/MX2017006652A/es unknown
- 2015-11-20 EP EP15861347.1A patent/EP3221453A4/en not_active Ceased
- 2015-11-20 CN CN201580074023.XA patent/CN107532173A/zh active Pending
- 2015-11-20 BR BR112017010447-4A patent/BR112017010447A2/pt not_active Application Discontinuation
- 2015-11-20 CN CN202210236316.5A patent/CN114480440A/zh active Pending
- 2015-11-20 AU AU2015349759A patent/AU2015349759B2/en active Active
- 2015-11-20 KR KR1020247013508A patent/KR20240063169A/ko active Pending
- 2015-11-20 US US15/525,214 patent/US10532110B2/en active Active
- 2015-11-20 KR KR1020177014680A patent/KR20170086537A/ko not_active Ceased
- 2015-11-20 WO PCT/US2015/061788 patent/WO2016081811A1/en not_active Ceased
- 2015-11-20 RU RU2017121593A patent/RU2727015C2/ru active
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2017
- 2017-05-03 IL IL252072A patent/IL252072B/en unknown
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2019
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2020
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2021
- 2021-02-10 JP JP2021019477A patent/JP2021097675A/ja active Pending
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- 2022-03-04 AU AU2022201540A patent/AU2022201540B2/en active Active
- 2022-09-21 US US17/933,937 patent/US20230263910A1/en active Pending
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2023
- 2023-06-30 JP JP2023108201A patent/JP2023123765A/ja active Pending
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2025
- 2025-02-03 JP JP2025015920A patent/JP2025072468A/ja active Pending
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