JP2017536116A5 - - Google Patents
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- 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- Withdrawn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 (67)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| RU2727015C2 (ru) | 2014-11-21 | 2020-07-17 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Векторы aav, нацеленные на центральную нервную систему |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | 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 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| JP6745873B2 (ja) | 2015-05-22 | 2020-08-26 | ドミトリエヴィッチ ゲンキン，ドミトリー | 神経変性における治療標的としての細胞外ｄｎａ |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| US10983110B2 (en) | 2015-12-02 | 2021-04-20 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
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| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
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| BR112019002904A2 (pt) * | 2016-08-16 | 2019-06-25 | The University Of North Carolina At Chapel Hill | métodos e composições para transferência gênica direcionada |
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| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
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| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
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| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| WO2019094061A1 (en) * | 2017-11-07 | 2019-05-16 | The University Of North Carolina At Chapel Hill | Optimized aga genes and expression cassettes and their use |
| KR20200100735A (ko) * | 2017-12-20 | 2020-08-26 | 오비드 테라퓨틱스 인크. | 발작 장애 치료 시 hM4Di의 용도 |
| JP2021510539A (ja) | 2018-01-16 | 2021-04-30 | シーエルエス セラピューティクス リミテッドＣＬＳ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｌｉｍｉｔｅｄ | デオキシリボヌクレアーゼ（ＤＮａｓｅ）活性を有する酵素の肝臓発現による疾患の処置 |
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| 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. |
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| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
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| JP6387350B2 (ja) * | 2012-09-28 | 2018-09-05 | 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 | 希突起神経膠細胞を標的とするアデノ随伴ウイルスベクター |
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2015
- 2015-11-20 RU RU2017121593A patent/RU2727015C2/ru active
- 2015-11-20 WO PCT/US2015/061788 patent/WO2016081811A1/en not_active Ceased
- 2015-11-20 KR KR1020247013508A patent/KR20240063169A/ko active Pending
- 2015-11-20 BR BR112017010447-4A patent/BR112017010447A2/pt not_active Application Discontinuation
- 2015-11-20 CN CN201580074023.XA patent/CN107532173A/zh active Pending
- 2015-11-20 US US15/525,214 patent/US10532110B2/en active Active
- 2015-11-20 MX MX2017006652A patent/MX2017006652A/es unknown
- 2015-11-20 CN CN202210236316.5A patent/CN114480440A/zh active Pending
- 2015-11-20 EP EP23199459.1A patent/EP4344741A3/en active Pending
- 2015-11-20 AU AU2015349759A patent/AU2015349759B2/en active Active
- 2015-11-20 CA CA2967393A patent/CA2967393A1/en active Pending
- 2015-11-20 JP JP2017525107A patent/JP2017536116A/ja not_active Withdrawn
- 2015-11-20 KR KR1020177014680A patent/KR20170086537A/ko not_active Ceased
- 2015-11-20 EP EP15861347.1A patent/EP3221453A4/en not_active Ceased
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2017
- 2017-05-03 IL IL252072A patent/IL252072B/en unknown
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2019
- 2019-06-13 US US16/440,638 patent/US10561743B2/en active Active
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2020
- 2020-01-13 US US16/741,324 patent/US11491242B2/en active Active
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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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- 2023-06-30 JP JP2023108201A patent/JP2023123765A/ja active Pending
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