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- Withdrawn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) | EP3221453A4 (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 (66)
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| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
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| CA2967367C (en) | 2014-11-14 | 2023-05-16 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| US10570395B2 (en) | 2014-11-14 | 2020-02-25 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| KR20240063169A (ko) * | 2014-11-21 | 2024-05-10 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 중추 신경계에 표적화된 aav 벡터 |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| 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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| RU2726131C2 (ru) | 2015-05-22 | 2020-07-09 | Дмитрий Дмитриевич Генкин | Внеклеточная днк в качестве терапевтической мишени при нейродегенерации |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
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| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
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| US12410441B2 (en) | 2018-06-12 | 2025-09-09 | The University Of North Carolina At Chapel Hill | Synthetic liver-tropic adeno-associated virus capsids and uses thereof |
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| US20070026076A1 (en) | 2003-02-24 | 2007-02-01 | Tzyy-Choou Wu | Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins |
| JP4979067B2 (ja) | 2003-05-01 | 2012-07-18 | ジェンザイム・コーポレーション | 神経代謝性疾患についての遺伝子治療 |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| CA3072423A1 (en) | 2003-09-30 | 2005-04-14 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| MXPA06007269A (es) | 2003-12-23 | 2007-07-09 | Univ Pennsylvania | Composiciones y metodos para terapia combinada de enfermedad. |
| 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 |
| WO2006121532A2 (en) | 2005-03-31 | 2006-11-16 | Mytogen, Inc. | Treatment for heart disease |
| EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
| WO2007089632A2 (en) * | 2006-01-27 | 2007-08-09 | The University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| CN103849629B (zh) * | 2006-06-21 | 2017-06-09 | 尤尼克尔Ip股份有限公司 | 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体 |
| 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 |
| DK2561073T3 (en) * | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| 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 |
| US10738326B2 (en) * | 2010-10-27 | 2020-08-11 | Jichi Medical University | Adeno-associated virus vector for gene transfer to nervous system cells |
| KR101830651B1 (ko) | 2011-01-04 | 2018-02-21 | 엘지전자 주식회사 | 정보 표시 장치 및 그 방법 |
| ES2814901T3 (es) * | 2012-09-28 | 2021-03-29 | Univ North Carolina Chapel Hill | Vectores de AAV dirigidos a oligodendrocitos |
| KR20240063169A (ko) | 2014-11-21 | 2024-05-10 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 중추 신경계에 표적화된 aav 벡터 |
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2015
- 2015-11-20 KR KR1020247013508A patent/KR20240063169A/ko active Pending
- 2015-11-20 EP EP15861347.1A patent/EP3221453A4/en not_active Ceased
- 2015-11-20 AU AU2015349759A patent/AU2015349759B2/en active Active
- 2015-11-20 MX MX2017006652A patent/MX2017006652A/es unknown
- 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 WO PCT/US2015/061788 patent/WO2016081811A1/en not_active Ceased
- 2015-11-20 US US15/525,214 patent/US10532110B2/en active Active
- 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 EP EP23199459.1A patent/EP4344741A3/en active Pending
- 2015-11-20 RU RU2017121593A patent/RU2727015C2/ru active
- 2015-11-20 CN CN202210236316.5A patent/CN114480440A/zh active Pending
- 2015-11-20 CA CA2967393A patent/CA2967393A1/en active Pending
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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
- 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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