JP2023123765A5 - - Google Patents
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- JP2023123765A5 JP2023123765A5 JP2023108201A JP2023108201A JP2023123765A5 JP 2023123765 A5 JP2023123765 A5 JP 2023123765A5 JP 2023108201 A JP2023108201 A JP 2023108201A JP 2023108201 A JP2023108201 A JP 2023108201A JP 2023123765 A5 JP2023123765 A5 JP 2023123765A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- nucleic acid
- particles
- polypeptide
- capsid
- 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 33
- 150000007523 nucleic acids Chemical class 0.000 claims 26
- 108020004707 nucleic acids Proteins 0.000 claims 25
- 102000039446 nucleic acids Human genes 0.000 claims 25
- 239000002245 particle Substances 0.000 claims 23
- 210000000234 capsid Anatomy 0.000 claims 13
- 238000000034 method Methods 0.000 claims 12
- 229920001184 polypeptide Polymers 0.000 claims 9
- 102000004196 processed proteins & peptides Human genes 0.000 claims 9
- 108090000765 processed proteins & peptides Proteins 0.000 claims 9
- 239000013607 AAV vector Substances 0.000 claims 6
- 210000004027 cell Anatomy 0.000 claims 6
- 241000700605 Viruses Species 0.000 claims 5
- 108091026890 Coding region Proteins 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 238000000338 in vitro Methods 0.000 claims 3
- 108090000565 Capsid Proteins Proteins 0.000 claims 2
- 102100023321 Ceruloplasmin Human genes 0.000 claims 2
- 150000001413 amino acids Chemical class 0.000 claims 2
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 230000001939 inductive effect Effects 0.000 claims 2
- 230000010415 tropism Effects 0.000 claims 2
- 108020005544 Antisense RNA Proteins 0.000 claims 1
- 108700011259 MicroRNAs Proteins 0.000 claims 1
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 1
- 108091030071 RNAI Proteins 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
- 210000004556 brain Anatomy 0.000 claims 1
- 239000003184 complementary RNA Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 230000009368 gene silencing by RNA Effects 0.000 claims 1
- 208000015181 infectious disease Diseases 0.000 claims 1
- 239000002679 microRNA Substances 0.000 claims 1
- 239000000825 pharmaceutical preparation Substances 0.000 claims 1
- 239000013612 plasmid Substances 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
- 239000013603 viral vector Substances 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 (6)
| 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 | ||
| JP2017525107A 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 JP2021097675A (ja) | 2014-11-21 | 2021-02-10 | 中枢神経系を標的化したａａｖベクター |
Related Parent 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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025015920A Division JP2025072468A (ja) | 2014-11-21 | 2025-02-03 | 中枢神経系を標的化したａａｖベクター |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023123765A JP2023123765A (ja) | 2023-09-05 |
| JP2023123765A5 true JP2023123765A5 (enExample) | 2023-11-24 |
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 Before (2)
| 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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
|---|---|---|---|---|
| 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 |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| JP2019517274A (ja) | 2016-06-13 | 2019-06-24 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルＴｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｎｏｒｔｈ Ｃａｒｏｌｉｎａ Ａｔ Ｃｈａｐｅｌ Ｈｉｌｌ | 最適化されたｃｌｎ１遺伝子および発現カセットおよびそれらの使用 |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| BR112019002904A2 (pt) * | 2016-08-16 | 2019-06-25 | The University Of North Carolina At Chapel Hill | métodos e composições para transferência gênica direcionada |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| 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 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| CN108676097B (zh) * | 2018-05-24 | 2020-01-14 | 北京肽和生物科技有限公司 | 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用 |
| EP4659766A2 (en) * | 2018-06-12 | 2025-12-10 | The University of North Carolina at Chapel Hill | Synthetic liver-tropic adeno-associated virus capsids and uses thereof |
| BR112020025268A2 (pt) | 2018-06-28 | 2021-03-09 | The University Of North Carolina At Chapel Hill | Cassetes de expressão e genes cln5 otimizados e seu uso |
| IL280300B2 (en) | 2018-08-10 | 2025-06-01 | Univ North Carolina Chapel Hill | Optimized cln7 genes and expression cassettes and their use |
| KR20210084459A (ko) | 2018-09-26 | 2021-07-07 | 캘리포니아 인스티튜트 오브 테크놀로지 | 표적화된 유전자 요법을 위한 아데노-연관 바이러스 조성물 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019354995B2 (en) * | 2018-10-02 | 2024-12-12 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| WO2020097002A1 (en) | 2018-11-05 | 2020-05-14 | The University Of North Carolina At Chapel Hill | Optimized fig4 genes and expression cassettes and their use |
| JP7590968B2 (ja) * | 2018-12-05 | 2024-11-27 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
| EP3884057A4 (en) | 2018-12-21 | 2022-09-07 | The University of North Carolina at Chapel Hill | OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE |
| PH12021552608A1 (en) * | 2019-04-29 | 2022-06-20 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| JP7616668B2 (ja) | 2019-05-22 | 2025-01-17 | 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 | Ｕｂｅ３ａ遺伝子および発現カセットならびにそれらの使用 |
| US20220241394A1 (en) * | 2019-07-12 | 2022-08-04 | Cls Therapeutics Limited | TREATMENT OF DISEASES ASSOCIATED WITH PROTEIN MISFOLDING BY NERVOUS SYSTEM EXPRESSION OF AN ENZYME WHICH HAS A DEOXYRIBONUCLEASE (DNase) ACTIVITY |
| CA3151087A1 (en) * | 2019-08-29 | 2021-03-04 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-associated viral vectors for crossing the human blood brain barrier |
| EP4025258A4 (en) * | 2019-09-03 | 2023-09-06 | University of Cincinnati | METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| JP2023515795A (ja) * | 2020-02-18 | 2023-04-14 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | Ａａｖカプシド－プロモーター相互作用および細胞選択的遺伝子発現 |
| EP4143305A4 (en) * | 2020-05-01 | 2024-09-11 | The Broad Institute, Inc. | GENETICALLY MODIFIED CENTRAL NERVOUS SYSTEM COMPOSITIONS |
| WO2021225921A1 (en) * | 2020-05-04 | 2021-11-11 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof |
| JP2023524577A (ja) * | 2020-05-05 | 2023-06-12 | 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 | 改変アデノ随伴ウイルス５カプシドおよびその使用 |
| CN115806596A (zh) * | 2020-05-22 | 2023-03-17 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 腺相关病毒突变体及其应用 |
| WO2022011390A1 (en) | 2020-07-08 | 2022-01-13 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
| JP2023534026A (ja) | 2020-07-14 | 2023-08-07 | アビオナ・セラピュ―ティクス・インコーポレイテッド | 多部分遺伝子送達のための組み換えアデノ随伴ウイルスベクター |
| US20230159949A1 (en) * | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| US20230285595A1 (en) | 2020-07-23 | 2023-09-14 | The University Of North Carolina At Chapel Hill | Optimized slc13a5 genes and expression cassettes and their use |
| WO2022034130A1 (en) * | 2020-08-12 | 2022-02-17 | UCB Biopharma SRL | Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| CN113563430B (zh) * | 2021-07-30 | 2022-05-31 | 上海信致医药科技有限公司 | 用于治疗眼部疾病的基因递送系统及其应用 |
| KR20240113624A (ko) | 2021-11-02 | 2024-07-22 | 보이저 테라퓨틱스, 인크. | Aav 캡시드 변이체 및 이의 용도 |
| WO2023183304A2 (en) * | 2022-03-21 | 2023-09-28 | Capsigen Inc. | Transcription-dependent directed evolution of aav capsids having enhanced tropism |
| WO2025015388A1 (en) * | 2023-07-20 | 2025-01-23 | Children's Medical Research Institute | Modified cardiotropic aav capsid polypeptides and vectors |
| WO2025084951A1 (en) * | 2023-10-16 | 2025-04-24 | Joint Stock Company "Axelgen" | Recombinant genetic construct, adeno-associated virus for the treatment of metachromatic leukodystrophy |
| WO2025147741A1 (en) * | 2024-01-11 | 2025-07-17 | Children's Medical Research Institute | Aav capsids and vectors for transduction of cells |
Family Cites Families (59)
| 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 |
| US5023243A (en) | 1981-10-23 | 1991-06-11 | Molecular Biosystems, Inc. | Oligonucleotide therapeutic agent and method of making same |
| 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 |
| JPH01503675A (ja) | 1986-09-08 | 1989-12-14 | アプライド・バイオテクノロジー・インコーポレーテツド | 中空ウイルスキヤプシドのワクチン |
| US4987071A (en) | 1986-12-03 | 1991-01-22 | University Patents, Inc. | RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| EP0442926A1 (en) | 1988-11-10 | 1991-08-28 | Imperial Cancer Research Technology Limited | 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 |
| US5149797A (en) | 1990-02-15 | 1992-09-22 | The Worcester Foundation For Experimental Biology | Method of site-specific alteration of rna and production of encoded polypeptides |
| 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 |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| EP0883344B2 (en) | 1995-12-15 | 2010-06-09 | VIRxSYS Corporation | Therapeutic molecules generated by trans-splicing |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE 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 |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| NZ511171A (en) | 1998-09-22 | 2004-02-27 | Univ Florida | Methods for large-scale production of recombinant AAV vectors |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| 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 |
| US6582692B1 (en) | 1999-11-17 | 2003-06-24 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
| WO2001077350A2 (en) | 2000-04-07 | 2001-10-18 | Large Scale Biology Corporation | Compositions and methods for inhibiting gene expression |
| US6653467B1 (en) | 2000-04-26 | 2003-11-25 | Jcr Pharmaceutical Co., Ltd. | Medicament for treatment of Duchenne muscular dystrophy |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ES2256265T3 (es) | 2000-06-01 | 2006-07-16 | University Of North Carolina At Chapel Hill | Vectores de parvovirus duplicados. |
| US6727355B2 (en) | 2000-08-25 | 2004-04-27 | Jcr Pharmaceuticals Co., Ltd. | Pharmaceutical composition for treatment of Duchenne muscular dystrophy |
| US20030083299A1 (en) | 2000-11-04 | 2003-05-01 | Ferguson Ian A. | Non-invasive delivery of polypeptides through the blood-brain barrier |
| 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 |
| JP4769417B2 (ja) * | 2001-12-17 | 2011-09-07 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用 |
| 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. |
| US20070026076A1 (en) | 2003-02-24 | 2007-02-01 | Tzyy-Choou Wu | Molecular vaccines employing nucleic acid encoding anti-apoptotic proteins |
| PL1620133T3 (pl) | 2003-05-01 | 2016-05-31 | Genzyme Corp | Terapia genowa dla zaburzeń neurometabolicznych |
| 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 |
| 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 |
| 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 |
| EP1865992A2 (en) | 2005-03-31 | 2007-12-19 | Mytogen Inc. | Treatment for heart disease |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7867484B2 (en) * | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| US8512981B2 (en) * | 2006-06-21 | 2013-08-20 | Amsterdam Molecular Therapeutics B.V. | Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV |
| 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 |
| EP3514232A1 (en) * | 2010-04-23 | 2019-07-24 | University of Massachusetts | Cns targeting aav vectors 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 |
| CN107828820B (zh) * | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| KR101830651B1 (ko) | 2011-01-04 | 2018-02-21 | 엘지전자 주식회사 | 정보 표시 장치 및 그 방법 |
| 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 |
| RU2727015C2 (ru) * | 2014-11-21 | 2020-07-17 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Векторы aav, нацеленные на центральную нервную систему |
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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
- 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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