JPWO2020113034A5 - - Google Patents
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- JPWO2020113034A5 JPWO2020113034A5 JP2021530078A JP2021530078A JPWO2020113034A5 JP WO2020113034 A5 JPWO2020113034 A5 JP WO2020113034A5 JP 2021530078 A JP2021530078 A JP 2021530078A JP 2021530078 A JP2021530078 A JP 2021530078A JP WO2020113034 A5 JPWO2020113034 A5 JP WO2020113034A5
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- BPYKTIZUTYGOLE-IFADSCNNSA-N bilirubin Chemical compound N1C(=O)C(C)=C(C=C)\C1=C\C1=C(C)C(CCC(O)=O)=C(CC2=C(C(C)=C(\C=C/3C(=C(C=C)C(=O)N\3)C)N2)CCC(O)=O)N1 BPYKTIZUTYGOLE-IFADSCNNSA-N 0.000 claims description 2
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- 229960001025 iohexol Drugs 0.000 claims description 2
- 201000009673 liver disease Diseases 0.000 claims description 2
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- 230000017613 viral reproduction Effects 0.000 claims description 2
- 235000018102 proteins Nutrition 0.000 claims 3
- 108010082126 Alanine Transaminase Proteins 0.000 claims 2
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- 229940098773 Bovine Serum Albumin Drugs 0.000 claims 2
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- 239000012216 imaging agent Substances 0.000 description 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 3
- 241000725303 Human immunodeficiency virus Species 0.000 description 2
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862773894P | 2018-11-30 | 2018-11-30 | |
| US62/773,894 | 2018-11-30 | ||
| US201962835242P | 2019-04-17 | 2019-04-17 | |
| US62/835,242 | 2019-04-17 | ||
| PCT/US2019/063649 WO2020113034A1 (fr) | 2018-11-30 | 2019-11-27 | Vecteurs viraux aav et leurs utilisations |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022511776A JP2022511776A (ja) | 2022-02-01 |
| JPWO2020113034A5 true JPWO2020113034A5 (fr) | 2022-12-01 |
Family
ID=69167903
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021530078A Pending JP2022511776A (ja) | 2018-11-30 | 2019-11-27 | Ａａｖウイルスベクター及びその使用 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20220001028A1 (fr) |
| EP (1) | EP3886919A1 (fr) |
| JP (1) | JP2022511776A (fr) |
| KR (1) | KR20210099025A (fr) |
| CN (1) | CN113226380A (fr) |
| AU (1) | AU2019389047A1 (fr) |
| BR (1) | BR112021009739A2 (fr) |
| CA (1) | CA3116630A1 (fr) |
| IL (1) | IL282885A (fr) |
| MX (1) | MX2021006359A (fr) |
| TW (1) | TW202039859A (fr) |
| WO (1) | WO2020113034A1 (fr) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020037161A1 (fr) * | 2018-08-15 | 2020-02-20 | Biogen Ma Inc. | Polythérapie pour atrophie musculaire spinale |
| TW202140791A (zh) * | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| CN112121179A (zh) * | 2020-09-15 | 2020-12-25 | 山东兴瑞生物科技有限公司 | 一种组合物及其在治疗脊髓性肌萎缩症中的应用 |
| US20240084323A1 (en) * | 2021-01-13 | 2024-03-14 | Dignity Health | Modulation of chitinase protein expression |
| AU2022318664A1 (en) | 2021-07-30 | 2024-02-29 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| WO2023010133A2 (fr) | 2021-07-30 | 2023-02-02 | Tune Therapeutics, Inc. | Compositions et procédés de modulation de l'expression de la frataxine |
| WO2023015189A2 (fr) * | 2021-08-03 | 2023-02-09 | The Regents Of The University Of California | Expression de sphingosine 1-phosphate lyase (spl) médiée par un virus adéno-associé (aav) pour le traitement de la fibrose pulmonaire |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| KR20230152503A (ko) * | 2022-04-27 | 2023-11-03 | 주식회사 헬릭스미스 | 척수강 내 투여에 최적화 된 간세포 성장인자 유전자가 도입된 aav 벡터 |
| WO2023219394A1 (fr) * | 2022-05-10 | 2023-11-16 | 서울대학교산학협력단 | Variant de protéine smn1 humaine et son utilisation |
| WO2023246734A1 (fr) | 2022-06-21 | 2023-12-28 | Skyline Therapeutics (Shanghai) Co., Ltd. | Vaa recombinant pour la thérapie génique de la maladie sma |
| WO2024015881A2 (fr) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | Compositions, systèmes et procédés d'activation transcriptionnelle ciblée |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| DE69433592T2 (de) | 1993-11-09 | 2005-02-10 | Targeted Genetics Corp., Seattle | Die erzielung hoher titer des rekombinanten aav-vektors |
| EP0728214B1 (fr) | 1993-11-09 | 2004-07-28 | Medical College Of Ohio | Lignees cellulaires stables aptes a exprimer le gene de replication du virus adeno-associe |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| WO1996017947A1 (fr) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| CA2625279A1 (fr) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Purification d'adenovirus et de virus adeno-associe (aav) par voie chromatographique |
| JPH11514853A (ja) | 1995-09-08 | 1999-12-21 | ジエンザイム コーポレイション | 遺伝子治療のための改良されたａａｖベクター |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| PT1944362E (pt) | 1997-09-05 | 2016-01-27 | Genzyme Corp | Métodos de produção de preparações de alto título de vetores aav recombinantes desprovidos de adjuvantes |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| 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 |
| 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 |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| DK1910395T3 (da) | 2005-06-23 | 2013-02-04 | Isis Pharmaceuticals Inc | Sammensætninger og fremgangsmåder til modulation af SMN2-splejsning |
| EP3305302B1 (fr) | 2009-06-17 | 2018-09-19 | Biogen MA Inc. | Compositions et méthodes pour moduler l'épissage de smn2 chez un sujet |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| WO2015141521A1 (fr) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | Appareil de traitement de substrat, procédé de fabrication de dispositif semi-conducteur et support d'enregistrement |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
-
2019
- 2019-11-27 BR BR112021009739-2A patent/BR112021009739A2/pt unknown
- 2019-11-27 AU AU2019389047A patent/AU2019389047A1/en active Pending
- 2019-11-27 TW TW108143173A patent/TW202039859A/zh unknown
- 2019-11-27 CA CA3116630A patent/CA3116630A1/fr active Pending
- 2019-11-27 KR KR1020217019147A patent/KR20210099025A/ko unknown
- 2019-11-27 JP JP2021530078A patent/JP2022511776A/ja active Pending
- 2019-11-27 WO PCT/US2019/063649 patent/WO2020113034A1/fr active Application Filing
- 2019-11-27 CN CN201980078349.8A patent/CN113226380A/zh active Pending
- 2019-11-27 MX MX2021006359A patent/MX2021006359A/es unknown
- 2019-11-27 EP EP19836872.2A patent/EP3886919A1/fr active Pending
- 2019-11-27 US US17/309,403 patent/US20220001028A1/en active Pending
-
2021
- 2021-05-03 IL IL282885A patent/IL282885A/en unknown
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