JP2025060641A5 - - Google Patents
Info
- Publication number
- JP2025060641A5 JP2025060641A5 JP2024213225A JP2024213225A JP2025060641A5 JP 2025060641 A5 JP2025060641 A5 JP 2025060641A5 JP 2024213225 A JP2024213225 A JP 2024213225A JP 2024213225 A JP2024213225 A JP 2024213225A JP 2025060641 A5 JP2025060641 A5 JP 2025060641A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- approximately
- less
- administration
- patient
- 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.)
- Granted
Links
Applications Claiming Priority (6)
| 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 (en) | 2018-11-30 | 2019-11-27 | Aav viral vectors and uses thereof |
| JP2021530078A JP7677887B2 (ja) | 2018-11-30 | 2019-11-27 | Ａａｖウイルスベクター及びその使用 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021530078A Division JP7677887B2 (ja) | 2018-11-30 | 2019-11-27 | Ａａｖウイルスベクター及びその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2025060641A JP2025060641A (ja) | 2025-04-10 |
| JP2025060641A5 true JP2025060641A5 (enExample) | 2025-10-23 |
| JP7808674B2 JP7808674B2 (ja) | 2026-01-29 |
Family
ID=69167903
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021530078A Active JP7677887B2 (ja) | 2018-11-30 | 2019-11-27 | Ａａｖウイルスベクター及びその使用 |
| JP2024213225A Active JP7808674B2 (ja) | 2018-11-30 | 2024-12-06 | Ａａｖウイルスベクター及びその使用 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021530078A Active JP7677887B2 (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 (enExample) |
| EP (1) | EP3886919A1 (enExample) |
| JP (2) | JP7677887B2 (enExample) |
| KR (1) | KR20210099025A (enExample) |
| CN (2) | CN120860258A (enExample) |
| AU (1) | AU2019389047A1 (enExample) |
| BR (1) | BR112021009739A2 (enExample) |
| CA (1) | CA3116630A1 (enExample) |
| IL (1) | IL282885A (enExample) |
| MX (1) | MX2021006359A (enExample) |
| TW (2) | TWI844587B (enExample) |
| WO (1) | WO2020113034A1 (enExample) |
Families Citing this family (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20210308281A1 (en) * | 2018-08-15 | 2021-10-07 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| 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 |
| WO2022155364A1 (en) * | 2021-01-13 | 2022-07-21 | Dignity Health | Modulation of chitinase protein expression |
| CA3227103A1 (en) | 2021-07-30 | 2023-02-02 | Matthew P. GEMBERLING | Compositions and methods for modulating expression of frataxin (fxn) |
| US20240252684A1 (en) | 2021-07-30 | 2024-08-01 | Tune Therapeutics, Inc. | Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) |
| EP4380602A4 (en) * | 2021-08-03 | 2025-06-11 | The Regents Of The University Of California | ADENO-ASSOCIATED VIRAL (AAV)-MEDIATED EXPRESSION OF SPHINGOSINE 1-PHOSPHATE LYASE (SPL) FOR THE TREATMENT OF PULMONARY FIBROSIS |
| 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 (ko) * | 2022-05-10 | 2023-11-16 | 서울대학교산학협력단 | 인간 smn1 단백질 변이체 및 이의 용도 |
| US20250369012A1 (en) | 2022-06-21 | 2025-12-04 | Skyline Therapeutics Limited | Recombinant aav for the gene therapy of sma disease |
| CA3261865A1 (en) | 2022-07-12 | 2024-01-18 | Tune Therapeutics, Inc. | TARGETED TRANSCRIPTIONAL ACTIVATION COMPOSITIONS, SYSTEMS AND METHODS |
| WO2024163678A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods |
| WO2024163683A2 (en) | 2023-02-01 | 2024-08-08 | Tune Therapeutics, Inc. | Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist) |
| EP4713028A1 (en) * | 2023-05-18 | 2026-03-25 | Ultragenyx Pharmaceutical Inc. | Formulations comprising recombinant aav and methods of administering the same |
| WO2026028089A1 (en) | 2024-07-29 | 2026-02-05 | Novartis Ag | Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d) |
Family Cites Families (31)
| 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 |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| 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 |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| 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 |
| DK2573170T3 (en) | 2001-12-17 | 2018-04-09 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use |
| 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 |
| EP2345731B1 (en) * | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP3470072A1 (en) | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| WO2011094198A1 (en) * | 2010-01-28 | 2011-08-04 | The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center | A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| AU2013296425B2 (en) * | 2012-08-01 | 2018-06-07 | Nationwide Children's Hospital | Intrathecal delivery of recombinant adeno-associated virus 9 |
| MX380973B (es) * | 2013-05-01 | 2025-03-12 | Genzyme Corp | Composiciones y metodos para tratar la atrofia muscular espinal. |
| CN115120746A (zh) * | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| 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 |
| AU2014340149B2 (en) * | 2013-10-22 | 2020-12-24 | Shire Human Genetic Therapies, Inc. | CNS delivery of mRNA and uses thereof |
| CA2942515C (en) * | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| 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 |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| WO2019094253A1 (en) * | 2017-11-08 | 2019-05-16 | Avexis Inc. | Means and method for preparing viral vectors and uses of same |
-
2019
- 2019-11-27 CN CN202511011473.6A patent/CN120860258A/zh active Pending
- 2019-11-27 WO PCT/US2019/063649 patent/WO2020113034A1/en not_active Ceased
- 2019-11-27 CA CA3116630A patent/CA3116630A1/en active Pending
- 2019-11-27 JP JP2021530078A patent/JP7677887B2/ja active Active
- 2019-11-27 AU AU2019389047A patent/AU2019389047A1/en active Pending
- 2019-11-27 KR KR1020217019147A patent/KR20210099025A/ko not_active Ceased
- 2019-11-27 EP EP19836872.2A patent/EP3886919A1/en active Pending
- 2019-11-27 US US17/309,403 patent/US20220001028A1/en active Pending
- 2019-11-27 CN CN201980078349.8A patent/CN113226380A/zh active Pending
- 2019-11-27 TW TW108143173A patent/TWI844587B/zh active
- 2019-11-27 MX MX2021006359A patent/MX2021006359A/es unknown
- 2019-11-27 BR BR112021009739-2A patent/BR112021009739A2/pt unknown
- 2019-11-27 TW TW113118096A patent/TWI897418B/zh active
-
2021
- 2021-05-03 IL IL282885A patent/IL282885A/en unknown
-
2024
- 2024-12-06 JP JP2024213225A patent/JP7808674B2/ja active Active
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