JP2023507794A5 - - Google Patents
Info
- Publication number
- JP2023507794A5 JP2023507794A5 JP2022538188A JP2022538188A JP2023507794A5 JP 2023507794 A5 JP2023507794 A5 JP 2023507794A5 JP 2022538188 A JP2022538188 A JP 2022538188A JP 2022538188 A JP2022538188 A JP 2022538188A JP 2023507794 A5 JP2023507794 A5 JP 2023507794A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- cdna
- ires
- variant
- wild
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025172828A JP2026009430A (ja) | 2019-12-20 | 2025-10-14 | 筋肉疾患の筋肉を標的とするための最適化された遺伝子療法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962951564P | 2019-12-20 | 2019-12-20 | |
| US62/951,564 | 2019-12-20 | ||
| PCT/US2020/066477 WO2021127655A1 (en) | 2019-12-20 | 2020-12-21 | Optimized gene therapy for targeting muscle in muscle diseases |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025172828A Division JP2026009430A (ja) | 2019-12-20 | 2025-10-14 | 筋肉疾患の筋肉を標的とするための最適化された遺伝子療法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023507794A JP2023507794A (ja) | 2023-02-27 |
| JP2023507794A5 true JP2023507794A5 (https=) | 2024-01-04 |
Family
ID=74181377
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022538188A Pending JP2023507794A (ja) | 2019-12-20 | 2020-12-21 | 筋肉疾患の筋肉を標的とするための最適化された遺伝子療法 |
| JP2025172828A Pending JP2026009430A (ja) | 2019-12-20 | 2025-10-14 | 筋肉疾患の筋肉を標的とするための最適化された遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025172828A Pending JP2026009430A (ja) | 2019-12-20 | 2025-10-14 | 筋肉疾患の筋肉を標的とするための最適化された遺伝子療法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230227515A1 (https=) |
| EP (1) | EP4077687A1 (https=) |
| JP (2) | JP2023507794A (https=) |
| CN (1) | CN115516100A (https=) |
| AU (1) | AU2020408225A1 (https=) |
| CA (1) | CA3165469A1 (https=) |
| IL (1) | IL294072A (https=) |
| WO (1) | WO2021127655A1 (https=) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3947700A4 (en) | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| BR112023006305A2 (pt) | 2020-10-09 | 2023-05-09 | Tenaya Therapeutics Inc | Métodos e composições de terapia gênica com placofilina-2 |
| EP4423264A4 (en) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | AAV-IDUA VECTOR FOR TREATING MPS I |
| WO2024191778A1 (en) | 2023-03-10 | 2024-09-19 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| EP4698651A1 (en) * | 2023-04-19 | 2026-02-25 | Gradalis, Inc. | Compositions and methods for modulating sialic acid production and treating hereditary inclusion body myopathy (hibm) |
| WO2025157262A1 (en) * | 2024-01-25 | 2025-07-31 | Sichuan Real & Best Biotech Co., Ltd. | Nucleic acids encoding follistatin and uses thereof |
| TW202545971A (zh) | 2024-02-08 | 2025-12-01 | 美商戴諾治療公司 | 殼體多肽及其使用方法 |
| WO2025188993A2 (en) | 2024-03-07 | 2025-09-12 | Research Institute At Nationwide Children's Hospital | Gene therapy for treating gne-related disorders |
| WO2026064428A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064444A1 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5041538A (en) | 1987-08-28 | 1991-08-20 | The Salk Institute For Biological Studies | Mammalian follistatin |
| 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 |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | 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 | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| US6953662B2 (en) | 1997-08-29 | 2005-10-11 | Human Genome Sciences, Inc. | Follistatin-3 |
| JP2001513982A (ja) | 1997-08-29 | 2001-09-11 | ヒューマン ジノーム サイエンシーズ， インコーポレイテッド | フォリスタチン−３ |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US5942420A (en) | 1997-11-17 | 1999-08-24 | Millennium Biotherapeutics, Inc. | Molecules of the follistatin-related protein family and uses therefor |
| 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 |
| WO2002002148A2 (en) * | 2000-06-30 | 2002-01-10 | Collateral Therapeutics, Inc. | Dual recombinant gene therapy compositions and methods of use |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| US20070123486A1 (en) * | 2005-11-15 | 2007-05-31 | Andrea Banfi | Composition for coordinated VEGF and PDGF expression, and methods of use |
| WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| 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 |
| CN105745326A (zh) * | 2013-10-24 | 2016-07-06 | 优尼科Ip有限公司 | 用于基因治疗神经疾病的aav-5假型载体 |
| 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 |
| EP3194600B1 (en) * | 2014-07-26 | 2019-08-28 | Consiglio Nazionale Delle Ricerche | Compositions and methods for treatment of muscular dystrophy |
| 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 |
| CN116196388A (zh) * | 2016-01-06 | 2023-06-02 | 康德生物医疗有限公司 | 用于预防和治疗杜氏肌肉萎缩症的方法和组合物 |
-
2020
- 2020-12-21 CN CN202080097838.0A patent/CN115516100A/zh active Pending
- 2020-12-21 AU AU2020408225A patent/AU2020408225A1/en active Pending
- 2020-12-21 US US17/786,721 patent/US20230227515A1/en active Pending
- 2020-12-21 CA CA3165469A patent/CA3165469A1/en active Pending
- 2020-12-21 IL IL294072A patent/IL294072A/en unknown
- 2020-12-21 WO PCT/US2020/066477 patent/WO2021127655A1/en not_active Ceased
- 2020-12-21 EP EP20839521.0A patent/EP4077687A1/en active Pending
- 2020-12-21 JP JP2022538188A patent/JP2023507794A/ja active Pending
-
2025
- 2025-10-14 JP JP2025172828A patent/JP2026009430A/ja active Pending
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