JPWO2021127655A5 - - Google Patents
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- Publication number
- JPWO2021127655A5 JPWO2021127655A5 JP2022538188A JP2022538188A JPWO2021127655A5 JP WO2021127655 A5 JPWO2021127655 A5 JP WO2021127655A5 JP 2022538188 A JP2022538188 A JP 2022538188A JP 2022538188 A JP2022538188 A JP 2022538188A JP WO2021127655 A5 JPWO2021127655 A5 JP WO2021127655A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- cdna
- ires
- variant
- gne
- 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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2023507794A JP2023507794A (ja) | 2023-02-27 |
| JPWO2021127655A5 true JPWO2021127655A5 (https=) | 2024-01-04 |
| JP2023507794A5 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 |
|---|---|---|---|---|
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| IL301675A (en) | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
| CA3236188A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of mps i |
| TW202502803A (zh) | 2023-03-10 | 2025-01-16 | 美商戴諾治療公司 | 衣殼多肽及其使用方法 |
| 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 |
| WO2026064442A2 (en) | 2024-09-18 | 2026-03-26 | Dyno Therapeutics, Inc. | Capsid polypeptides and methods of use thereof |
| WO2026064428A2 (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 |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| 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 |
| 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 |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | 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 |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved 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 |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| US6953662B2 (en) | 1997-08-29 | 2005-10-11 | Human Genome Sciences, Inc. | Follistatin-3 |
| US6372454B2 (en) | 1997-08-29 | 2002-04-16 | Human Genome Sciences, Inc. | Nucleic acid molecules encoding Follistatin-3 |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| 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 |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| CA2412847A1 (en) * | 2000-06-30 | 2002-01-10 | Collateral Therapeutics, Inc. | Dual recombinant gene therapy compositions and methods of use |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| EP1948247A4 (en) * | 2005-11-15 | 2010-07-07 | Univ Leland Stanford Junior | COMPOSITIONS FOR COORDINATED VEGF AND PDGF EXPRESSION AND METHOD OF USE THEREOF |
| JP2015503924A (ja) * | 2012-01-05 | 2015-02-05 | ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド | 遺伝子導入のための方法及び組成物 |
| 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 |
| CN120174012A (zh) * | 2013-10-24 | 2025-06-20 | 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 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 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プログラム |
| US10301367B2 (en) * | 2014-07-26 | 2019-05-28 | 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 |
| CA3010599C (en) * | 2016-01-06 | 2025-05-20 | Stealth Biotherapeutics Inc. | METHODS AND COMPOSITIONS FOR THE PREVENTION AND TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY |
-
2020
- 2020-12-21 AU AU2020408225A patent/AU2020408225A1/en active Pending
- 2020-12-21 EP EP20839521.0A patent/EP4077687A1/en active Pending
- 2020-12-21 JP JP2022538188A patent/JP2023507794A/ja 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 CN CN202080097838.0A patent/CN115516100A/zh active Pending
- 2020-12-21 IL IL294072A patent/IL294072A/en unknown
- 2020-12-21 WO PCT/US2020/066477 patent/WO2021127655A1/en not_active Ceased
-
2025
- 2025-10-14 JP JP2025172828A patent/JP2026009430A/ja active Pending
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