JP2024543253A5 - - Google Patents
Info
- Publication number
- JP2024543253A5 JP2024543253A5 JP2024527624A JP2024527624A JP2024543253A5 JP 2024543253 A5 JP2024543253 A5 JP 2024543253A5 JP 2024527624 A JP2024527624 A JP 2024527624A JP 2024527624 A JP2024527624 A JP 2024527624A JP 2024543253 A5 JP2024543253 A5 JP 2024543253A5
- Authority
- JP
- Japan
- Prior art keywords
- composition according
- gene therapy
- polynucleotide
- seq
- transgene
- 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
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163278905P | 2021-11-12 | 2021-11-12 | |
| US63/278,905 | 2021-11-12 | ||
| PCT/US2022/079756 WO2023086966A1 (en) | 2021-11-12 | 2022-11-11 | Materials and methods for slc6a1 gene therapy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024543253A JP2024543253A (ja) | 2024-11-20 |
| JP2024543253A5 true JP2024543253A5 (enExample) | 2025-10-27 |
Family
ID=86336739
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024527624A Pending JP2024543253A (ja) | 2021-11-12 | 2022-11-11 | Ｓｌｃ６ａ１遺伝子治療のための材料及び方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20250002939A1 (enExample) |
| EP (1) | EP4429715A4 (enExample) |
| JP (1) | JP2024543253A (enExample) |
| AU (1) | AU2022386664A1 (enExample) |
| CA (1) | CA3238030A1 (enExample) |
| WO (1) | WO2023086966A1 (enExample) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP4727966A1 (en) * | 2023-06-13 | 2026-04-22 | Research Institute at Nationwide Children's Hospital | Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom |
| WO2025228976A1 (en) * | 2024-04-29 | 2025-11-06 | UCB Biopharma SRL | Gene therapy |
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 |
| EP0733103B1 (en) | 1993-11-09 | 2004-03-03 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| DE69433922T2 (de) | 1993-11-09 | 2005-07-28 | Medical College Of Ohio, Toledo | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| 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 (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 |
| JPH11511326A (ja) | 1995-08-30 | 1999-10-05 | ジエンザイム コーポレイション | アデノウィルスおよびａａｖの精製 |
| 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 |
| CA2995542A1 (en) | 1997-09-05 | 1999-03-11 | Genzyme Corporation | Methods for generating high titer helper-free preparations of recombinant aav vectors |
| 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 |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | 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 | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| 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 |
| 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 |
| WO2005111241A2 (en) * | 2004-05-07 | 2005-11-24 | Applera Corporation | Genetic polymorphisms associated with liver fibrosis methods of detection and uses thereof |
| US9434928B2 (en) | 2011-11-23 | 2016-09-06 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| 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 (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 |
| WO2021092518A1 (en) | 2019-11-08 | 2021-05-14 | The Board Of Regents Of The University Of Texas | Transgene cassettes, aav vectors and aav viral vectors for the expression of human codon-optimized slc6a1 |
-
2022
- 2022-11-11 AU AU2022386664A patent/AU2022386664A1/en active Pending
- 2022-11-11 WO PCT/US2022/079756 patent/WO2023086966A1/en not_active Ceased
- 2022-11-11 US US18/708,777 patent/US20250002939A1/en active Pending
- 2022-11-11 JP JP2024527624A patent/JP2024543253A/ja active Pending
- 2022-11-11 CA CA3238030A patent/CA3238030A1/en active Pending
- 2022-11-11 EP EP22893893.2A patent/EP4429715A4/en active Pending
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