JP2021520811A5 - - Google Patents
Info
- Publication number
- JP2021520811A5 JP2021520811A5 JP2020555467A JP2020555467A JP2021520811A5 JP 2021520811 A5 JP2021520811 A5 JP 2021520811A5 JP 2020555467 A JP2020555467 A JP 2020555467A JP 2020555467 A JP2020555467 A JP 2020555467A JP 2021520811 A5 JP2021520811 A5 JP 2021520811A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- isolated nucleic
- promoter
- acid construct
- itr
- 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.)
- Withdrawn
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862657243P | 2018-04-13 | 2018-04-13 | |
| US62/657,243 | 2018-04-13 | ||
| PCT/US2019/027271 WO2019200286A1 (en) | 2018-04-13 | 2019-04-12 | Bicistronic aav vectors encoding hexosaminidase alpha and beta-subunits and uses thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021520811A JP2021520811A (ja) | 2021-08-26 |
| JP2021520811A5 true JP2021520811A5 (https=) | 2022-04-18 |
Family
ID=68163786
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020555467A Withdrawn JP2021520811A (ja) | 2018-04-13 | 2019-04-12 | ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックａａｖベクターならびにその使用 |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US12486518B2 (https=) |
| EP (1) | EP3773748A4 (https=) |
| JP (1) | JP2021520811A (https=) |
| CN (1) | CN112203697A (https=) |
| BR (1) | BR112020020836A2 (https=) |
| CA (1) | CA3096102A1 (https=) |
| WO (1) | WO2019200286A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
| CA3164335A1 (en) * | 2020-01-10 | 2021-07-15 | Fatih Ozsolak | Viral vector for combination therapy |
| WO2023150051A1 (en) * | 2022-02-04 | 2023-08-10 | M6P Therapeutics, Inc. | Compositions and methods of using two-promoter vector for treatment of lysosomal storage disorders |
| KR20250158063A (ko) * | 2023-03-16 | 2025-11-05 | 젠자임 코포레이션 | 건식 연령-관련 황반 변성의 치료 |
| WO2026064462A1 (en) * | 2024-09-18 | 2026-03-26 | Genzyme Corporation | Production of recombinant aav |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5217865A (en) | 1986-07-25 | 1993-06-08 | The United States Of America As Represented By The Department Of Health And Human Services | Screening for Tay-Sachs disease with cloned DNA for beta-hexosaminidase |
| ATE404683T1 (de) | 1992-09-25 | 2008-08-15 | Aventis Pharma Sa | Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| WO2000012740A2 (en) | 1998-08-28 | 2000-03-09 | Duke University | ADENOVIRUSES DELETED IN THE IVa2, 100K AND/OR PRETERMINAL PROTEIN SEQUENCES |
| AU2013202568B2 (en) | 2001-11-13 | 2015-09-17 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus rh.8 (AAVrh.8) sequences and recombinant AAVs comprising same |
| CN1429905A (zh) * | 2002-01-04 | 2003-07-16 | 北京大学 | 治疗帕金森病的可调控双基因表达的重组腺病毒伴随病毒载体 |
| US7858367B2 (en) | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| CA2483915A1 (en) | 2002-05-02 | 2003-11-13 | University Of Rochester | Vectors having both isoforms of b-hexosaminidase |
| US20040192630A1 (en) * | 2002-05-02 | 2004-09-30 | Stephanos Kyrkanides | Vectors having both isoforms of beta-hexosaminidase and uses of the same |
| EP1620133B1 (en) | 2003-05-01 | 2015-12-09 | Genzyme Corporation | Gene therapy for neurometabolic disorders |
| ES2635726T3 (es) | 2007-06-06 | 2017-10-04 | Genzyme Corporation | Terapia génica para enfermedades de almacenamiento lisosómico |
| AU2009288657A1 (en) | 2008-09-04 | 2010-03-11 | Galenea Corp. | Synaptic vesicle cycling assays and systems |
| KR20120089743A (ko) | 2009-11-09 | 2012-08-13 | 제네포드 테라퓨틱스 에이비 | 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물 |
| CA2781332C (en) | 2009-11-19 | 2018-09-04 | National University Corporation Okayama University | System for increasing gene expression and vector comprising the system |
| US20130090374A1 (en) | 2011-04-20 | 2013-04-11 | Miguel Sena-Esteves | Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis |
| CA2840224C (en) | 2011-06-22 | 2019-08-13 | The General Hospital Corporation | Treatment of proteinopathies |
| CA2888628A1 (en) | 2012-10-19 | 2014-04-24 | Tokushima University | Novel high functional enzyme having modified substrate specificity of human beta-hexosaminidase b and exhibiting protease resistance |
| 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 |
| 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 |
| WO2015150922A2 (en) | 2014-03-17 | 2015-10-08 | The Hospital For Sick Children | β-HEXOSAMINIDASE PROTEIN VARIANTS AND ASSOCIATED METHODS FOR TREATING GM2 GANGLIOSIDOSES |
| CN107530447A (zh) | 2015-02-10 | 2018-01-02 | 建新公司 | 病毒颗粒至纹状体和皮质的增强递送 |
| EP3285788B1 (en) | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| US10016514B2 (en) | 2015-05-15 | 2018-07-10 | New Hope Research Foundation | Polynucleotides, vectors and methods for insertion and expression of transgenes |
| CA3011939A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| US11045557B2 (en) | 2016-06-09 | 2021-06-29 | Queen's University At Kingston | Methods and gene therapy constructs for treating GM2 gangliosidoses |
| WO2019222314A1 (en) | 2018-05-15 | 2019-11-21 | University Of Massachusetts | Raav vectors encoding of lysosomal beta-galactorsidase (glb1) and cathepsin a |
| US12448629B2 (en) | 2018-10-05 | 2025-10-21 | University Of Massachusetts | rAAV vectors for the treatment of GM1 and GM2 gangliosidosis |
-
2019
- 2019-04-12 CN CN201980034667.4A patent/CN112203697A/zh active Pending
- 2019-04-12 WO PCT/US2019/027271 patent/WO2019200286A1/en not_active Ceased
- 2019-04-12 JP JP2020555467A patent/JP2021520811A/ja not_active Withdrawn
- 2019-04-12 US US17/046,756 patent/US12486518B2/en active Active
- 2019-04-12 EP EP19784280.0A patent/EP3773748A4/en not_active Withdrawn
- 2019-04-12 BR BR112020020836-1A patent/BR112020020836A2/pt not_active IP Right Cessation
- 2019-04-12 CA CA3096102A patent/CA3096102A1/en active Pending
-
2025
- 2025-10-30 US US19/374,990 patent/US20260049336A1/en active Pending
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