JP2020519292A5 - - Google Patents
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- Publication number
- JP2020519292A5 JP2020519292A5 JP2019562418A JP2019562418A JP2020519292A5 JP 2020519292 A5 JP2020519292 A5 JP 2020519292A5 JP 2019562418 A JP2019562418 A JP 2019562418A JP 2019562418 A JP2019562418 A JP 2019562418A JP 2020519292 A5 JP2020519292 A5 JP 2020519292A5
- Authority
- JP
- Japan
- Prior art keywords
- raav
- suspension
- aav
- itr
- vector genome
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023073941A JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762504817P | 2017-05-11 | 2017-05-11 | |
| US62/504,817 | 2017-05-11 | ||
| US201762599816P | 2017-12-18 | 2017-12-18 | |
| US62/599,816 | 2017-12-18 | ||
| US201862652006P | 2018-04-03 | 2018-04-03 | |
| US62/652,006 | 2018-04-03 | ||
| PCT/US2018/032278 WO2018209205A1 (en) | 2017-05-11 | 2018-05-11 | Gene therapy for neuronal ceroid lipofuscinoses |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023073941A Division JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020519292A JP2020519292A (ja) | 2020-07-02 |
| JP2020519292A5 true JP2020519292A5 (enExample) | 2021-05-27 |
| JP7273730B2 JP7273730B2 (ja) | 2023-05-15 |
Family
ID=64105028
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019562418A Active JP7273730B2 (ja) | 2017-05-11 | 2018-05-11 | 神経セロイドリポフスチン症のための遺伝子療法 |
| JP2023073941A Pending JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023073941A Pending JP2023099113A (ja) | 2017-05-11 | 2023-04-28 | 神経セロイドリポフスチン症のための遺伝子療法 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11591614B2 (enExample) |
| EP (1) | EP3621612A4 (enExample) |
| JP (2) | JP7273730B2 (enExample) |
| KR (1) | KR102719222B1 (enExample) |
| AU (1) | AU2018265531B2 (enExample) |
| BR (1) | BR112019023303A2 (enExample) |
| CA (1) | CA3061655A1 (enExample) |
| IL (2) | IL316926A (enExample) |
| RU (1) | RU2019139555A (enExample) |
| SG (2) | SG10201912401QA (enExample) |
| WO (1) | WO2018209205A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| CA3041548A1 (en) * | 2016-11-04 | 2018-05-11 | The Children's Hospital Of Philadelphia | Gene transfer compositions, methods and uses for treating neurodegenerative diseases |
| WO2019060662A1 (en) | 2017-09-22 | 2019-03-28 | The Trustees Of The University Of Pennsylvania | GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II |
| SG11202104295UA (en) * | 2018-11-14 | 2021-06-29 | Regenxbio Inc | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2020112802A1 (en) * | 2018-11-28 | 2020-06-04 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disease |
| US20200360491A1 (en) * | 2019-04-08 | 2020-11-19 | The Children's Hospital Of Philadelphia | Treatment of lysosomal storage disease in the eye through administration of aavs expressing tpp1 |
| JP2022526425A (ja) * | 2019-04-12 | 2022-05-24 | エンコーデッド セラピューティクス， インコーポレイテッド | 治療剤の投与のための組成物および方法 |
| EP4162059A1 (en) | 2020-05-12 | 2023-04-12 | The Trustees of The University of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| CN116437968A (zh) * | 2020-08-10 | 2023-07-14 | 普利维尔治疗公司 | 用于神经退行性病症的基因疗法 |
| KR20230083287A (ko) * | 2020-10-07 | 2023-06-09 | 리젠엑스바이오 인크. | Cln2 질환의 안구 징후에 대한 유전자 요법 |
| EP4284335A1 (en) * | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| WO2025090598A1 (en) * | 2023-10-23 | 2025-05-01 | The Children's Hospital Of Philadelphia | Aav vectors for treatment of cln2 disease |
Family Cites Families (46)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| EP2327797B1 (en) | 1997-04-01 | 2015-11-25 | Illumina Cambridge Limited | Method of nucleic acid sequencing |
| US6548286B1 (en) | 1997-04-14 | 2003-04-15 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant AAV product |
| EP1080218A1 (en) | 1998-05-27 | 2001-03-07 | University of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| ATE403715T1 (de) | 1999-08-09 | 2008-08-15 | Targeted Genetics Corp | Erhöhung der expression einer einzelsträngigen, heterologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| AU2001268149B2 (en) | 2000-06-01 | 2005-08-18 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| WO2002095073A1 (en) | 2001-05-18 | 2002-11-28 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences |
| CN103555677B (zh) | 2001-11-13 | 2018-01-30 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| WO2003052051A2 (en) | 2001-12-17 | 2003-06-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| DK3211085T3 (da) | 2003-09-30 | 2021-06-21 | Univ Pennsylvania | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
| US8999678B2 (en) | 2005-04-07 | 2015-04-07 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| WO2010053572A2 (en) | 2008-11-07 | 2010-05-14 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| WO2010071832A1 (en) | 2008-12-19 | 2010-06-24 | Nationwide Children's Hospital | Delivery of polynucleotides across the blood brain barrier using recombinant aav9 |
| US11219696B2 (en) * | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| CA2832151C (en) | 2011-03-31 | 2021-06-15 | University Of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| PT2717893T (pt) | 2011-06-08 | 2019-08-20 | Translate Bio Inc | Composições de nanopartículas lipídicas e métodos para transferência de arnm |
| EP2834259A4 (en) | 2012-04-02 | 2016-08-24 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| US9719106B2 (en) | 2013-04-29 | 2017-08-01 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof |
| BR112016001592A2 (pt) | 2013-07-26 | 2017-11-28 | Univ Iowa Res Found | métodos e composições para o tratamento de doenças cerebrais |
| WO2015164723A1 (en) | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| CN108601771B (zh) | 2015-10-23 | 2023-02-21 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
| US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
| WO2017136500A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| SI3589730T1 (sl) | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
-
2018
- 2018-05-11 IL IL316926A patent/IL316926A/en unknown
- 2018-05-11 CA CA3061655A patent/CA3061655A1/en active Pending
- 2018-05-11 JP JP2019562418A patent/JP7273730B2/ja active Active
- 2018-05-11 SG SG10201912401QA patent/SG10201912401QA/en unknown
- 2018-05-11 EP EP18798476.0A patent/EP3621612A4/en active Pending
- 2018-05-11 BR BR112019023303-2A patent/BR112019023303A2/pt active Search and Examination
- 2018-05-11 RU RU2019139555A patent/RU2019139555A/ru unknown
- 2018-05-11 SG SG11201910015S patent/SG11201910015SA/en unknown
- 2018-05-11 WO PCT/US2018/032278 patent/WO2018209205A1/en not_active Ceased
- 2018-05-11 AU AU2018265531A patent/AU2018265531B2/en active Active
- 2018-05-11 KR KR1020197036041A patent/KR102719222B1/ko active Active
- 2018-05-11 IL IL270422A patent/IL270422B2/en unknown
- 2018-05-11 US US16/611,512 patent/US11591614B2/en active Active
-
2022
- 2022-12-29 US US18/148,077 patent/US12428653B2/en active Active
-
2023
- 2023-04-28 JP JP2023073941A patent/JP2023099113A/ja active Pending
-
2025
- 2025-08-26 US US19/309,870 patent/US20260078406A1/en active Pending
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