JPWO2020223232A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020223232A5 JPWO2020223232A5 JP2021564269A JP2021564269A JPWO2020223232A5 JP WO2020223232 A5 JPWO2020223232 A5 JP WO2020223232A5 JP 2021564269 A JP2021564269 A JP 2021564269A JP 2021564269 A JP2021564269 A JP 2021564269A JP WO2020223232 A5 JPWO2020223232 A5 JP WO2020223232A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- acid sequence
- aav
- nucleic acid
- sequence
- 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
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962840184P | 2019-04-29 | 2019-04-29 | |
| US62/840,184 | 2019-04-29 | ||
| US201962913314P | 2019-10-10 | 2019-10-10 | |
| US62/913,314 | 2019-10-10 | ||
| US201962924095P | 2019-10-21 | 2019-10-21 | |
| US62/924,095 | 2019-10-21 | ||
| PCT/US2020/030273 WO2020223232A1 (en) | 2019-04-29 | 2020-04-28 | Novel aav capsids and compositions containing same |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022530633A JP2022530633A (ja) | 2022-06-30 |
| JPWO2020223232A5 true JPWO2020223232A5 (enExample) | 2023-04-18 |
| JP7690401B2 JP7690401B2 (ja) | 2025-06-10 |
Family
ID=73028724
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564543A Pending JP2022530656A (ja) | 2019-04-29 | 2020-04-28 | 新規aavカプシドおよびそれを含む組成物 |
| JP2021564542A Active JP7644026B2 (ja) | 2019-04-29 | 2020-04-28 | 新規aavカプシドおよびそれを含む組成物 |
| JP2021564269A Active JP7690401B2 (ja) | 2019-04-29 | 2020-04-28 | 新規aavカプシドおよびそれを含む組成物 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564543A Pending JP2022530656A (ja) | 2019-04-29 | 2020-04-28 | 新規aavカプシドおよびそれを含む組成物 |
| JP2021564542A Active JP7644026B2 (ja) | 2019-04-29 | 2020-04-28 | 新規aavカプシドおよびそれを含む組成物 |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US12516351B2 (enExample) |
| EP (3) | EP3962507A4 (enExample) |
| JP (3) | JP2022530656A (enExample) |
| KR (3) | KR20220004680A (enExample) |
| CN (3) | CN113853208A (enExample) |
| AU (3) | AU2020264976A1 (enExample) |
| BR (3) | BR112021020054A2 (enExample) |
| CA (3) | CA3134379A1 (enExample) |
| CL (3) | CL2021002768A1 (enExample) |
| CO (3) | CO2021015924A2 (enExample) |
| IL (3) | IL287492A (enExample) |
| MX (3) | MX2021013268A (enExample) |
| PE (3) | PE20220014A1 (enExample) |
| PH (3) | PH12021552607A1 (enExample) |
| SG (3) | SG11202111373YA (enExample) |
| WO (3) | WO2020223232A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL299167A (en) | 2020-06-17 | 2023-02-01 | Univ Pennsylvania | Compositions and methods for treating patients with gene therapy |
| EP4200429A1 (en) | 2020-08-24 | 2023-06-28 | The Trustees of The University of Pennsylvania | Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases |
| WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| AU2021378075B2 (en) | 2020-11-11 | 2025-03-27 | Borea Therapeutics S.R.L. | Modified viral particles for gene therapy |
| KR20230128001A (ko) * | 2020-12-01 | 2023-09-01 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도 |
| EP4256065A2 (en) | 2020-12-01 | 2023-10-11 | The Trustees of The University of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| KR20240137067A (ko) | 2022-01-25 | 2024-09-19 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 개선된 심장 형질도입 및 간의 탈표적화를 위한 aav 캡시드 |
| AU2023228669A1 (en) * | 2022-03-01 | 2024-09-19 | Scout Bio, Inc. | Viral vector genome encoding an insulin fusion protein |
| CN119137138A (zh) * | 2022-03-03 | 2024-12-13 | 宾夕法尼亚州大学信托人 | 编码甲状旁腺激素融合物的病毒载体及其在治疗甲状旁腺功能减退中的用途 |
| CN119317645A (zh) | 2022-04-06 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于治疗her2阳性转移性乳腺癌及其他癌症的组合物和方法 |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| US20260021205A1 (en) * | 2022-07-15 | 2026-01-22 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| KR20250135916A (ko) | 2022-12-17 | 2025-09-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물 |
| WO2024130070A2 (en) | 2022-12-17 | 2024-06-20 | The Trustees Of The University Of Pennsylvania | Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| WO2025035143A1 (en) | 2023-08-10 | 2025-02-13 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of spinal muscular atrophy |
| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
| GB202319008D0 (en) * | 2023-12-13 | 2024-01-24 | Ucl Business Ltd | AAV-mediated gene therapy of diseases caused by impaired function of GLDC |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
Family Cites Families (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
| US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
| 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 |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
| EP1660970A4 (en) | 2003-08-01 | 2007-02-14 | Dna Twopointo Inc | SYSTEMS AND METHODS FOR BIOPOLYMER TECHNOLOGY |
| 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 |
| WO2006119432A2 (en) * | 2005-04-29 | 2006-11-09 | The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services | Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes |
| US20090317417A1 (en) * | 2006-04-28 | 2009-12-24 | The Trustees Of The University Of Pennsylvania | Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof |
| EP2012122A1 (en) * | 2007-07-06 | 2009-01-07 | Medigene AG | Mutated parvovirus structural proteins as vaccines |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| WO2010138263A2 (en) * | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| EP2287323A1 (en) | 2009-07-31 | 2011-02-23 | Association Institut de Myologie | Widespread gene delivery to the retina using systemic administration of AAV vectors |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| WO2013155222A2 (en) | 2012-04-10 | 2013-10-17 | The Regents Of The University Of California | Brain-specific enhancers for cell-based therapy |
| 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 |
| CA3209883A1 (en) * | 2013-07-22 | 2015-01-29 | The Children's Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| ES2857751T3 (es) * | 2013-10-11 | 2021-09-29 | Massachusetts Eye & Ear Infirmary | Métodos para predecir secuencias de virus ancestrales y usos de los mismos |
| KR102572449B1 (ko) * | 2014-03-10 | 2023-08-31 | 유니큐어 아이피 비.브이. | 곤충 세포에서 생산된 더욱 향상된 aav 벡터 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016065001A1 (en) * | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| BR112017010447A2 (pt) * | 2014-11-21 | 2018-05-15 | The University Of North Carolina At Chapel Hill | vetores de aav direcionados ao sistema nervoso central |
| CN107249646B (zh) | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| WO2016115503A1 (en) | 2015-01-16 | 2016-07-21 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| EP3288594B1 (en) | 2015-04-27 | 2022-06-29 | The Trustees of The University of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
| CA2994160C (en) | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| MX2018004755A (es) * | 2015-10-29 | 2018-12-19 | Voyager Therapeutics Inc | Entrega de polinucleotidos dirigidos al sistema nervioso central. |
| EP3448874A4 (en) * | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| AU2017345470B2 (en) * | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| 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 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| ES2966692T3 (es) | 2017-03-15 | 2024-04-23 | Univ North Carolina Chapel Hill | Vectores de virus adenoasociados poliploides y métodos de fabricación y uso de los mismos |
| WO2018200419A1 (en) * | 2017-04-23 | 2018-11-01 | The Trustees Of The University Of Pennsylvania | Viral vectors comprising engineered aav capsids and compositions containing the same |
| JOP20190269A1 (ar) * | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019028306A2 (en) * | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
| CN111727201A (zh) | 2017-10-18 | 2020-09-29 | 再生生物股份有限公司 | 完全人源翻译后修饰的抗体治疗剂 |
| MX2020008932A (es) | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| IL276859B2 (en) | 2018-02-27 | 2025-12-01 | Univ Pennsylvania | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
| PE20211581A1 (es) | 2018-12-21 | 2021-08-17 | Univ Pennsylvania | Composiciones para la reduccion de la expresion transgenica especifica de drg |
-
2020
- 2020-04-28 US US17/605,868 patent/US12516351B2/en active Active
- 2020-04-28 SG SG11202111373YA patent/SG11202111373YA/en unknown
- 2020-04-28 CA CA3134379A patent/CA3134379A1/en active Pending
- 2020-04-28 BR BR112021020054A patent/BR112021020054A2/pt unknown
- 2020-04-28 EP EP20798753.8A patent/EP3962507A4/en not_active Withdrawn
- 2020-04-28 PH PH1/2021/552607A patent/PH12021552607A1/en unknown
- 2020-04-28 BR BR112021020545A patent/BR112021020545A2/pt unknown
- 2020-04-28 WO PCT/US2020/030273 patent/WO2020223232A1/en not_active Ceased
- 2020-04-28 CA CA3134468A patent/CA3134468A1/en active Pending
- 2020-04-28 JP JP2021564543A patent/JP2022530656A/ja active Pending
- 2020-04-28 SG SG11202111375RA patent/SG11202111375RA/en unknown
- 2020-04-28 PH PH1/2021/552608A patent/PH12021552608A1/en unknown
- 2020-04-28 EP EP20798752.0A patent/EP3976078A4/en active Pending
- 2020-04-28 EP EP20798442.8A patent/EP3976077A4/en active Pending
- 2020-04-28 SG SG11202111102SA patent/SG11202111102SA/en unknown
- 2020-04-28 JP JP2021564542A patent/JP7644026B2/ja active Active
- 2020-04-28 BR BR112021021502A patent/BR112021021502A2/pt unknown
- 2020-04-28 CA CA3134507A patent/CA3134507A1/en active Pending
- 2020-04-28 KR KR1020217037659A patent/KR20220004680A/ko not_active Withdrawn
- 2020-04-28 MX MX2021013268A patent/MX2021013268A/es unknown
- 2020-04-28 WO PCT/US2020/030281 patent/WO2020223236A1/en not_active Ceased
- 2020-04-28 KR KR1020217038207A patent/KR20220004695A/ko active Pending
- 2020-04-28 PE PE2021001788A patent/PE20220014A1/es unknown
- 2020-04-28 CN CN202080032773.1A patent/CN113853208A/zh active Pending
- 2020-04-28 AU AU2020264976A patent/AU2020264976A1/en active Pending
- 2020-04-28 US US17/605,874 patent/US12516352B2/en active Active
- 2020-04-28 MX MX2021013266A patent/MX2021013266A/es unknown
- 2020-04-28 MX MX2021013267A patent/MX2021013267A/es unknown
- 2020-04-28 JP JP2021564269A patent/JP7690401B2/ja active Active
- 2020-04-28 PE PE2021001789A patent/PE20212334A1/es unknown
- 2020-04-28 PE PE2021001795A patent/PE20212267A1/es unknown
- 2020-04-28 AU AU2020264975A patent/AU2020264975A1/en active Pending
- 2020-04-28 PH PH1/2021/552540A patent/PH12021552540A1/en unknown
- 2020-04-28 US US17/605,881 patent/US20220249705A1/en not_active Abandoned
- 2020-04-28 WO PCT/US2020/030266 patent/WO2020223231A1/en not_active Ceased
- 2020-04-28 AU AU2020266523A patent/AU2020266523A1/en active Pending
- 2020-04-28 KR KR1020217037661A patent/KR20220004681A/ko active Pending
- 2020-04-28 CN CN202080032666.9A patent/CN113853207A/zh active Pending
- 2020-04-28 CN CN202080033196.8A patent/CN113853209B/zh active Active
-
2021
- 2021-10-21 IL IL287492A patent/IL287492A/en unknown
- 2021-10-21 CL CL2021002768A patent/CL2021002768A1/es unknown
- 2021-10-21 IL IL287491A patent/IL287491A/en unknown
- 2021-10-21 IL IL287498A patent/IL287498A/en unknown
- 2021-10-26 CL CL2021002811A patent/CL2021002811A1/es unknown
- 2021-10-27 CL CL2021002824A patent/CL2021002824A1/es unknown
- 2021-11-25 CO CONC2021/0015924A patent/CO2021015924A2/es unknown
- 2021-11-25 CO CONC2021/0015928A patent/CO2021015928A2/es unknown
- 2021-11-25 CO CONC2021/0015916A patent/CO2021015916A2/es unknown