JP2022530633A5 - - Google Patents
Info
- Publication number
- JP2022530633A5 JP2022530633A5 JP2021564269A JP2021564269A JP2022530633A5 JP 2022530633 A5 JP2022530633 A5 JP 2022530633A5 JP 2021564269 A JP2021564269 A JP 2021564269A JP 2021564269 A JP2021564269 A JP 2021564269A JP 2022530633 A5 JP2022530633 A5 JP 2022530633A5
- Authority
- JP
- Japan
- Prior art keywords
- aav
- acid sequence
- seq
- sequence
- nucleic acid
- 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 |
| JP2022530633A5 true JP2022530633A5 (enExample) | 2023-04-18 |
| JP7690401B2 JP7690401B2 (ja) | 2025-06-10 |
Family
ID=73028724
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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 |
| 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 |
Family Applications After (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 (4) | US20220249705A1 (enExample) |
| EP (3) | EP3976077A4 (enExample) |
| JP (3) | JP7690401B2 (enExample) |
| KR (3) | KR20220004695A (enExample) |
| CN (3) | CN113853207A (enExample) |
| AU (3) | AU2020264975A1 (enExample) |
| BR (3) | BR112021020054A2 (enExample) |
| CA (3) | CA3134379A1 (enExample) |
| CL (3) | CL2021002768A1 (enExample) |
| CO (3) | CO2021015924A2 (enExample) |
| IL (3) | IL287498A (enExample) |
| MX (3) | MX2021013268A (enExample) |
| PE (3) | PE20212334A1 (enExample) |
| PH (3) | PH12021552608A1 (enExample) |
| SG (3) | SG11202111375RA (enExample) |
| WO (3) | WO2020223232A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112022011916A2 (pt) | 2019-12-19 | 2022-09-06 | Univ Pennsylvania | Composições para tratar a ataxia de friedreich |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| WO2022036220A1 (en) * | 2020-08-14 | 2022-02-17 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| US20230372539A1 (en) | 2020-08-24 | 2023-11-23 | 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 |
| MX2023005446A (es) | 2020-11-11 | 2023-07-18 | European Molecular Biology Laboratory | Particulas virales modificadas para terapia genica. |
| WO2022119890A1 (en) * | 2020-12-01 | 2022-06-09 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| AR124216A1 (es) | 2020-12-01 | 2023-03-01 | Univ Pennsylvania | Composiciones nuevas con motivos selectivos específicos del tejido y composiciones que las contienen |
| AU2022262771A1 (en) | 2021-04-23 | 2023-11-02 | The Trustees Of The University Of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| CN119317718A (zh) | 2022-01-25 | 2025-01-14 | 宾夕法尼亚州大学信托人 | 用于改善心脏转导和肝脏去靶向的aav衣壳 |
| AU2023228669A1 (en) * | 2022-03-01 | 2024-09-19 | Scout Bio, Inc. | Viral vector genome encoding an insulin fusion protein |
| EP4486758A2 (en) * | 2022-03-03 | 2025-01-08 | The Trustees of the University of Pennsylvania | Viral vectors encoding parathyroid hormone fusions and uses thereof in treating hypoparathyroidism |
| US20250250326A1 (en) | 2022-04-06 | 2025-08-07 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti-aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| CA3247109A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers |
| WO2024015966A2 (en) * | 2022-07-15 | 2024-01-18 | The Trustees Of The University Of Pennsylvania | Recombinant aav having aav clade d and clade e capsids and compositions containing same |
| EP4634394A2 (en) | 2022-12-17 | 2025-10-22 | The Trustees of The University of Pennsylvania | Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same |
| 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 |
| JP2026522354A (ja) | 2023-06-12 | 2026-07-07 | 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 | ムコ多糖症ｉｉｉｂのためのａａｖ遺伝子療法 |
| WO2025007046A1 (en) | 2023-06-29 | 2025-01-02 | The Trustees Of The University Of Pennsylvania | Mutant aav with central nervous system targeting motifs and compositions containing same |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ 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 |
| KR20260114566A (ko) | 2023-11-14 | 2026-07-22 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도 |
| 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 |
| WO2026122921A1 (en) | 2024-12-05 | 2026-06-11 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2026151888A1 (en) | 2025-01-08 | 2026-07-16 | The Trustees Of The University Of Pennsylvania | Compositions for treating friedreich's ataxia |
Family Cites Families (47)
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|---|---|---|---|---|
| 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 |
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 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 |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| 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 |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| EP1668143B1 (en) | 2003-09-30 | 2013-03-20 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| DE23193606T1 (de) * | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US8283151B2 (en) * | 2005-04-29 | 2012-10-09 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes |
| JP2009535339A (ja) | 2006-04-28 | 2009-10-01 | 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 | 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用 |
| 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 |
| US8734809B2 (en) * | 2009-05-28 | 2014-05-27 | University Of Massachusetts | 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 |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| DK2561073T3 (en) | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| 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 |
| NZ716102A (en) * | 2013-07-22 | 2021-12-24 | The Children’S Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| HUE052676T2 (hu) * | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| WO2015137802A1 (en) | 2014-03-10 | 2015-09-17 | Uniqure Ip B.V. | Further improved aav vectors produced in insect cells |
| US10577627B2 (en) * | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016065001A1 (en) * | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| AU2015349759B2 (en) * | 2014-11-21 | 2022-01-06 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| JP6754361B2 (ja) * | 2014-12-16 | 2020-09-09 | ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ | 若年型バッテン病のための遺伝子療法 |
| EP3632923A1 (en) | 2015-01-16 | 2020-04-08 | Voyager Therapeutics, Inc. | Central nervous system targeting polynucleotides |
| JP6851319B2 (ja) | 2015-04-27 | 2021-03-31 | 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 | ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系 |
| EP4134375A1 (en) | 2015-07-30 | 2023-02-15 | Massachusetts Eye & Ear Infirmary | Ancestral adeno associated virus sequences and uses thereof |
| US20180230489A1 (en) * | 2015-10-28 | 2018-08-16 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
| AU2016343979A1 (en) * | 2015-10-29 | 2018-05-10 | Voyager Therapeutics, Inc. | Delivery of central nervous system targeting polynucleotides |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| AU2017345470B2 (en) * | 2016-10-19 | 2023-08-03 | Adverum Biotechnologies, Inc. | Modified AAV capsids and uses thereof |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
| 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 |
| WO2018170310A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| 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 |
| US11512327B2 (en) | 2017-08-03 | 2022-11-29 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of AAV |
| KR20200067195A (ko) | 2017-10-18 | 2020-06-11 | 리젠엑스바이오 인크. | 완전-인간 번역후 변형된 항체 치료제 |
| EP3768695A4 (en) | 2018-02-27 | 2022-04-06 | The Trustees of the University of Pennsylvania | NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| 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 |
| MX2021007600A (es) | 2018-12-21 | 2021-08-11 | Univ Pennsylvania | Composiciones para la reduccion de la expresion transgenica especifica de drg. |
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2020
- 2020-04-28 WO PCT/US2020/030273 patent/WO2020223232A1/en not_active Ceased
- 2020-04-28 PH PH1/2021/552608A patent/PH12021552608A1/en unknown
- 2020-04-28 JP JP2021564269A patent/JP7690401B2/ja active Active
- 2020-04-28 JP JP2021564543A patent/JP2022530656A/ja active Pending
- 2020-04-28 MX MX2021013268A patent/MX2021013268A/es unknown
- 2020-04-28 AU AU2020264975A patent/AU2020264975A1/en active Pending
- 2020-04-28 PE PE2021001789A patent/PE20212334A1/es unknown
- 2020-04-28 SG SG11202111375RA patent/SG11202111375RA/en unknown
- 2020-04-28 CA CA3134379A patent/CA3134379A1/en active Pending
- 2020-04-28 CN CN202080032666.9A patent/CN113853207A/zh active Pending
- 2020-04-28 KR KR1020217038207A patent/KR20220004695A/ko active Pending
- 2020-04-28 WO PCT/US2020/030266 patent/WO2020223231A1/en not_active Ceased
- 2020-04-28 KR KR1020217037659A patent/KR20220004680A/ko not_active Withdrawn
- 2020-04-28 WO PCT/US2020/030281 patent/WO2020223236A1/en not_active Ceased
- 2020-04-28 EP EP20798442.8A patent/EP3976077A4/en active Pending
- 2020-04-28 SG SG11202111102SA patent/SG11202111102SA/en unknown
- 2020-04-28 AU AU2020264976A patent/AU2020264976A1/en active Pending
- 2020-04-28 BR BR112021020054A patent/BR112021020054A2/pt unknown
- 2020-04-28 MX MX2021013266A patent/MX2021013266A/es unknown
- 2020-04-28 EP EP20798753.8A patent/EP3962507A4/en not_active Withdrawn
- 2020-04-28 AU AU2020266523A patent/AU2020266523A1/en active Pending
- 2020-04-28 PE PE2021001788A patent/PE20220014A1/es unknown
- 2020-04-28 SG SG11202111373YA patent/SG11202111373YA/en unknown
- 2020-04-28 PE PE2021001795A patent/PE20212267A1/es unknown
- 2020-04-28 BR BR112021021502A patent/BR112021021502A2/pt not_active IP Right Cessation
- 2020-04-28 US US17/605,881 patent/US20220249705A1/en not_active Abandoned
- 2020-04-28 US US17/605,874 patent/US12516352B2/en active Active
- 2020-04-28 CA CA3134468A patent/CA3134468A1/en active Pending
- 2020-04-28 CA CA3134507A patent/CA3134507A1/en active Pending
- 2020-04-28 BR BR112021020545A patent/BR112021020545A2/pt unknown
- 2020-04-28 KR KR1020217037661A patent/KR20220004681A/ko active Pending
- 2020-04-28 EP EP20798752.0A patent/EP3976078A4/en active Pending
- 2020-04-28 US US17/605,868 patent/US12516351B2/en active Active
- 2020-04-28 PH PH1/2021/552607A patent/PH12021552607A1/en unknown
- 2020-04-28 CN CN202080032773.1A patent/CN113853208A/zh active Pending
- 2020-04-28 CN CN202080033196.8A patent/CN113853209B/zh active Active
- 2020-04-28 JP JP2021564542A patent/JP7644026B2/ja active Active
- 2020-04-28 MX MX2021013267A patent/MX2021013267A/es unknown
- 2020-04-28 PH PH1/2021/552540A patent/PH12021552540A1/en unknown
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2021
- 2021-10-21 IL IL287498A patent/IL287498A/en unknown
- 2021-10-21 IL IL287491A patent/IL287491A/en unknown
- 2021-10-21 CL CL2021002768A patent/CL2021002768A1/es unknown
- 2021-10-21 IL IL287492A patent/IL287492A/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 CONC2021/0015916A patent/CO2021015916A2/es unknown
- 2021-11-25 CO CONC2021/0015928A patent/CO2021015928A2/es unknown
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2026
- 2026-01-05 US US19/440,028 patent/US20260201416A1/en active Pending