JP2021515548A5 - - Google Patents
Info
- Publication number
- JP2021515548A5 JP2021515548A5 JP2020545159A JP2020545159A JP2021515548A5 JP 2021515548 A5 JP2021515548 A5 JP 2021515548A5 JP 2020545159 A JP2020545159 A JP 2020545159A JP 2020545159 A JP2020545159 A JP 2020545159A JP 2021515548 A5 JP2021515548 A5 JP 2021515548A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- capsid
- acid sequence
- proteins
- amino 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024088641A JP7860169B2 (ja) | 2018-02-27 | 2024-05-31 | 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 |
Applications Claiming Priority (29)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862635968P | 2018-02-27 | 2018-02-27 | |
| US201862635964P | 2018-02-27 | 2018-02-27 | |
| US62/635,964 | 2018-02-27 | ||
| US62/635,968 | 2018-02-27 | ||
| US201862663797P | 2018-04-27 | 2018-04-27 | |
| US201862663788P | 2018-04-27 | 2018-04-27 | |
| US62/663,788 | 2018-04-27 | ||
| US62/663,797 | 2018-04-27 | ||
| US201862667587P | 2018-05-06 | 2018-05-06 | |
| US62/667,587 | 2018-05-06 | ||
| US201862667881P | 2018-05-07 | 2018-05-07 | |
| US201862667888P | 2018-05-07 | 2018-05-07 | |
| US62/667,881 | 2018-05-07 | ||
| US62/667,888 | 2018-05-07 | ||
| US201862677474P | 2018-05-29 | 2018-05-29 | |
| US201862667585P | 2018-05-29 | 2018-05-29 | |
| US201862677471P | 2018-05-29 | 2018-05-29 | |
| US62/677,471 | 2018-05-29 | ||
| US62/667,585 | 2018-05-29 | ||
| US62/677,474 | 2018-05-29 | ||
| US201862703670P | 2018-07-26 | 2018-07-26 | |
| US201862703673P | 2018-07-26 | 2018-07-26 | |
| US62/703,670 | 2018-07-26 | ||
| US62/703,673 | 2018-07-26 | ||
| US201862722382P | 2018-08-24 | 2018-08-24 | |
| US201862722388P | 2018-08-24 | 2018-08-24 | |
| US62/722,388 | 2018-08-24 | ||
| US62/722,382 | 2018-08-24 | ||
| PCT/US2019/019861 WO2019169004A1 (en) | 2018-02-27 | 2019-02-27 | Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024088641A Division JP7860169B2 (ja) | 2018-02-27 | 2024-05-31 | 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021515548A JP2021515548A (ja) | 2021-06-24 |
| JP2021515548A5 true JP2021515548A5 (enExample) | 2022-02-18 |
| JP7498665B2 JP7498665B2 (ja) | 2024-06-12 |
Family
ID=67809202
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020545159A Active JP7498665B2 (ja) | 2018-02-27 | 2019-02-27 | 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 |
| JP2024088641A Active JP7860169B2 (ja) | 2018-02-27 | 2024-05-31 | 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024088641A Active JP7860169B2 (ja) | 2018-02-27 | 2024-05-31 | 新規アデノ随伴ウイルス（ａａｖ）ベクター、低減されたカプシド脱アミド化を有するａａｖベクター、およびその使用 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US12416016B2 (enExample) |
| EP (1) | EP3768695A4 (enExample) |
| JP (2) | JP7498665B2 (enExample) |
| KR (1) | KR102848518B1 (enExample) |
| CN (2) | CN120519403A (enExample) |
| AU (1) | AU2019228504B2 (enExample) |
| BR (1) | BR112020017278A2 (enExample) |
| CA (1) | CA3091795A1 (enExample) |
| CL (1) | CL2020002201A1 (enExample) |
| IL (1) | IL276860A (enExample) |
| MA (1) | MA52087A (enExample) |
| MX (1) | MX2020008932A (enExample) |
| SG (1) | SG11202007942YA (enExample) |
| WO (1) | WO2019169004A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| 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 |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| EP3884057A4 (en) * | 2018-12-21 | 2022-09-07 | The University of North Carolina at Chapel Hill | OPTIMIZED GALC GENES AND EXPRESSION CASSETTE AND THEIR USE |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| WO2020223232A1 (en) | 2019-04-29 | 2020-11-05 | The Trustees Of The University Of Pennsylvania | Novel aav capsids and compositions containing same |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| AU2020369508A1 (en) * | 2019-10-21 | 2022-05-26 | The Trustees Of The University Of Pennsylvania | AAV3B variants with improved production yield and liver tropism |
| EP4085255A1 (en) | 2020-01-03 | 2022-11-09 | Sarepta Therapeutics, Inc. | Methods for analyzing aav capsid proteins |
| WO2021183433A1 (en) * | 2020-03-09 | 2021-09-16 | University Of Massachusetts | Gene replacement therapy for foxg1 syndrome |
| AU2021259422A1 (en) | 2020-04-20 | 2022-12-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| US20230175014A1 (en) * | 2020-04-27 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for reducing nuclease expression and off-target activity using a promoter with low transcriptional activity |
| CA3183153A1 (en) | 2020-06-17 | 2021-12-23 | Christian HINDERER | Compositions and methods for treatment of gene therapy patients |
| WO2022015715A1 (en) | 2020-07-13 | 2022-01-20 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス， インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| WO2022082109A1 (en) * | 2020-10-18 | 2022-04-21 | The Trustees Of The University Of Pennsylvania | Improved adeno-associated virus (aav) vector and uses therefor |
| MX2023008826A (es) | 2021-02-01 | 2023-09-15 | Regenxbio Inc | Terapia génica para lipofuscinosis neuronal ceroidea. |
| IL307633A (en) | 2021-04-12 | 2023-12-01 | Univ Pennsylvania | Useful preparations in the treatment of spinal muscular atrophy (SBMA) |
| WO2023081807A1 (en) * | 2021-11-04 | 2023-05-11 | The Trustees of the University of Pennsylvania Penn Center for Innovation | Compositions and methods for reducing pcsk9 levels in a subject |
| TW202340467A (zh) | 2022-01-10 | 2023-10-16 | 賓州大學委員會 | 有用於治療c9orf72介導之病症之組成物及方法 |
| US20250177495A1 (en) | 2022-01-10 | 2025-06-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| 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 |
| CN121487959A (zh) * | 2023-03-25 | 2026-02-06 | 杜克大学 | 包含肾脏趋向性aav的组合物及其使用方法 |
| WO2025250683A1 (en) * | 2024-05-29 | 2025-12-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
Family Cites Families (38)
| 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 |
| 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 |
| EP1007637B1 (en) | 1997-04-14 | 2004-06-30 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| 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 |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US6596535B1 (en) | 1999-08-09 | 2003-07-22 | Targeted Genetics Corporation | Metabolically activated recombinant viral vectors and methods for the preparation and use |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| 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 |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| US20070172460A1 (en) * | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | Random peptide library displayed on aav vectors |
| WO2005012877A2 (en) | 2003-08-01 | 2005-02-10 | Dna Twopointo Inc. | Systems and methods for antibody engineering |
| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer 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 |
| US20060246079A1 (en) | 2003-11-14 | 2006-11-02 | Morrow Phillip R | Neutralizing human antibodies to anthrax toxin |
| DE23193606T1 (de) * | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| JP4495210B2 (ja) | 2005-06-09 | 2010-06-30 | パナソニック株式会社 | 振幅誤差補償装置及び直交度誤差補償装置 |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| WO2016049230A1 (en) * | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| US20180057839A1 (en) * | 2014-11-26 | 2018-03-01 | The Regents Of The University Of California | Therapeutic compositions comprising transcription factors and methods of making and using the same |
| US11135313B2 (en) | 2015-10-28 | 2021-10-05 | The Trustees Of The University Of Pennsylvania | Intrathecal administration of adeno-associated-viral vectors for gene therapy |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387138B2 (en) | 2015-12-11 | 2026-05-27 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| US11015174B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV8 |
| CN109661470A (zh) | 2016-04-15 | 2019-04-19 | 宾夕法尼亚州大学信托人 | 新型aav8突变衣壳和含有其的组合物 |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| JP6994018B2 (ja) * | 2016-07-26 | 2022-01-14 | バイオマリン ファーマシューティカル インコーポレイテッド | 新規アデノ随伴ウイルスキャプシドタンパク質 |
| HUE053747T2 (hu) * | 2016-08-15 | 2021-07-28 | Genzyme Corp | Eljárások AAV kimutatására |
| RS65241B1 (sr) | 2017-02-28 | 2024-03-29 | Univ Pennsylvania | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
| JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| 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 |
-
2019
- 2019-02-27 EP EP19760152.9A patent/EP3768695A4/en active Pending
- 2019-02-27 MA MA052087A patent/MA52087A/fr unknown
- 2019-02-27 MX MX2020008932A patent/MX2020008932A/es unknown
- 2019-02-27 CA CA3091795A patent/CA3091795A1/en active Pending
- 2019-02-27 WO PCT/US2019/019861 patent/WO2019169004A1/en not_active Ceased
- 2019-02-27 BR BR112020017278-2A patent/BR112020017278A2/pt unknown
- 2019-02-27 SG SG11202007942YA patent/SG11202007942YA/en unknown
- 2019-02-27 JP JP2020545159A patent/JP7498665B2/ja active Active
- 2019-02-27 CN CN202510015875.7A patent/CN120519403A/zh active Pending
- 2019-02-27 US US16/975,545 patent/US12416016B2/en active Active
- 2019-02-27 AU AU2019228504A patent/AU2019228504B2/en active Active
- 2019-02-27 CN CN201980029022.1A patent/CN112236443B/zh active Active
- 2019-02-27 KR KR1020207027462A patent/KR102848518B1/ko active Active
-
2020
- 2020-08-23 IL IL276860A patent/IL276860A/en unknown
- 2020-08-26 CL CL2020002201A patent/CL2020002201A1/es unknown
-
2024
- 2024-05-31 JP JP2024088641A patent/JP7860169B2/ja active Active
-
2025
- 2025-08-12 US US19/297,827 patent/US20260049334A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022530656A5 (enExample) | ||
| JP7561788B2 (ja) | 臨床使用に適した無血清懸濁細胞培養システムにおいて組換えアデノ随伴ウイルス（ａａｖ）ベクターを産生するスケーラブルな方法 | |
| JP2022530544A5 (enExample) | ||
| JP2022530633A5 (enExample) | ||
| JP2020510429A5 (enExample) | ||
| JP7801093B2 (ja) | 細胞トランスフェクション及び／又はｒＡＡＶベクター産生の改善のための増強剤 | |
| JP2021516046A5 (enExample) | ||
| FI3589730T3 (fi) | Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja | |
| US12398403B2 (en) | Methods for the manufacture of recombinant viral vectors | |
| JP2019509748A (ja) | 組換えタンパク質及び／又はウイルスベクター製造のための細胞株 | |
| WO2019173538A1 (en) | Aav chimeras | |
| CN110462037A (zh) | 腺相关病毒(aav)衣壳蛋白的突变体 | |
| CN104487579A (zh) | 使用aav衣壳变异体的高度有效的基因转移的组合物和方法 | |
| JP2023550581A5 (enExample) | ||
| AU2025271061A1 (en) | Manufacturing and use of recombinant aav vectors | |
| AR123838A1 (es) | Vector mejorado de virus adenoasociado (aav) y usos de este | |
| JP2024542950A5 (enExample) | ||
| JP2023529855A (ja) | Ｓｍｎ１タンパク質をコードするコドン最適化した核酸およびその使用 | |
| CN111718418A (zh) | 一种增强基因编辑的融合蛋白及其应用 | |
| JP2024507732A5 (enExample) | ||
| JP2022552892A5 (enExample) | ||
| JP7142815B2 (ja) | ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン | |
| US20250179520A1 (en) | Recombinant aav vectors and uses thereof | |
| RU2820088C1 (ru) | Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| CN121203037B (zh) | 基于整合素改造的特异性靶向心脏及肌肉的aav9优势突变体 |