JP2021515548A5 - - Google Patents

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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
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JP
Japan
Prior art keywords
seq
capsid
acid sequence
proteins
amino acid
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Application number
JP2020545159A
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English (en)
Japanese (ja)
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JP7498665B2 (ja
JP2021515548A (ja
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Priority claimed from PCT/US2019/019861 external-priority patent/WO2019169004A1/en
Publication of JP2021515548A publication Critical patent/JP2021515548A/ja
Publication of JP2021515548A5 publication Critical patent/JP2021515548A5/ja
Priority to JP2024088641A priority Critical patent/JP2024115558A/ja
Application granted granted Critical
Publication of JP7498665B2 publication Critical patent/JP7498665B2/ja
Active legal-status Critical Current
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JP2020545159A 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 Active JP7498665B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024088641A JP2024115558A (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
US201862635964P 2018-02-27 2018-02-27
US201862635968P 2018-02-27 2018-02-27
US62/635,968 2018-02-27
US62/635,964 2018-02-27
US201862663788P 2018-04-27 2018-04-27
US201862663797P 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
US201862667888P 2018-05-07 2018-05-07
US201862667881P 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
US62/677,474 2018-05-29
US62/667,585 2018-05-29
US62/677,471 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862703673P 2018-07-26 2018-07-26
US62/703,673 2018-07-26
US62/703,670 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 JP2024115558A (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 Pending JP2024115558A (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 Pending JP2024115558A (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) CN112236443B (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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
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
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
SG11202111375RA (en) 2019-04-29 2021-11-29 Univ Pennsylvania Novel aav capsids and compositions containing same
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
EP4048681A4 (en) * 2019-10-21 2024-03-20 The Trustees of The University of Pennsylvania AAV3B VARIANTS WITH IMPROVED PRODUCTION YIELD AND LIVER TROPISM
CN114902051B (zh) * 2020-01-03 2026-04-17 萨勒普塔医疗公司 用于分析aav衣壳蛋白的方法
US12497612B2 (en) 2020-03-09 2025-12-16 University Of Massachusetts Gene replacement therapy for FOXG1 syndrome
US12552838B2 (en) 2020-04-20 2026-02-17 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
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Useful preparations for the treatment of Charcot-Marietot disease
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
WO2022082109A1 (en) * 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2025250683A1 (en) * 2024-05-29 2025-12-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid

Family Cites Families (38)

* Cited by examiner, † Cited by third party
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
US6548286B1 (en) 1997-04-14 2003-04-15 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
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
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
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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
AU2015320694B2 (en) * 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
CA2969145A1 (en) * 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
CN108779167A (zh) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 鞘内施用腺伴随病毒载体用于基因治疗
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
SG11201808812RA (en) * 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
IL305149A (en) * 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CA3033856A1 (en) 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
PT3589730T (pt) 2017-02-28 2024-02-28 Univ Pennsylvania Vetor de vírus adenoassociado (aav) de clado f e suas utilizações
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

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