JP2021516046A5 - - Google Patents

Info

Publication number
JP2021516046A5
JP2021516046A5 JP2020545160A JP2020545160A JP2021516046A5 JP 2021516046 A5 JP2021516046 A5 JP 2021516046A5 JP 2020545160 A JP2020545160 A JP 2020545160A JP 2020545160 A JP2020545160 A JP 2020545160A JP 2021516046 A5 JP2021516046 A5 JP 2021516046A5
Authority
JP
Japan
Prior art keywords
numbering
capsid
sequence
amino acid
aav8
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.)
Pending
Application number
JP2020545160A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021516046A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/019804 external-priority patent/WO2019168961A1/en
Publication of JP2021516046A publication Critical patent/JP2021516046A/ja
Publication of JP2021516046A5 publication Critical patent/JP2021516046A5/ja
Priority to JP2023131761A priority Critical patent/JP2023159235A/ja
Priority to JP2024005924A priority patent/JP2024041967A/ja
Priority to JP2025188265A priority patent/JP2026027394A/ja
Pending legal-status Critical Current

Links

JP2020545160A 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 Pending JP2021516046A (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2023131761A JP2023159235A (ja) 2018-02-27 2023-08-14 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
JP2024005924A JP2024041967A (ja) 2018-02-27 2024-01-18 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
JP2025188265A JP2026027394A (ja) 2018-02-27 2025-11-07 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 (11)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US62/635,964 2018-02-27
US201862677471P 2018-05-29 2018-05-29
US201862667585P 2018-05-29 2018-05-29
US62/667,585 2018-05-29
US62/677,471 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US62/703,670 2018-07-26
US201862722382P 2018-08-24 2018-08-24
US62/722,382 2018-08-24
PCT/US2019/019804 WO2019168961A1 (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 (2)

Application Number Title Priority Date Filing Date
JP2023131761A Division JP2023159235A (ja) 2018-02-27 2023-08-14 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
JP2024005924A Division JP2024041967A (ja) 2018-02-27 2024-01-18 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 (2)

Publication Number Publication Date
JP2021516046A JP2021516046A (ja) 2021-07-01
JP2021516046A5 true JP2021516046A5 (https=) 2022-02-18

Family

ID=67805569

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2020545160A Pending JP2021516046A (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
JP2023131761A Pending JP2023159235A (ja) 2018-02-27 2023-08-14 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
JP2024005924A Pending JP2024041967A (ja) 2018-02-27 2024-01-18 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
JP2025188265A Pending JP2026027394A (ja) 2018-02-27 2025-11-07 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 (3)

Application Number Title Priority Date Filing Date
JP2023131761A Pending JP2023159235A (ja) 2018-02-27 2023-08-14 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
JP2024005924A Pending JP2024041967A (ja) 2018-02-27 2024-01-18 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
JP2025188265A Pending JP2026027394A (ja) 2018-02-27 2025-11-07 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 (13)

Country Link
US (1) US20200407750A1 (https=)
EP (1) EP3758724A4 (https=)
JP (4) JP2021516046A (https=)
KR (1) KR20210010434A (https=)
CN (2) CN119842635A (https=)
AU (1) AU2019227726B2 (https=)
BR (1) BR112020017348A2 (https=)
CA (1) CA3091806A1 (https=)
CL (2) CL2020002200A1 (https=)
IL (1) IL276859B2 (https=)
MX (1) MX2020008933A (https=)
SG (1) SG11202008182TA (https=)
WO (1) WO2019168961A1 (https=)

Families Citing this family (42)

* 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
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
BR112021006052A2 (pt) 2018-10-01 2021-09-08 Ultragenyx Pharmaceutical Inc. Terapia gênica para tratar acidemia propiônica
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
WO2021067598A1 (en) 2019-10-04 2021-04-08 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
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
WO2023172491A1 (en) 2022-03-07 2023-09-14 Ultragenyx Pharmaceutical Inc. Modified batch aav production systems and methods
JP2023514204A (ja) * 2020-02-14 2023-04-05 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Ｃｄｋｌ５欠損障害を処置するための遺伝子治療
WO2021202532A1 (en) 2020-03-31 2021-10-07 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
KR20230003569A (ko) * 2020-04-27 2023-01-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물
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
JP2023550581A (ja) * 2020-10-29 2023-12-04 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 Ａａｖカプシド及びそれを含有する組成物
TW202237850A (zh) 2020-12-01 2022-10-01 賓州大學委員會 具有組織特異性靶向基序的新穎構成物及含有其之組成物
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)
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
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
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
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
CN115011601B (zh) * 2022-06-27 2023-07-21 山东大学齐鲁医院 一种干扰JUND表达的shRNA、重组腺相关病毒载体及其应用
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
CN116350801A (zh) * 2022-11-22 2023-06-30 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
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
WO2024138129A2 (en) * 2022-12-23 2024-06-27 Spark Therapeutics, Inc. Adeno-associated virus formulations
KR20260021096A (ko) * 2023-05-03 2026-02-12 보이저 테라퓨틱스, 인크. Cdkl5 결핍과 관련된 장애의 치료를 위한 조성물 및 방법
WO2024229161A1 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
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
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
WO2026074494A1 (en) 2024-10-03 2026-04-09 Sarepta Therapeutics, Inc. Modified viral capsids and uses thereof

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
NZ701693A (en) * 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
WO2017100674A1 (en) * 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
SG11201808812RA (en) * 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
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

Similar Documents

Publication Publication Date Title
JP2021516046A5 (https=)
JP2021515548A5 (https=)
US12534717B2 (en) AAV capsid proteins for nucleic acid transfer
JP2020510429A5 (https=)
JP2022530544A5 (https=)
JP2022530656A5 (https=)
IL276859B1 (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
ES2580044T3 (es) Método de incremento de la función de un vector de AAV
JP2022530633A5 (https=)
JP2021106619A5 (https=)
JP2022529631A5 (https=)
FI3589730T3 (fi) Kladin f adenoassosioitunut virus (aav) -vektori ja sen käyttötapoja
JP2019533803A5 (https=)
AU2022259813A1 (en) Novel adeno-associated virus capsid proteins
JP2024009857A5 (https=)
JP2020505936A5 (https=)
JP2022190081A5 (https=)
JP2007507223A5 (https=)
JP2020522269A5 (https=)
JP2018108113A5 (https=)
IL317938A (en) Recombinant viral vectors with altered tropism and their uses for the targeted introduction of genetic material into human cells
JP2021514659A5 (https=)
HRP20241180T1 (hr) Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva
JP2022520803A5 (https=)
JP2022524434A5 (https=)