JP2023537625A5 - - Google Patents

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Publication number
JP2023537625A5
JP2023537625A5 JP2023511556A JP2023511556A JP2023537625A5 JP 2023537625 A5 JP2023537625 A5 JP 2023537625A5 JP 2023511556 A JP2023511556 A JP 2023511556A JP 2023511556 A JP2023511556 A JP 2023511556A JP 2023537625 A5 JP2023537625 A5 JP 2023537625A5
Authority
JP
Japan
Prior art keywords
seq
aav
acid sequence
amino acid
encoding
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
JP2023511556A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023537625A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/045945 external-priority patent/WO2022036220A1/en
Publication of JP2023537625A publication Critical patent/JP2023537625A/ja
Publication of JP2023537625A5 publication Critical patent/JP2023537625A5/ja
Pending legal-status Critical Current

Links

JP2023511556A 2020-08-14 2021-08-13 新規aavカプシド及びそれを含む組成物 Pending JP2023537625A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063065616P 2020-08-14 2020-08-14
US63/065,616 2020-08-14
US202063109734P 2020-11-04 2020-11-04
US63/109,734 2020-11-04
PCT/US2021/045945 WO2022036220A1 (en) 2020-08-14 2021-08-13 Novel aav capsids and compositions containing same

Publications (2)

Publication Number Publication Date
JP2023537625A JP2023537625A (ja) 2023-09-04
JP2023537625A5 true JP2023537625A5 (https=) 2024-08-13

Family

ID=77595666

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023511556A Pending JP2023537625A (ja) 2020-08-14 2021-08-13 新規aavカプシド及びそれを含む組成物

Country Status (11)

Country Link
US (1) US20230287451A1 (https=)
EP (1) EP4196170A1 (https=)
JP (1) JP2023537625A (https=)
KR (1) KR20230051208A (https=)
CN (1) CN116209769A (https=)
AU (1) AU2021325954A1 (https=)
CA (1) CA3188956A1 (https=)
IL (1) IL300410A (https=)
MX (1) MX2023001863A (https=)
TW (1) TW202221127A (https=)
WO (1) WO2022036220A1 (https=)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN119173269A (zh) * 2022-03-03 2024-12-20 宾夕法尼亚州大学信托人 用于递送glp-1受体激动剂融合物的aav载体
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
CN117801116A (zh) * 2022-09-30 2024-04-02 上海玮美基因科技有限责任公司 一种融合型新型腺相关病毒及其应用
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
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
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
WO2025039622A1 (zh) * 2023-08-23 2025-02-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途
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
CN120677245A (zh) * 2024-01-25 2025-09-19 鼐济医药科技(杭州)有限公司 Aav载体、载体组合物、嵌合载体和治疗或预防sma的方法

Family Cites Families (16)

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Publication number Priority date Publication date Assignee Title
US6174666B1 (en) 1992-03-27 2001-01-16 The United States Of America As Represented By The Department Of Health And Human Services Method of eliminating inhibitory/instability regions from mRNA
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
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
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.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
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
EP2434420A3 (en) 2003-08-01 2012-07-25 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids

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