JP2022541793A5 - - Google Patents

Info

Publication number
JP2022541793A5
JP2022541793A5 JP2022503802A JP2022503802A JP2022541793A5 JP 2022541793 A5 JP2022541793 A5 JP 2022541793A5 JP 2022503802 A JP2022503802 A JP 2022503802A JP 2022503802 A JP2022503802 A JP 2022503802A JP 2022541793 A5 JP2022541793 A5 JP 2022541793A5
Authority
JP
Japan
Prior art keywords
raav
raav vector
seq
gene
vector according
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
JP2022503802A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022541793A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/042663 external-priority patent/WO2021016126A1/en
Publication of JP2022541793A publication Critical patent/JP2022541793A/ja
Publication of JP2022541793A5 publication Critical patent/JP2022541793A5/ja
Pending legal-status Critical Current

Links

JP2022503802A 2019-07-19 2020-07-17 ヒトにおける心筋症のためのaav心臓遺伝子治療 Pending JP2022541793A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962876540P 2019-07-19 2019-07-19
US62/876,540 2019-07-19
PCT/US2020/042663 WO2021016126A1 (en) 2019-07-19 2020-07-17 Aav cardiac gene therapy for cardiomyopathy in humans

Publications (2)

Publication Number Publication Date
JP2022541793A JP2022541793A (ja) 2022-09-27
JP2022541793A5 true JP2022541793A5 (enExample) 2023-07-26

Family

ID=74193792

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022503802A Pending JP2022541793A (ja) 2019-07-19 2020-07-17 ヒトにおける心筋症のためのaav心臓遺伝子治療

Country Status (16)

Country Link
US (1) US20220265858A1 (enExample)
EP (1) EP3999074A4 (enExample)
JP (1) JP2022541793A (enExample)
KR (1) KR20220034801A (enExample)
CN (1) CN114206351A (enExample)
AR (1) AR122289A1 (enExample)
AU (1) AU2020316339A1 (enExample)
BR (1) BR112022000898A2 (enExample)
CA (1) CA3142534A1 (enExample)
CL (1) CL2022000115A1 (enExample)
CO (1) CO2022001686A2 (enExample)
IL (1) IL289667A (enExample)
MX (1) MX2022000767A (enExample)
SG (1) SG11202112140SA (enExample)
TW (1) TW202117017A (enExample)
WO (1) WO2021016126A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022140402A1 (en) * 2020-12-23 2022-06-30 University Of Florida Research Foundation, Incorporated Increased packaging efficiency of vector for cardiac gene therapy
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
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
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
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19915485A1 (de) * 1999-04-07 2000-10-19 Hugo A Katus Therapie der Herzinsuffizienz
WO2002002148A2 (en) * 2000-06-30 2002-01-10 Collateral Therapeutics, Inc. Dual recombinant gene therapy compositions and methods of use
WO2008054713A2 (en) * 2006-10-30 2008-05-08 Thomas Jefferson University Tissue specific gene therapy treatment
EP2295072A1 (en) * 2009-09-15 2011-03-16 Max-Delbrück-Centrum für Molekulare Medizin (MDC) Use of ARC for inhibiting cell death during liver failure
CA2925691A1 (en) * 2013-10-01 2015-04-09 Ruprecht-Karls-Univeristat Heidelberg S100 based treatment of cardiac power failure
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
WO2019073058A1 (en) * 2017-10-12 2019-04-18 Universität Heidelberg GENE THERAPY OF CARDIAC ARRHYTHMIA BASED ON KCNK3
CA3100280A1 (en) * 2018-06-08 2019-12-12 University Of Florida Research Foundation, Incorporated Aav cardiac gene therapy for cardiomyopathy

Similar Documents

Publication Publication Date Title
JP2022541793A5 (enExample)
US12257317B2 (en) Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
JP2023538519A5 (enExample)
US12151001B2 (en) Optimized expression cassettes for gene therapy
US12564647B2 (en) Optimized expression cassettes for gene therapy
JP2023536618A5 (enExample)
JP2020533959A5 (enExample)
US7655467B2 (en) Compositions and methods for systemic nucleic acid sequence delivery
JP2023537358A5 (enExample)
JP2024546103A5 (enExample)
JP2024545507A5 (enExample)
CN115885043A (zh) 用于dwarf开放阅读框的腺相关病毒载体
JP2024515902A5 (enExample)
WO2025214477A1 (en) Treatment of genetic cardiomyopathies with aav gene therapy vectors
AU2020366242A1 (en) Materials and methods for the treatment of disorders associated mutations in the IRF2BPL gene
CN116179605B (zh) 一种重组腺相关病毒载体及其应用
RU2825667C2 (ru) Выделенный модифицированный белок VPI капсида аденоассоциированного вируса 9 серотипа (AAV9), капсид и вектор на его основе
WO2025162449A1 (en) Compositions and methods for the treatment of heart disease
RU2023105139A (ru) Генная терапия в отношении плакофилина-2 (pkp2) с применением вектора на основе aav
JP2026502465A (ja) Aavベクターを用いたプラコフィリン-2(pkp2)遺伝子療法
US20240254181A1 (en) Recombinant adeno-associated virus products and methods for treating dystroglycanopathies and laminin-deficient muscular dystrophies
WO2024064863A2 (en) Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
EA048262B1 (ru) Повышенная эффективность упаковки вектора для генотерапии сердечных заболеваний
TW202242120A (zh) 用於心臟基因療法之載體的增加之包裝效率
CN119968387A (zh) 用于治疗癫痫的表达前强啡肽原变体的基因治疗载体