JP2022529457A5 - - Google Patents

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Publication number
JP2022529457A5
JP2022529457A5 JP2021561742A JP2021561742A JP2022529457A5 JP 2022529457 A5 JP2022529457 A5 JP 2022529457A5 JP 2021561742 A JP2021561742 A JP 2021561742A JP 2021561742 A JP2021561742 A JP 2021561742A JP 2022529457 A5 JP2022529457 A5 JP 2022529457A5
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JP
Japan
Prior art keywords
seq
raav
variant
polynucleotide
acid sequence
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
Application number
JP2021561742A
Other languages
English (en)
Japanese (ja)
Other versions
JP7731801B2 (ja
JP2022529457A (ja
Filing date
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Application filed filed Critical
Priority claimed from PCT/US2020/028264 external-priority patent/WO2020214668A1/en
Publication of JP2022529457A publication Critical patent/JP2022529457A/ja
Publication of JP2022529457A5 publication Critical patent/JP2022529457A5/ja
Application granted granted Critical
Publication of JP7731801B2 publication Critical patent/JP7731801B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021561742A 2019-04-15 2020-04-15 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス Active JP7731801B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962833972P 2019-04-15 2019-04-15
US62/833,972 2019-04-15
US201962926308P 2019-10-25 2019-10-25
US62/926,308 2019-10-25
US202062967214P 2020-01-29 2020-01-29
US62/967,214 2020-01-29
PCT/US2020/028264 WO2020214668A1 (en) 2019-04-15 2020-04-15 Compositions and methods for treatment of cystic fibrosis

Publications (3)

Publication Number Publication Date
JP2022529457A JP2022529457A (ja) 2022-06-22
JP2022529457A5 true JP2022529457A5 (https=) 2023-04-18
JP7731801B2 JP7731801B2 (ja) 2025-09-01

Family

ID=70847479

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021561742A Active JP7731801B2 (ja) 2019-04-15 2020-04-15 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス

Country Status (14)

Country Link
US (1) US20220241436A1 (https=)
EP (1) EP3955971A1 (https=)
JP (1) JP7731801B2 (https=)
KR (1) KR20220044899A (https=)
CN (1) CN114641318A (https=)
AU (1) AU2020260076A1 (https=)
BR (1) BR112021020708A2 (https=)
CA (1) CA3137015A1 (https=)
CL (1) CL2021002702A1 (https=)
IL (1) IL287260A (https=)
MX (1) MX2021012681A (https=)
PH (1) PH12021552610A1 (https=)
SG (1) SG11202111334SA (https=)
WO (1) WO2020214668A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3016985C (en) 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
BR112021020706A2 (pt) * 2019-04-15 2022-03-15 Spirovant Sciences Inc Métodos e composições para expressão de transgene
WO2022006253A2 (en) * 2020-06-30 2022-01-06 University Of Iowa Research Foundation Methods and compositions for administering recombinant viral vectors
WO2022221684A1 (en) 2021-04-15 2022-10-20 Spirovant Sciences, Inc. Methods and compositions for treatment of cystic fibrosis
WO2026072721A1 (en) * 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy

Family Cites Families (15)

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Publication number Priority date Publication date Assignee Title
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
AU5445998A (en) 1996-11-19 1998-06-10 Surgx Corporation A transient voltage protection device and method of making same
JP2001506133A (ja) 1996-12-18 2001-05-15 ターゲティッド ジェネティクス コーポレイション 組換えaavベクターの産生における使用のための、aavスプリット−パッケージング遺伝子およびこのような遺伝子を含む細胞株
AU2004227358B8 (en) 2003-03-31 2009-12-10 Targeted Genetics Corporation Compounds and methods to enhance rAAV transduction
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US7407801B2 (en) * 2003-12-05 2008-08-05 University Of Iowa Research Foundation Truncated CMV promoters and vectors containing same
RS57739B1 (sr) * 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
WO2014168953A1 (en) * 2013-04-08 2014-10-16 University Of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
US11000597B2 (en) * 2015-01-21 2021-05-11 University Of Florida Research Foundation, Incorporated Engineered receptor/ligand system for delivery of therapeutic agents
CA3016985C (en) * 2016-03-07 2023-07-04 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US10550405B2 (en) * 2017-03-15 2020-02-04 The University Of North Carolina At Chapel Hill Rational polyploid adeno-associated virus vectors and methods of making and using the same
BR112021020706A2 (pt) * 2019-04-15 2022-03-15 Spirovant Sciences Inc Métodos e composições para expressão de transgene

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