JP2019524890A5 - - Google Patents

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JP2019524890A5
JP2019524890A5 JP2019530369A JP2019530369A JP2019524890A5 JP 2019524890 A5 JP2019524890 A5 JP 2019524890A5 JP 2019530369 A JP2019530369 A JP 2019530369A JP 2019530369 A JP2019530369 A JP 2019530369A JP 2019524890 A5 JP2019524890 A5 JP 2019524890A5
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Japan
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nucleic acid
raav
promoter
item
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JP2019530369A
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JP2019524890A (ja
JP7165410B2 (ja
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Priority claimed from PCT/US2017/047589 external-priority patent/WO2018035451A1/en
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JP2019530369A 2016-08-19 2017-08-18 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物 Active JP7165410B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022111264A JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662377297P 2016-08-19 2016-08-19
US62/377,297 2016-08-19
PCT/US2017/047589 WO2018035451A1 (en) 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus

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JP2022111264A Division JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

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JP2019524890A JP2019524890A (ja) 2019-09-05
JP2019524890A5 true JP2019524890A5 (enExample) 2020-12-24
JP7165410B2 JP7165410B2 (ja) 2022-11-04

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JP2019530369A Active JP7165410B2 (ja) 2016-08-19 2017-08-18 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
JP2022111264A Active JP7653150B2 (ja) 2016-08-19 2022-07-11 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物

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Country Status (9)

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US (2) US11207382B2 (enExample)
EP (1) EP3500278B1 (enExample)
JP (2) JP7165410B2 (enExample)
KR (2) KR102759317B1 (enExample)
CN (1) CN109843306A (enExample)
AU (1) AU2017313844B2 (enExample)
CA (1) CA3073137A1 (enExample)
ES (1) ES2982489T3 (enExample)
WO (1) WO2018035451A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11207382B2 (en) 2016-08-19 2021-12-28 University Of Florida Research Foundation, Incorporated Compositions for treating conditions using recombinant self-complementary adeno-associated virus
KR20250121159A (ko) * 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
RS64499B1 (sr) 2018-01-17 2023-09-29 Meiragtx Uk Ii Ltd Modifikovani protein kapsida raav za gensku terapiju
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
TW202045529A (zh) 2019-02-15 2020-12-16 美商聖加莫治療股份有限公司 用於生產重組aav之組合物及方法
KR20220081971A (ko) * 2019-07-15 2022-06-16 메이라지티엑스 유케이 Ii 리미티드 관절염 질환 치료용 변형된 aav 캡시드 단백질
IL294278A (en) * 2019-12-31 2022-08-01 Swanbio Therapeutics Ltd Improved aav-abcd1 constructs and use for the treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156304A (en) 1990-12-20 2000-12-05 University Of Pittsburgh Of The Commonwealth System Of Higher Education Gene transfer for studying and treating a connective tissue of a mammalian host
US5747072A (en) 1993-07-30 1998-05-05 University Of Michigan Adenoviral-mediated gene transfer to synovial cells in vivo
EP0690673A4 (en) * 1993-12-14 1996-05-29 Univ Pittsburgh SYSTEMIC GENETIC TREATMENT OF TISSUE DISEASES
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
BRPI9908967B1 (pt) 1998-03-20 2017-05-30 Benitec Australia Ltd processos para reprimir, retardar ou de outro modo reduzir a expressão de um gene alvo em uma célula de planta
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
EP1180159B1 (en) 1999-05-28 2008-09-03 Targeted Genetics Corporation Methods and compositions for lowering the level of tumor necrosis factor (tnf) in tnf-associated disorders
WO2001055361A2 (en) 2000-01-26 2001-08-02 Chiron Corporation Recombinant aav packaging systems
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
DE10056210A1 (de) 2000-11-13 2002-05-29 Arimedes Biotechnology Gmbh Virales Expressionssystem
JP2004519230A (ja) 2001-02-06 2004-07-02 メルク　パテント　ゲゼルシャフト　ミット　ベシュレンクテル　ハフトング 低減された免疫原性を有する修飾されたインターロイキン−１受容体アンタゴニスト（ｉｌ−１ｒａ）
KR100472297B1 (ko) 2001-09-27 2005-03-07 주식회사 두산 리소인지질의 수용성 조성물
KR100471942B1 (ko) 2002-03-19 2005-03-09 주식회사 에스원 복합형 카드와 카드정보 등록 제어시스템
DE10318048A1 (de) 2003-04-17 2004-11-04 Axaron Bioscience Ag Nicht fluoreszierende, durch Proteolyse zur Fluoreszenz aktivierbare Reporterproteine und ihre Verwendung zur Detektion Protease-abhängiger Ereignisse
US8529885B2 (en) 2003-09-01 2013-09-10 Academisch Medisch Centrum AAV vectors for in vivo gene therapy of rheumatoid arthritis
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US7196112B2 (en) 2004-07-16 2007-03-27 Biogen Idec Ma Inc. Cell adhesion inhibitors
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
FR2891544A1 (fr) 2005-09-30 2007-04-06 Genethon Ass Loi De 1901 Substrat proteique pour la detection de l'activite calpaine 3
US20090105148A1 (en) 2006-03-23 2009-04-23 Caritas St. Elizabeth Medical Center Of Boston, Inc. Compositions and methods for treating myocardial infarction
WO2007124148A2 (en) 2006-04-21 2007-11-01 The University Of North Carolina At Chapel Hill Treatment of connective tissue disorders
US20080187576A1 (en) 2006-06-16 2008-08-07 University Of Florida Research Foundation, Inc. Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
US20080166762A1 (en) 2006-09-27 2008-07-10 Reliance Life Science Pvt.Ltd. HETEROLOGOUS PRODUCTION OF INTERLEUKIN 1 RECEPTOR ANTAGONIST (IL-1Ra) IN PICHIA PASTORIS
US7452696B2 (en) 2006-10-27 2008-11-18 National Taiwan University Recombinant plasmid and method for expressing hepatitis B viral antigens and virions in vivo
US7892824B2 (en) 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
GB0708376D0 (en) * 2007-05-01 2007-06-06 Alligator Bioscience Ab Novel polypeptides and uses thereof
US8961994B2 (en) * 2008-11-17 2015-02-24 Vgx Pharmaceuticals, Llc DNA constructs eliciting immune response against flavivirus and effective adjuvants
US9610363B2 (en) 2009-04-16 2017-04-04 President And Fellows Of Harvard College Methods for inhibiting starvation of a cell
AU2010300813A1 (en) 2009-09-29 2012-04-26 Gilead Biologics, Inc. Methods and compositions for treatment of ocular fibrosis
DE202009017772U1 (de) 2009-12-10 2011-04-21 Orthogen Ag Kombinationspräparate mit Cytokin-Antagonist und Corticosteroid
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012047093A1 (en) 2010-10-05 2012-04-12 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of the sjögren's syndrome
KR101333958B1 (ko) * 2010-10-20 2013-11-27 주식회사 한독 인간 인터루킨-1 수용체 길항제-하이브리드 Ｆｃ 융합단백질
US8736207B2 (en) 2011-01-03 2014-05-27 General Electric Company Method and system for power conversion
KR20150080033A (ko) 2011-02-08 2015-07-08 애브비 인코포레이티드 골관절염 및 통증의 치료 방법
US20130021786A1 (en) 2011-07-21 2013-01-24 Noble Matthew D Utility bag lighting
PL2623604T3 (pl) * 2012-02-02 2015-07-31 Baylor College Medicine Biologiczny system dostarczania i ekspresji oparty na adenowirusie do zastosowania w leczeniu choroby zwyrodnieniowej stawów
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
CN103509100B (zh) * 2012-06-15 2017-10-27 上海百迈博制药有限公司 一种白细胞介素‑1受体拮抗剂突变体
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
PL2948553T3 (pl) * 2013-01-25 2020-11-16 Baylor College Of Medicine Adenowirusowy system dostarczania i ekspresji terapii genowej zależny od pomocnika
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
CN105682674A (zh) 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
WO2015044292A1 (en) 2013-09-26 2015-04-02 Universitat Autonoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
DE102013220859B4 (de) 2013-10-15 2016-09-08 Plasmidfactory Gmbh & Co. Kg Minicircles mit viralen Expressionskassetten und ihre Verwendung zur Transformation von Zellen zur Erzeugung rekombinanter Viren oder viraler Genvektoren
US9534225B2 (en) 2014-04-15 2017-01-03 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR)
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
EP3137497B2 (en) * 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
EP3500279A4 (en) 2016-08-19 2020-04-22 Calimmune, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANINE CONDITIONS USING A RECOMBINANT SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS.
US11207382B2 (en) 2016-08-19 2021-12-28 University Of Florida Research Foundation, Incorporated Compositions for treating conditions using recombinant self-complementary adeno-associated virus
WO2018035441A1 (en) * 2016-08-19 2018-02-22 Colorado State University Research Foundation Methods and compositions for treating equine conditions using recombinant self-complementary adeno-associated virus
KR20250121159A (ko) * 2016-12-07 2025-08-11 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA

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