JP2016512683A5 - - Google Patents

Download PDF

Info

Publication number
JP2016512683A5
JP2016512683A5 JP2016501864A JP2016501864A JP2016512683A5 JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5 JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016512683 A5 JP2016512683 A5 JP 2016512683A5
Authority
JP
Japan
Prior art keywords
seq
raav
enhancer
change
hidua
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
JP2016501864A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016512683A (ja
JP6591956B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2014/025509 external-priority patent/WO2014151341A1/en
Publication of JP2016512683A publication Critical patent/JP2016512683A/ja
Publication of JP2016512683A5 publication Critical patent/JP2016512683A5/ja
Application granted granted Critical
Publication of JP6591956B2 publication Critical patent/JP6591956B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2016501864A 2013-03-15 2014-03-13 Mps1を治療するための組成物および方法 Active JP6591956B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361788724P 2013-03-15 2013-03-15
US61/788,724 2013-03-15
PCT/US2014/025509 WO2014151341A1 (en) 2013-03-15 2014-03-13 Compositions and methods for treating mpsi

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2019079881A Division JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法

Publications (3)

Publication Number Publication Date
JP2016512683A JP2016512683A (ja) 2016-05-09
JP2016512683A5 true JP2016512683A5 (enExample) 2017-04-13
JP6591956B2 JP6591956B2 (ja) 2019-10-16

Family

ID=51580934

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2016501864A Active JP6591956B2 (ja) 2013-03-15 2014-03-13 Mps1を治療するための組成物および方法
JP2019079881A Pending JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法
JP2020148103A Active JP7168621B2 (ja) 2013-03-15 2020-09-03 Mps1を治療するための組成物および方法
JP2022172314A Active JP7553527B2 (ja) 2013-03-15 2022-10-27 Mps1を治療するための組成物および方法

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2019079881A Pending JP2019134717A (ja) 2013-03-15 2019-04-19 Mps1を治療するための組成物および方法
JP2020148103A Active JP7168621B2 (ja) 2013-03-15 2020-09-03 Mps1を治療するための組成物および方法
JP2022172314A Active JP7553527B2 (ja) 2013-03-15 2022-10-27 Mps1を治療するための組成物および方法

Country Status (21)

Country Link
US (3) US10137176B2 (enExample)
EP (2) EP3747998B1 (enExample)
JP (4) JP6591956B2 (enExample)
KR (2) KR102346455B1 (enExample)
CN (1) CN105026554B (enExample)
AU (2) AU2014235096B2 (enExample)
BR (1) BR112015021036A2 (enExample)
CA (1) CA2901328C (enExample)
CY (1) CY1123169T1 (enExample)
DK (1) DK2984166T3 (enExample)
ES (2) ES2805355T3 (enExample)
HR (1) HRP20201063T1 (enExample)
HU (1) HUE051373T2 (enExample)
LT (1) LT2984166T (enExample)
MX (2) MX386059B (enExample)
PL (1) PL2984166T3 (enExample)
PT (1) PT2984166T (enExample)
RU (1) RU2708318C2 (enExample)
SG (2) SG10201707319UA (enExample)
SI (1) SI2984166T1 (enExample)
WO (1) WO2014151341A1 (enExample)

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3747998B1 (en) 2013-03-15 2024-07-24 The Trustees of the University of Pennsylvania Compositions for treating mpsi
WO2015164778A1 (en) * 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
EP3400304B1 (en) * 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11241506B2 (en) 2015-12-14 2022-02-08 The Trustees Of The University Of Pennsylvania Composition for treatment of Crigler-Najjar syndrome
US10973929B2 (en) * 2016-02-03 2021-04-13 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type I
WO2017136533A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Methods for treating diagnosing, and monitoring treatment of mucopolysaccharidoses
MX2018009426A (es) * 2016-02-22 2018-12-19 Univ North Carolina Chapel Hill Vector aav-idua para el tratamiento de ceguera asociada con mucopolisacaridosis i (mps i).
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
MA44873A (fr) * 2016-04-15 2019-03-13 Univ Pennsylvania Composition pour le traitement de la dégénérescence maculaire liée a l'âge exsudative
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL263801B2 (en) 2016-07-26 2024-01-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
IL264872B2 (en) 2016-08-18 2025-02-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
WO2018126112A1 (en) * 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
JP2020526514A (ja) 2017-07-06 2020-08-31 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 ムコ多糖症i型を治療するための遺伝子治療
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
CN107699590A (zh) * 2017-10-13 2018-02-16 成都中医药大学 一种制备重组人α‑L‑艾杜糖醛酸酶的方法
CA3083761A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iiib
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
EP3780952A4 (en) 2018-04-16 2022-03-09 The Trustees of the University of Pennsylvania COMPOSITIONS AND METHODS OF TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
WO2020016655A2 (en) 2018-07-17 2020-01-23 Helixmith Co., Ltd. Treatment of neuropathy with dna constructs expressing igf-1 isoforms
US20230193315A1 (en) 2019-01-31 2023-06-22 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
AU2021270447A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
KR20230118075A (ko) 2020-10-09 2023-08-10 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 파브리병 치료를 위한 조성물 및 방법
KR102590276B1 (ko) * 2021-01-04 2023-10-16 부산대학교 산학협력단 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
EP4148078B1 (de) 2021-09-08 2025-11-05 Covestro Deutschland AG Auf bernsteinsäure basierende polyesterpolyole
CN114181318B (zh) * 2021-11-08 2023-08-01 四川大学 一种组织特异性表达穿透血脑屏障的idua融合蛋白的重组腺相关病毒及应用
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CA3247109A1 (en) 2022-04-06 2023-10-12 Univ Pennsylvania Compositions and treatment methods for metastatic breast cancer that is human epidermal growth factor receptor 2 positive and other cancers
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6149909A (en) * 1995-06-23 2000-11-21 Women's And Children's Hospital Synthetic α-L-iduronidase and genetic sequences encoding same
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
CA2328518A1 (en) 1998-05-13 1999-11-18 Harbor-Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
DE60039766D1 (de) 1999-08-09 2008-09-18 Targeted Genetics Corp Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen
US6426208B1 (en) 1999-11-12 2002-07-30 Harbor-Ucla Research And Education Institute Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
RU2196988C2 (ru) * 2000-04-19 2003-01-20 Государственный новосибирский областной клинический диагностический центр Способ диагностики мукополисахаридозов
CA2915124C (en) 2001-11-13 2018-08-14 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
WO2004055157A2 (en) 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
US7155804B2 (en) 2003-09-17 2007-01-02 Moog Inc. Method of forming an electric motor
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP2357010B1 (en) 2005-04-07 2013-06-12 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
US20090062144A1 (en) 2007-04-03 2009-03-05 Nancy Lan Guo Gene signature for prognosis and diagnosis of lung cancer
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
RU2012123145A (ru) * 2009-11-05 2013-12-10 Проекто Де Биомедисина Сима, С.Л. Генный конструкт (варианты), вектор и рекомбинантный вирусный геном на его основе, вирион, их фармацевтическая композиция, способ in vitro экспрессии полинуклеотида в клетке печеночной природы, лекарственное средство, способ лечения заболевания печени (варианты), индуцируемый двунаправленный оператор-промотор
EP2333074A1 (en) * 2009-12-14 2011-06-15 Robert Steinfeld Substances and methods for the treatment of lysosmal storage diseases
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
EP3514232A1 (en) 2010-04-23 2019-07-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012054723A2 (en) * 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
JP6224459B2 (ja) 2011-02-17 2017-11-01 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 組織特異性を改変し、aav9媒介遺伝子導入を改善するための組成物および方法
RS66184B1 (sr) 2011-04-22 2024-12-31 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
ES2697912T3 (es) 2012-07-11 2019-01-29 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de enfermedades monogénicas
CN104903302B (zh) 2012-11-21 2019-03-29 阿奇克斯制药公司 大环广谱抗生素
WO2014125647A1 (ja) 2013-02-18 2014-08-21 富士通オプティカルコンポーネンツ株式会社 光受信装置
EP3747998B1 (en) * 2013-03-15 2024-07-24 The Trustees of the University of Pennsylvania Compositions for treating mpsi
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
EP3800260A1 (en) 2014-09-24 2021-04-07 City of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
JP2017531652A (ja) 2014-10-06 2017-10-26 アルスロジェン ビー.ブイ.Arthrogen B.V. Aavに基づく遺伝子治療
WO2016187017A1 (en) 2015-05-15 2016-11-24 Mcivor R Scott Adeno-associated for therapeutic delivery to central nervous system
US10604747B2 (en) 2015-08-06 2020-03-31 Academia Sinica Engineered enzyme for enzyme replacement therapy
WO2017070678A1 (en) 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3402533B1 (en) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
US10973929B2 (en) 2016-02-03 2021-04-13 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type I
JP2020526514A (ja) 2017-07-06 2020-08-31 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 ムコ多糖症i型を治療するための遺伝子治療

Similar Documents

Publication Publication Date Title
JP2016512683A5 (enExample)
HRP20201063T1 (hr) Pripravci za liječenje mpsi
JP7374119B2 (ja) 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
JP2017512466A5 (enExample)
JP2020528734A5 (enExample)
JP2017513504A5 (enExample)
JP2020514286A5 (enExample)
JP2018522529A5 (enExample)
HRP20201544T1 (hr) Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc)
CN104520428B (zh) 将基因转移到细胞、器官和组织的aav载体组合物和方法
JP2020114235A5 (enExample)
HRP20241180T1 (hr) Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva
JP2018520997A5 (enExample)
JP2020522269A5 (enExample)
JP2017529395A5 (enExample)
RU2017125234A (ru) Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний
JP2018531609A5 (enExample)
JP2017509632A5 (enExample)
JP2018108113A5 (enExample)
JP2017518271A5 (enExample)
JP2020500541A5 (enExample)
JP2019530464A5 (enExample)
JP2019502378A5 (enExample)
KR20240090694A (ko) 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
JP2020510428A5 (enExample)