JP6591956B2 - Mps1を治療するための組成物および方法 - Google Patents

Mps1を治療するための組成物および方法 Download PDF

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JP6591956B2
JP6591956B2 JP2016501864A JP2016501864A JP6591956B2 JP 6591956 B2 JP6591956 B2 JP 6591956B2 JP 2016501864 A JP2016501864 A JP 2016501864A JP 2016501864 A JP2016501864 A JP 2016501864A JP 6591956 B2 JP6591956 B2 JP 6591956B2
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ウイルソン,ジエームズ・エム
ガーダ,ブリトニー・エル
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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
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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)

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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

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JP2016512683A5 JP2016512683A5 (enExample) 2017-04-13
JP6591956B2 true JP6591956B2 (ja) 2019-10-16

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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

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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

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US (3) US10137176B2 (enExample)
EP (2) EP2984166B1 (enExample)
JP (4) JP6591956B2 (enExample)
KR (2) KR102581652B1 (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) ES2991471T3 (enExample)
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LT (1) LT2984166T (enExample)
MX (2) MX386059B (enExample)
PL (1) PL2984166T3 (enExample)
PT (1) PT2984166T (enExample)
RU (1) RU2708318C2 (enExample)
SG (2) SG10201707319UA (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019134717A (ja) * 2013-03-15 2019-08-15 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 Mps1を治療するための組成物および方法

Families Citing this family (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2016366560A1 (en) * 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
JP7061067B2 (ja) * 2015-12-14 2022-04-27 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 クリグラー・ナジャー症候群の処置のための組成物
CA3012195A1 (en) * 2016-02-03 2017-08-10 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
CA3011943A1 (en) * 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
SG11201808426XA (en) * 2016-04-15 2018-10-30 Univ Pennsylvania Compositions for treatment of wet age-related macular degeneration
WO2018022511A1 (en) * 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL305149A (en) 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
IL315358A (en) 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
KR102738752B1 (ko) * 2016-12-30 2024-12-06 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 페닐케톤뇨증을 치료하기 위한 유전자 치료
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
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
CN111989396A (zh) 2017-11-30 2020-11-24 宾夕法尼亚州大学信托人 用于iiib型粘多糖贮积病的基因疗法
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
TW202016314A (zh) 2018-05-09 2020-05-01 美商拜奧馬林製藥公司 治療苯丙酮尿症之方法
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
EP3823981A4 (en) 2018-07-17 2022-04-06 Neuromyon Inc. TREATMENT OF NEUROPATHY WITH DNA CONSTRUCTS EXPRESSING IGF-1 ISOFORMS
WO2020160508A1 (en) 2019-01-31 2020-08-06 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
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
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
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
KR102590276B1 (ko) * 2021-01-04 2023-10-16 부산대학교 산학협력단 조직 특이적 발현을 위한 융합 프로모터 및 이의 용도
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
ES3061188T3 (en) 2021-09-08 2026-03-31 Covestro Deutschland Ag Succinic acid based polyester polyols
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
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treating 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
AU4072499A (en) * 1998-05-13 1999-11-29 Harbor-Ucla Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
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
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
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
US7155804B2 (en) 2003-09-17 2007-01-02 Moog Inc. Method of forming an electric motor
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and 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
BR112012010755A2 (pt) * 2009-11-05 2015-09-22 Proyecto Biomedicina Cima Sl constructo de gene que permite a expressão hepato-específica induzível de um polinucleotídeo de interesse em resposta a um agente indutor, vetor, genoma viral recombinante, vírion, método in vitro para a expressão de um polinucleotídeo de interesse em uma célula de origem hepática, composição farmacêutica e operador-promotor bi-direcional induzível adequado para a expressão hepato-específica induzível de dois polinucleotídeos de interesse por um agente indutor
EP2333074A1 (en) * 2009-12-14 2011-06-15 Robert Steinfeld Substances and methods for the treatment of lysosmal storage diseases
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2561073B1 (en) 2010-04-23 2016-08-24 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
KR101865433B1 (ko) * 2010-10-22 2018-07-13 큐알엔에이, 인크. 알파-l 이두로니다아제 (idua)에 대한 자연 안티센스 전사체의 저해에 의한 idua 관련된 질환의 치료
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
KR20150088292A (ko) 2012-11-21 2015-07-31 알큐엑스 파마슈티컬스, 인크. 광범위한 마크로사이클릭 항생제
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
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
EP3204503A1 (en) 2014-10-06 2017-08-16 Arthrogen BV Aav-based gene therapy
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
TWI617573B (zh) 2015-08-06 2018-03-11 中央研究院 用於酵素置換療法的改造酵素
KR102738860B1 (ko) 2015-10-23 2024-12-04 유니버시티 오브 아이오와 리써치 파운데이션 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017123757A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019134717A (ja) * 2013-03-15 2019-08-15 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 Mps1を治療するための組成物および方法

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