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

Info

Publication number
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
Authority
JP
Japan
Prior art keywords
raav
sequence
seq
enhancer
gene
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.)
Active
Application number
JP2016501864A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016512683A (ja
JP2016512683A5 (enExample
Inventor
ウイルソン，ジエームズ・エム
ガーダ，ブリトニー・エル
Original Assignee
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
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by 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 filed Critical 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
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

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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 JP2016512683A5 (enExample) 2017-04-13
JP6591956B2 true 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) SG11201507507PA (enExample)
SI (1) SI2984166T1 (enExample)
WO (1) WO2014151341A1 (enExample)

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 (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2876409T3 (es) * 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
MX2018007080A (es) * 2015-12-11 2018-11-12 Univ Pennsylvania Terapia genica para tratar hipercolesterolemia familiar.
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
SG11201806270XA (en) * 2016-02-03 2018-08-30 Univ 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
CN108697774A (zh) 2016-02-22 2018-10-23 北卡罗来纳大学教堂山分校 治疗mps i-相关失明的aav-idua载体
AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
IL262207B1 (en) 2016-04-15 2025-09-01 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
SG11201811603WA (en) 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
CA3034089A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
BR112019013576A2 (pt) * 2016-12-30 2020-02-04 Univ Pennsylvania terapia genica para o tratamento da fenilcetonuria
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
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
JP7575273B2 (ja) 2018-04-16 2024-10-29 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 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
SG11202010830WA (en) 2018-05-09 2020-11-27 Biomarin Pharm 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
CN119390787A (zh) 2019-01-31 2025-02-07 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
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
CN115803064A (zh) 2020-05-12 2023-03-14 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
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
TW202229560A (zh) 2020-10-09 2022-08-01 賓州大學委員會 治療法布瑞氏症之組成物及方法
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
WO2023087019A2 (en) 2021-11-15 2023-05-19 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
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
JP2025512969A (ja) 2022-04-06 2025-04-22 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 Ｈｅｒ２陽性転移性乳がん及び他のがんを治療するための組成物及び方法
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
WO1999058691A2 (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
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
BR122016004546B8 (pt) 2001-11-13 2021-07-27 Univ Pennsylvania vírus adenoassociado recombinante, método de geração do referido vírus e composição compreendendo o referido vírus
DK1453547T3 (en) 2001-12-17 2016-12-05 Univ Pennsylvania ADENOASSOCATED VIRUS (AAV) SEROTYPE 8 SEQUENCES, VECTORS CONTAINING THESE AND APPLICATIONS THEREOF
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
CA3072423A1 (en) 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
EP3409296A1 (en) 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
EP1777906A1 (en) 2005-06-09 2007-04-25 Matsushita Electric Industrial Co., Ltd. Amplitude error compensating apparatus and orthogonality error compensating apparatus
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
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
CN102869779A (zh) 2010-03-29 2013-01-09 宾夕法尼亚大学托管会 药理学诱导的转基因消融系统
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system 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
EP2630241B1 (en) * 2010-10-22 2018-10-17 CuRNA, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
BR112013020734A2 (pt) 2011-02-17 2017-06-13 Univ Pennsylvania composições e métodos para alterar a especificidade do tecido e aprimorar a transferência gênica mediada por aav9
KR20220056884A (ko) 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
JP6225182B2 (ja) 2012-07-11 2017-11-01 サンガモ セラピューティクス， インコーポレイテッド リソソーム貯蔵疾患の処置のための方法および組成物
EA201590871A1 (ru) 2012-11-21 2015-11-30 АрКьюЭкс ФАРМАСЪЮТИКЛС, ИНК. Макроциклические антибиотики широкого спектра действия
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
SG11201507507PA (en) * 2013-03-15 2015-10-29 Univ 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
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
AU2015330092A1 (en) 2014-10-06 2017-04-13 Arthrogen B.V. AAV-based gene therapy
HUE057795T2 (hu) 2015-05-15 2022-06-28 Regenxbio Inc Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására
WO2017024204A1 (en) 2015-08-06 2017-02-09 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
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
EP4215605A1 (en) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Scalable purification method for aav8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
BR112018014288A2 (pt) 2016-01-15 2018-12-18 Univ Minnesota métodos e composições para o tratamento de doença neurológica
SG11201806270XA (en) * 2016-02-03 2018-08-30 Univ 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

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

Also Published As

Publication number Publication date
RU2708318C2 (ru) 2019-12-05
EP3747998B1 (en) 2024-07-24
JP7553527B2 (ja) 2024-09-18
JP2016512683A (ja) 2016-05-09
EP2984166A1 (en) 2016-02-17
AU2014235096A1 (en) 2015-09-10
RU2015144234A (ru) 2017-04-24
JP2023011752A (ja) 2023-01-24
SG10201707319UA (en) 2017-10-30
US20160000887A1 (en) 2016-01-07
KR102581652B1 (ko) 2023-09-22
JP2019134717A (ja) 2019-08-15
AU2020203826B2 (en) 2022-09-15
MX2021010918A (es) 2021-10-01
AU2020203826A1 (en) 2020-07-02
ES2805355T3 (es) 2021-02-11
SG11201507507PA (en) 2015-10-29
CY1123169T1 (el) 2021-10-29
BR112015021036A2 (pt) 2017-10-10
US20200397872A1 (en) 2020-12-24
CN105026554B (zh) 2020-08-11
EP3747998A1 (en) 2020-12-09
WO2014151341A1 (en) 2014-09-25
US10792343B2 (en) 2020-10-06
US10137176B2 (en) 2018-11-27
PT2984166T (pt) 2020-07-16
EP2984166B1 (en) 2020-04-22
HRP20201063T1 (hr) 2020-11-27
US20190111116A1 (en) 2019-04-18
SI2984166T1 (sl) 2020-09-30
MX2015012739A (es) 2016-02-19
EP3747998A9 (en) 2021-08-04
HK1221735A1 (en) 2017-06-09
KR102346455B1 (ko) 2022-01-04
PL2984166T3 (pl) 2021-01-11
CA2901328C (en) 2023-08-01
MX386059B (es) 2025-03-18
KR20150129678A (ko) 2015-11-20
CA2901328A1 (en) 2014-09-25
JP7168621B2 (ja) 2022-11-09
ES2991471T3 (es) 2024-12-03
KR20220005599A (ko) 2022-01-13
LT2984166T (lt) 2020-08-10
JP2020202863A (ja) 2020-12-24
HUE051373T2 (hu) 2021-03-01
CN105026554A (zh) 2015-11-04
EP2984166A4 (en) 2016-09-07
AU2014235096B2 (en) 2020-03-19
DK2984166T3 (da) 2020-07-20

Similar Documents

Publication Publication Date Title
JP7553527B2 (ja) Ｍｐｓ１を治療するための組成物および方法
CN111902539B (zh) 杂合调控元件
KR20200052333A (ko) 아데노-관련 바이러스 변이체 캡시드 및 그 용도
JP2019533992A (ja) 酸性αグルコシダーゼ変異体及びその使用
JP2022523766A (ja) Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
CN111718947B (zh) 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途
JP2021532810A (ja) 糖原病ｉｉｉの処置のためのミニｇｄｅ
Lin et al. Adeno-associated virus-mediated transfer of human acid maltase gene results in a transient reduction of glycogen accumulation in muscle of Japanese quail with acid maltase deficiency
KR20230003569A (ko) Cdkl5 결핍 장애 (cdd)의 치료에 유용한 조성물
TW202045728A (zh) 用於治療克拉培氏病之組成物
HK40042501A (en) Compositions for treating mpsi
HK40042501B (en) Compositions for treating mpsi
US20230190966A1 (en) Compositions useful for treating gm1 gangliosidosis
HK1221735B (en) Compositions for treating mpsi
WO2024213660A1 (en) Inflammation-inducible promoters
JP2023526311A (ja) クラッベ病の治療に有用な組成物

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20170308

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20170308

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20180115

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20180131

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20180424

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20180611

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180727

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20181220

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190419

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20190611

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20190627

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20190821

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20190919

R150 Certificate of patent or registration of utility model

Ref document number: 6591956

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250