JP2021512601A5 - - Google Patents

Info

Publication number
JP2021512601A5
JP2021512601A5 JP2020541862A JP2020541862A JP2021512601A5 JP 2021512601 A5 JP2021512601 A5 JP 2021512601A5 JP 2020541862 A JP2020541862 A JP 2020541862A JP 2020541862 A JP2020541862 A JP 2020541862A JP 2021512601 A5 JP2021512601 A5 JP 2021512601A5
Authority
JP
Japan
Prior art keywords
aav
seq
sequence
capsid protein
pharmaceutical composition
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
JP2020541862A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021512601A (ja
JP7066861B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/016351 external-priority patent/WO2019152841A1/en
Publication of JP2021512601A publication Critical patent/JP2021512601A/ja
Publication of JP2021512601A5 publication Critical patent/JP2021512601A5/ja
Priority to JP2022073374A priority Critical patent/JP7433360B2/ja
Application granted granted Critical
Publication of JP7066861B2 publication Critical patent/JP7066861B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020541862A 2018-02-01 2019-02-01 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法 Expired - Fee Related JP7066861B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022073374A JP7433360B2 (ja) 2018-02-01 2022-04-27 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862625150P 2018-02-01 2018-02-01
US62/625,150 2018-02-01
PCT/US2019/016351 WO2019152841A1 (en) 2018-02-01 2019-02-01 Adeno-associated virus compositions for pah gene transfer and methods of use thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022073374A Division JP7433360B2 (ja) 2018-02-01 2022-04-27 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法

Publications (3)

Publication Number Publication Date
JP2021512601A JP2021512601A (ja) 2021-05-20
JP2021512601A5 true JP2021512601A5 (enExample) 2022-02-09
JP7066861B2 JP7066861B2 (ja) 2022-05-13

Family

ID=67478560

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020541862A Expired - Fee Related JP7066861B2 (ja) 2018-02-01 2019-02-01 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法
JP2022073374A Active JP7433360B2 (ja) 2018-02-01 2022-04-27 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022073374A Active JP7433360B2 (ja) 2018-02-01 2022-04-27 Pah遺伝子移入のためのアデノ随伴ウイルス組成物及びそれらの使用方法

Country Status (15)

Country Link
US (3) US10610606B2 (enExample)
EP (1) EP3746113A4 (enExample)
JP (2) JP7066861B2 (enExample)
KR (2) KR20230065367A (enExample)
CN (1) CN111886021A (enExample)
AU (1) AU2019215150A1 (enExample)
BR (1) BR112020015511A2 (enExample)
CA (1) CA3089824A1 (enExample)
CL (1) CL2020001992A1 (enExample)
CO (1) CO2020010783A2 (enExample)
EA (1) EA202091844A1 (enExample)
IL (2) IL295830A (enExample)
MX (1) MX2020008085A (enExample)
PE (1) PE20210341A1 (enExample)
WO (1) WO2019152841A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6994018B2 (ja) 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質
WO2019152841A1 (en) * 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof
KR20210008501A (ko) 2018-05-09 2021-01-22 바이오마린 파머수티컬 인크. 페닐케톤뇨증을 치료하는 방법
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
PE20220233A1 (es) * 2019-06-10 2022-02-07 Homology Medicines Inc Composiciones de virus adenoasociados para la transferencia del gen de arsa y metodos de uso de las mismas
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
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
CN115461066B (zh) * 2020-04-28 2026-01-06 美国辉瑞有限公司 用于基因疗法的编码天冬氨酸酰化酶(aspa)的经修饰的核酸和载体
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
EP4211252A1 (en) * 2020-09-09 2023-07-19 Homology Medicines, Inc. Vectorized antibodies and uses thereof
KR20230079172A (ko) 2020-10-01 2023-06-05 젠자임 코포레이션 간-유도 유전자 대체 요법에 의한 pku의 치료를 위한 인간 pah 발현 카세트
CN115197967B (zh) * 2021-04-08 2023-09-15 广州派真生物技术有限公司 制备重组腺相关病毒的辅助质粒及其应用
US20230055381A1 (en) * 2021-06-25 2023-02-23 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
US20230090654A1 (en) * 2021-08-24 2023-03-23 Homology Medicines, Inc. Adeno-associated virus formulations
WO2023077085A2 (en) * 2021-10-29 2023-05-04 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
CN116656740A (zh) * 2023-05-30 2023-08-29 浙江大学 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用
CN119876192B (zh) * 2024-07-02 2025-11-07 浙江大学滨江研究院 用于治疗苯丙酮尿症的多核苷酸

Family Cites Families (165)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL71505A0 (en) 1983-04-14 1984-07-31 Hughes Howard Med Inst Phenylalanine hydroxylase cdna clones,their production and their use in the diagnosis of classical phenylketonuria
IL74761A0 (en) 1984-04-13 1985-06-30 Hughes Howard Med Inst Human phenylalanine hydroxylase cdna clones,their production and their use in diagnosing classical phenylketonuria
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
AU7906691A (en) 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE4339922C1 (de) 1993-09-03 1994-10-06 Max Planck Gesellschaft Vektor für Leber-Gentherapie
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
JPH10501686A (ja) 1994-04-13 1998-02-17 ザ ロックフェラー ユニヴァーシティ 神経系の細胞へのdnaのaav仲介送達
WO1996008560A1 (en) 1994-09-16 1996-03-21 The Uab Research Foundation Transduction of genes into human hematopoietic stem cells using recombinant adeno-associated viral vectors
CN1172435A (zh) 1994-09-23 1998-02-04 综合医院公司 使用非哺乳动物dna病毒在哺乳动物细胞中表达外源基因
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5650309A (en) 1995-05-16 1997-07-22 The Regents Of The University Of California Viral vectors
US7001764B2 (en) 1995-06-27 2006-02-21 Cell Genesys, Inc. Compositions comprising tissue specific adenoviral vectors
DE19525900C1 (de) 1995-07-15 1996-12-12 Max Planck Gesellschaft Leberspezifischer Adenovirus-Expressionsvektor
US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US5780447A (en) 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
AU4266597A (en) 1996-09-11 1998-04-14 General Hospital Corporation, The Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
DE19644500C2 (de) 1996-10-25 1998-11-05 Deutsches Krebsforsch AAV-DNA mit Helfervirus-Sequenzen
FR2755699B1 (fr) 1996-11-08 1998-12-18 Rhone Poulenc Rorer Sa Nouvelles constructions et vecteurs pour l'expression ciblee et inductible des genes
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
AU5603998A (en) 1996-12-18 1998-07-15 Targeted Genetics Corporation Recombinase-activatable AAV packaging cassettes for use in the production of AV vectors
WO1998028417A1 (en) 1996-12-23 1998-07-02 Gene-Cell Nucleic acid constructs and uses thereof for direct nucleic acid incorporation into cells
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
WO1998048005A1 (en) 1997-04-24 1998-10-29 University Of Washington Targeted gene modification by parvoviral vectors
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
GB9715064D0 (en) 1997-07-17 1997-09-24 Ppl Therapeutics Scotland Ltd Protein expression
WO1999018227A1 (en) 1997-10-08 1999-04-15 Advanced Research And Technology Institute Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
WO1999064569A1 (en) 1998-06-10 1999-12-16 Advanced Research And Technology Institute Methods and compositions for generating recombinant adeno-associated virus vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6919209B1 (en) 1998-12-04 2005-07-19 City Of Hope Method of genetically modifying very primitive quiescent human hematopoietic stem cells
US6200758B1 (en) 1999-02-19 2001-03-13 New York State Office Of Mental Health Phenylalanine hydroxylase gene variants, and amino acid and pterin homeostasis, in the definition, detection, treatment and prevention of psychotic, mood and personality disorders
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6610906B1 (en) 1999-06-09 2003-08-26 The Regents Of The University Of Michigan Nucleotide sequences for gene regulation and methods of use thereof
WO2001036620A2 (en) 1999-11-16 2001-05-25 Genzyme Corporation Vectors and transgenies with regulatory elements for gene delivery to the liver
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたaav5キャプシドおよびaav5ベクターを含む組換えaavベクター
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
WO2002066611A2 (en) 2001-02-16 2002-08-29 The Board Of Trustees Of The Leland Stanford Junior University Minimal plasmid vectors that provide for persistent and high level gene expression and methods for using the same
DE10120265A1 (de) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
WO2002092134A1 (en) 2001-05-14 2002-11-21 Cell Genesys, Inc. Lentiviral vectors encoding clotting factors for gene therapy
US8067156B2 (en) 2001-05-31 2011-11-29 The Rockefeller University Method for generating replication defective viral vectors that are helper free
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
JP2005526001A (ja) 2001-10-04 2005-09-02 アグイラルーコルドバ,カリオス,エスツアルド 遺伝子療法用キメラ型ウイルスベクター
CA2361462A1 (en) 2001-11-07 2003-05-08 Katherine A. High Induction of tolerance to a therapeutic polypeptide
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CA2369985A1 (en) 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
US20030198620A1 (en) * 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
US7235393B2 (en) 2002-04-29 2007-06-26 The Trustees Of The University Of Pennsylvania Method for direct rescue and amplification of integrated viruses from cellular DNA of tissues
WO2003093436A2 (en) * 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
US20070202587A1 (en) 2002-09-23 2007-08-30 Applied Genetic Technologies Corporation Recombinant AAV production in mammalian cells
US7091029B2 (en) 2002-09-23 2006-08-15 Applied Genetics Technologies Corporation High titer recombinant AAV production
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US7482156B2 (en) 2003-10-15 2009-01-27 Cell Genesys, Inc. Hepatocellular carcinoma specific promoter and uses thereof
EP1732383A4 (en) 2004-04-06 2007-05-02 Cedars Sinai Medical Center PREVENTION AND TREATMENT OF VASCULAR DISEASES WITH RECOMBINANT ADENO ASSOCIATED VIRUS VECTORS THAT CODE APOLIPOPROTEIN A-I AND APOLIPOPROTEIN A-I MILANO
WO2006065273A2 (en) 2004-05-24 2006-06-22 The Regents Of The University Of California New human parvovirus
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
TWI293307B (en) 2004-09-30 2008-02-11 Ind Tech Res Inst A liver-specific chimeric regulatory sequence and use thereof
US8658778B2 (en) 2005-03-09 2014-02-25 Board Of Regents, The University Of Texas System hTMC promoter and vectors for the tumor-selective and high-efficient expression of cancer therapeutic genes
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
WO2007019646A1 (en) 2005-08-19 2007-02-22 University Of Technology, Sydney Liver-directed gene therapy
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
WO2008016391A2 (en) 2006-01-31 2008-02-07 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
DK2623605T3 (en) 2007-04-09 2019-03-25 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A method for transducing cells with primary cilia
EP2008656A1 (en) 2007-06-28 2008-12-31 Bergen Teknologioverforing AS Compositions for the treatment of hyperphenylalaninemia
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009108274A2 (en) 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
US9617548B2 (en) 2008-04-22 2017-04-11 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US20100316623A1 (en) * 2009-04-23 2010-12-16 Andrew Turner Phenylalanine hydroxylase fusion protein and methods for treating phenylketonuria
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
SI3988660T1 (sl) 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
WO2010136549A2 (en) 2009-05-28 2010-12-02 Deutsches Krebsforschungszentrum Modified aav capsid polypeptides
JP5956331B2 (ja) 2009-06-16 2016-07-27 ジェンザイム・コーポレーション 組換えaavベクターの改良された精製方法
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2529020B1 (en) 2010-01-28 2018-04-25 The Children's Hospital of Philadelphia A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US9839696B2 (en) * 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8927514B2 (en) * 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
US20120014932A1 (en) 2010-07-14 2012-01-19 Golden William J Methods for treating disorders of amino acid metabolism
CA2826273C (en) 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
CN103502458B (zh) * 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP2681327B1 (en) 2011-03-04 2018-11-21 Intrexon Corporation Vectors conditionally expressing protein
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US9222105B2 (en) 2011-10-27 2015-12-29 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
EP2782596A4 (en) 2011-11-22 2015-07-29 Philadelphia Children Hospital VIRUS VECTORS FOR HIGHLY EFFICIENT TRANSGENIC ADMINISTRATION
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia Aavカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
US9604226B2 (en) * 2012-05-01 2017-03-28 Innovative Combustion Technologies, Inc. Pulverizer mill protection system
TWI698240B (zh) * 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
HK1210214A1 (en) 2012-07-06 2016-04-15 University Of Iowa Research Foundation Modified adeno-associated virus vector compositions
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
DK2911687T3 (da) 2012-10-26 2019-05-13 Univ Brussel Vrije Vektor til levermålrettet genterapi af hæmofili og fremgangsmåder og anvendelse deraf
CA2892448A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
WO2014109728A1 (en) 2013-01-11 2014-07-17 The Scripps Research Institute Methods and compositions for enchancing transduction efficiency of retroviral vectors
WO2014124282A1 (en) 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
US20140271550A1 (en) 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
JP2016517278A (ja) 2013-03-15 2016-06-16 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia スタッファー/フィラーポリヌクレオチド配列を含むベクターおよびその使用方法
EP2970966A4 (en) * 2013-03-15 2016-10-26 Univ North Carolina SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES
BR112015021036A2 (pt) 2013-03-15 2017-10-10 Univ Pennsylvania composições e métodos para tratar mps1
US9700486B2 (en) * 2013-04-24 2017-07-11 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
KR102298579B1 (ko) 2013-05-21 2021-09-07 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2015006743A1 (en) 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
NZ716102A (en) 2013-07-22 2021-12-24 The Children’S Hospital Of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
EP3058072B1 (en) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
US10612041B2 (en) 2014-03-21 2020-04-07 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
US10781459B2 (en) * 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
WO2016049230A1 (en) * 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
CN107208105A (zh) 2014-12-17 2017-09-26 基础应用医学研究基金会 用于治疗威尔森病的核酸构建体和基因治疗载体
US11118238B2 (en) 2014-12-17 2021-09-14 Fundación Para La Investigación Mèdica Aplicada Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions
KR102554850B1 (ko) 2015-02-06 2023-07-13 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 인간 응고 인자 viii 유전자 발현 카세트 및 그의 용도
JP6879486B2 (ja) 2015-03-17 2021-06-02 フリーイェ・ユニヴェルシテイト・ブリュッセルVrije Universieit Brussel Fviiiおよびfix用の最適化された肝臓特異的発現系
CN115094062A (zh) 2015-04-16 2022-09-23 埃默里大学 用于肝脏中蛋白质表达的重组启动子和载体及其用途
WO2017015154A1 (en) 2015-07-17 2017-01-26 Children's National Medical Center Methods and compositions for treating phenyltonuria
US10143760B2 (en) 2015-10-28 2018-12-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
WO2017100551A1 (en) 2015-12-09 2017-06-15 Alexion Pharmaceuticals, Inc. HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION
WO2018046737A1 (en) 2016-09-09 2018-03-15 Fondazione Telethon Combined therapy for mucopolysaccharidosis type vi (maroteaux-lamy-syndrome)
EP3562494A4 (en) * 2016-12-30 2020-08-19 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF PHENYLKETONURIS
KR102863181B1 (ko) 2016-12-30 2025-09-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 윌슨병을 치료하기 위한 유전자 치료
US11938197B2 (en) 2017-01-10 2024-03-26 The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) Polynucleotides and vectors for the expression of transgenes
WO2019010091A1 (en) 2017-07-06 2019-01-10 The Board Of Trustees Of The Leland Stanford Junior University METHODS AND COMPOSITIONS FOR FACILITATING HOMOLOGOUS RECOMBINATION
WO2019152841A1 (en) * 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for pah gene transfer and methods of use thereof

Similar Documents

Publication Publication Date Title
IL276215B2 (en) Adenovirus-linked compounds for phenylalanine hydroxylase (pah) gene transfer and methods of use thereof
US12534717B2 (en) AAV capsid proteins for nucleic acid transfer
ES3050313T3 (en) Liver-specific tropism of adeno-associated viruses
JP2020522269A5 (enExample)
US8906387B2 (en) In vivo transduction with a chimeric AAV capsid protein
US11938197B2 (en) Polynucleotides and vectors for the expression of transgenes
US7427396B2 (en) AAV vectors for gene delivery to the lung
HRP20241180T1 (hr) Modificirani faktor ix, te pripravci, postupci i upotreba za prijenos gena u stanice, organe i tkiva
JP2012521750A5 (enExample)
JP2022525302A5 (enExample)
RU2018116076A (ru) Модифицированные гены атаксии фридрейха и векторы для генной терапии
JP2018522529A5 (enExample)
HRP20250897T1 (hr) Genska terapija za neurodegenerativne poremećaje
RU2017125234A (ru) Конструкции нуклеиновой кислоты и векторы для генотерапии для применения для лечения болезни вильсона и других состояний
JP2019528793A5 (enExample)
JP2022524434A5 (enExample)
JP2017529395A5 (enExample)
JP2023528371A5 (enExample)
CN118103393A (zh) 工程化的itr序列及其使用方法
JP2023520763A5 (enExample)
EP4135661A1 (en) Compositions and methods for treatment of neurological disorders
TW202328451A (zh) 工程化itr序列及使用之方法
RU2018117179A (ru) Печень-специфичные конструкции, экспрессионные кассеты фактора viii и способы их применения
HK40044351A (en) Liver-specific tropism of adeno-associated viruses
HK40044351B (en) Liver-specific tropism of adeno-associated viruses