[go: up one dir, main page]

SG10202009849WA - Gene therapy for treating hemophilia a - Google Patents

Gene therapy for treating hemophilia a

Info

Publication number
SG10202009849WA
SG10202009849WA SG10202009849WA SG10202009849WA SG10202009849WA SG 10202009849W A SG10202009849W A SG 10202009849WA SG 10202009849W A SG10202009849W A SG 10202009849WA SG 10202009849W A SG10202009849W A SG 10202009849WA SG 10202009849W A SG10202009849W A SG 10202009849WA
Authority
SG
Singapore
Prior art keywords
gene therapy
treating hemophilia
hemophilia
treating
therapy
Prior art date
Application number
SG10202009849WA
Inventor
Lili Wang
James M Wilson
Jenny Agnes Sidrane
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10202009849WA publication Critical patent/SG10202009849WA/en

Links

Classifications

    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/36—Blood coagulation or fibrinolysis factors
    • A61K38/37—Factors VIII
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

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)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dispersion Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
SG10202009849WA 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a SG10202009849WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662323336P 2016-04-15 2016-04-15
US201662331807P 2016-05-04 2016-05-04
US201662428866P 2016-12-01 2016-12-01

Publications (1)

Publication Number Publication Date
SG10202009849WA true SG10202009849WA (en) 2020-11-27

Family

ID=59253992

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201808422QA SG11201808422QA (en) 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a
SG10202009849WA SG10202009849WA (en) 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG11201808422QA SG11201808422QA (en) 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a

Country Status (17)

Country Link
US (3) US11779656B2 (en)
EP (1) EP3452102B9 (en)
JP (2) JP7347933B2 (en)
KR (1) KR102450833B1 (en)
CN (1) CN109562191A (en)
AU (1) AU2017248659B2 (en)
BR (1) BR112018071200A2 (en)
CA (1) CA3019425A1 (en)
IL (1) IL262215B2 (en)
MX (1) MX2018012537A (en)
PE (2) PE20250736A1 (en)
RU (1) RU2762257C2 (en)
SA (1) SA518400233B1 (en)
SG (2) SG11201808422QA (en)
TW (1) TWI791433B (en)
WO (1) WO2017180857A1 (en)
ZA (1) ZA201806477B (en)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2737094C (en) 2008-09-15 2018-02-20 Paolo Simioni Factor ix polypeptide mutant, its uses and a method for its production
CN109562191A (en) * 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 For treating the gene therapy of hemophilia A
US11191847B2 (en) 2016-04-15 2021-12-07 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia B
AU2019216257A1 (en) 2018-01-31 2020-07-23 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
JOP20210054A1 (en) * 2018-09-21 2020-03-21 Nightstarx Ltd Compositions and methods for treating retinitis pigmentosa
CA3116283A1 (en) * 2018-10-12 2020-04-16 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
US20210324483A1 (en) * 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
GB201818816D0 (en) * 2018-11-19 2019-01-02 Synpromics Ltd Regulatory nucleic acid sequences
TWI851647B (en) * 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
KR20210133242A (en) * 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Adeno-associated viral vector delivery of B-sarcoglycans and treatment of muscular dystrophy
BR112021017853A2 (en) * 2019-03-13 2021-11-30 Generation Bio Co Non-viral dna vectors and uses thereof to express fviii therapeutics
AU2020331987A1 (en) 2019-08-21 2022-03-31 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
CN114829389A (en) * 2019-11-01 2022-07-29 自由行疗法有限公司 Transcriptional regulatory elements
CN111218446B (en) * 2019-12-25 2023-03-28 劲帆生物医药科技（武汉）有限公司 Liver specificity promoter and application thereof
PE20231100A1 (en) * 2020-01-16 2023-07-18 Takeda Pharmaceuticals Co GENE THERAPY BASED ON ADENO ASSOCIATED VIRUSES FOR PHENYLKETONURIA
KR20230010670A (en) 2020-05-12 2023-01-19 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions for DRG-specific reduction of transgene expression
WO2022165246A1 (en) * 2021-01-29 2022-08-04 The Trustees Of The University Of Pennsylvania Compositions and method of use of mutant ace2 decoy variants
MX2023008826A (en) 2021-02-01 2023-09-15 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses.
CA3205351A1 (en) * 2021-02-01 2022-08-04 James M. Wilson Compositions and methods for treatment of niemann pick type a disease
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023205300A2 (en) * 2022-04-20 2023-10-26 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii
WO2023240222A2 (en) * 2022-06-10 2023-12-14 University Of Florida Research Foundation, Incorporated Immune tolerance induction to viral capsids

Family Cites Families (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
US5439824A (en) 1993-05-06 1995-08-08 The United States Of America Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II
US6268212B1 (en) 1993-10-18 2001-07-31 Amgen Inc. Tissue specific transgene expression
US6818439B1 (en) 1994-12-30 2004-11-16 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders
WO1998000541A2 (en) * 1996-07-03 1998-01-08 Chiron Corporation Methods for administration of recombinant gene delivery vehicles for treatment of human disease
CA2312291A1 (en) 1997-12-05 1999-06-17 The Immune Response Corporation Novel vectors and genes exhibiting increased expression
US6783961B1 (en) 1999-02-26 2004-08-31 Genset S.A. Expressed sequence tags and encoded human proteins
US6797505B2 (en) 1998-05-27 2004-09-28 Cell Genesys, Inc. Recombinant AAV vectors for gene therapy of hemophilia A
US6221349B1 (en) * 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
EP1038959A1 (en) 1999-03-17 2000-09-27 Aventis Behring Gesellschaft mit beschränkter Haftung Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX
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
AU7879000A (en) 1999-10-12 2001-04-23 University Of North Carolina At Chapel Hill, The Adeno-associated virus vectors encoding factor viii and methods of using the same
AU1920201A (en) 1999-11-16 2001-05-30 Genzyme Corporation Improved regulatory elements for delivery to the liver
IL151857A0 (en) 2000-03-22 2003-04-10 Octagene Gmbh Production of recombinant blood clotting factors in human cell lines
US6974667B2 (en) 2000-06-14 2005-12-13 Gene Logic, Inc. Gene expression profiles in liver cancer
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
US6812339B1 (en) 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
US20030147853A1 (en) 2001-03-14 2003-08-07 Mcclelland Alan Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions
US8021875B2 (en) 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
WO2003031598A2 (en) 2001-10-05 2003-04-17 Emory University Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use
HU230406B1 (en) 2001-11-13 2016-04-28 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
AU2002360291A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences
WO2005012877A2 (en) 2003-08-01 2005-02-10 Dna Twopointo Inc. Systems and methods for antibody engineering
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
HUE054805T2 (en) 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
ES2319198T3 (en) 2003-10-16 2009-05-05 Claude Negrier MODIFIED cDNA FOR HIGH EXPRESSION LEVELS OF FACTOR VIII AND ITS DERIVATIVES.
DE602004026474D1 (en) 2003-12-03 2010-05-20 Univ Rochester RECOMBINANT FACTOR VIII WITH INCREASED SPECIFIC ACTIVITY
ATE549037T1 (en) 2004-09-22 2012-03-15 St Jude Childrens Res Hospital IMPROVED EXPRESSION OF FACTOR-IX IN GENE THERAPY VECTORS
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
WO2006108590A1 (en) 2005-04-14 2006-10-19 Csl Behring Gmbh Modified coagulation factor viii with enhanced stability and its derivates
WO2007016331A2 (en) 2005-07-29 2007-02-08 Children's Hospital Medical Center Classification and diagnosis of the molecular basis of cholestasis
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
WO2007078599A2 (en) 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
EP1979485A2 (en) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Self-complementary parvoviral vectors, and methods for making and using the same
WO2007120533A2 (en) * 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Minigene expression cassette
CN104087591A (en) 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 Modified factor viii and factor ix genes and vectors for gene therapy
WO2008073303A2 (en) 2006-12-07 2008-06-19 Switchgear Genomics Transcriptional regulatory elements of biological pathways, tools, and methods
KR101542752B1 (en) 2006-12-22 2015-08-10 체에스엘 베링 게엠베하 Modified coagulation factors with prolonged in vivo half-life
CA2676090A1 (en) 2007-02-06 2008-10-02 Genizon Biosciences Inc. Genemap of the human genes associated with adhd
KR101190593B1 (en) 2008-02-14 2013-03-08 재단법인 목암생명공학연구소 Expression vector suitable for expression of a coding sequence for gene therapy
KR100981092B1 (en) 2008-02-29 2010-09-08 고려대학교 산학협력단 Recombinant Expression Vector System of Eighth Coagulation Factor and Von Willy Blant Factor Variants
WO2009122401A2 (en) 2008-04-03 2009-10-08 Yeda Research And Development Co. Ltd. Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
CA2728537A1 (en) 2008-06-25 2009-12-30 Source Precision Medicine, Inc. D/B/A Source Mdx Gene expression profiling for identification, monitoring, and treatment of osteoarthritis
KR101034811B1 (en) 2008-08-25 2011-05-16 단국대학교 산학협력단 Recombinant adenoviruses and their use, including tissue-specific promoters and trans-splicing ribozymes targeting cancer specific genes
CN102448501A (en) 2009-03-27 2012-05-09 西马生物医学计划公司 Methods and compositions for treating cirrhosis and liver fibrosis
CN101935674A (en) * 2009-06-30 2011-01-05 中国医学科学院血液学研究所 A kind of adenoviral vector for hemophilia B gene therapy and application thereof
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2542247A4 (en) 2010-03-01 2014-05-07 Philadelphia Children Hospital NUCLEIC ACIDS FOR TARGETING MULTIPLE HCV GENOME REGIONS
SG10201502270TA (en) 2010-03-29 2015-05-28 Univ Pennsylvania Pharmacologically induced transgene ablation system
US9315825B2 (en) * 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
AU2011298333B2 (en) * 2010-09-02 2015-05-14 Molmed Spa Stable production of lentiviral vectors
FR2977562B1 (en) 2011-07-06 2016-12-23 Gaztransport Et Technigaz SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE
EP3513802B1 (en) * 2012-10-26 2023-11-29 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
CA2888931C (en) * 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
JP6735672B2 (en) * 2013-09-12 2020-08-05 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 Adeno-associated virus factor VIII vector
US10308705B2 (en) * 2015-02-06 2019-06-04 The University Of North Carolina At Chapel Hill Optimized human clotting factor VIII gene expression cassettes and their use
US20180110877A1 (en) * 2015-04-27 2018-04-26 The Trustees Of The University Of Pennsylvania DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
TWI777175B (en) * 2015-11-13 2022-09-11 日商武田藥品工業股份有限公司 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
CN109562191A (en) * 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 For treating the gene therapy of hemophilia A

Also Published As

Publication number Publication date
JP7347933B2 (en) 2023-09-20
IL262215A (en) 2018-11-29
BR112018071200A2 (en) 2019-04-09
PE20181922A1 (en) 2018-12-11
SA518400233B1 (en) 2022-08-23
US11779656B2 (en) 2023-10-10
JP2019513794A (en) 2019-05-30
EP3452102A1 (en) 2019-03-13
RU2762257C2 (en) 2021-12-17
US20190343966A1 (en) 2019-11-14
AU2017248659B2 (en) 2022-08-11
ZA201806477B (en) 2024-09-25
TWI791433B (en) 2023-02-11
IL262215B2 (en) 2023-07-01
US10888628B2 (en) 2021-01-12
US20240000974A1 (en) 2024-01-04
MX2018012537A (en) 2019-02-25
PE20250736A1 (en) 2025-03-10
US20200085975A1 (en) 2020-03-19
RU2018136611A3 (en) 2020-08-11
WO2017180857A1 (en) 2017-10-19
CN109562191A (en) 2019-04-02
KR102450833B1 (en) 2022-10-05
IL262215B1 (en) 2023-03-01
KR20190032274A (en) 2019-03-27
EP3452102B1 (en) 2024-06-12
RU2018136611A (en) 2020-05-15
TW201741458A (en) 2017-12-01
CA3019425A1 (en) 2017-10-19
SG11201808422QA (en) 2018-10-30
JP2022062121A (en) 2022-04-19
AU2017248659A1 (en) 2018-10-18
EP3452102B9 (en) 2024-09-04

Similar Documents

Publication Publication Date Title
SG10202009849WA (en) Gene therapy for treating hemophilia a
IL284741A (en) Gene therapy for retinitis pigmentosa
IL260959A (en) Gene therapy for treating mucopolysaccharidosis type i
IL284577A (en) Gene therapy
IL258580B (en) Combination therapy for treating malignancies
IL262211B1 (en) Gene therapy for treating mucopolysaccharidosis type ii
IL258685B (en) Combination therapy for treating malignancies
IL258269A (en) Combination therapy for treating malignancies
IL259850A (en) Gene therapy for treating familial hypercholesterolemia
IL258684A (en) Combination therapy for treating malignancies
GB201707212D0 (en) Gene therapy for ciliopathies
IL263085A (en) Gene therapy
HK1262143A1 (en) Gene therapy for treating hemophilia a
HK40007440A (en) Gene therapy for treating hemophilia b
GB201616821D0 (en) Gene therapy
GB201612104D0 (en) Gene therapy
GB201612105D0 (en) Gene therapy
AP2016009066A0 (en) A drug for treating cancer