CL2018000740A1 - Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden - Google Patents

Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden

Info

Publication number
CL2018000740A1
CL2018000740A1 CL2018000740A CL2018000740A CL2018000740A1 CL 2018000740 A1 CL2018000740 A1 CL 2018000740A1 CL 2018000740 A CL2018000740 A CL 2018000740A CL 2018000740 A CL2018000740 A CL 2018000740A CL 2018000740 A1 CL2018000740 A1 CL 2018000740A1
Authority
CL
Chile
Prior art keywords
vectors
fviii
adeno
vaa
factor viii
Prior art date
Application number
CL2018000740A
Other languages
English (en)
Spanish (es)
Inventor
Stuart Bunting
Peter Cameron Colosi
Erno Pungor
Original Assignee
Biomarin Pharm Inc
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 Biomarin Pharm Inc filed Critical Biomarin Pharm Inc
Publication of CL2018000740A1 publication Critical patent/CL2018000740A1/es

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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6854—Immunoglobulins
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/16044—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/005—Assays involving biological materials from specific organisms or of a specific nature from viruses
    • G01N2333/01—DNA viruses
    • G01N2333/015—Parvoviridae, e.g. feline panleukopenia virus, human Parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Wood Science & Technology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • General Engineering & Computer Science (AREA)
  • Inorganic Chemistry (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
CL2018000740A 2015-09-24 2018-03-21 Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden CL2018000740A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562232242P 2015-09-24 2015-09-24
US201662323182P 2016-04-15 2016-04-15
US201662365544P 2016-07-22 2016-07-22

Publications (1)

Publication Number Publication Date
CL2018000740A1 true CL2018000740A1 (es) 2018-06-08

Family

ID=57113749

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2018000740A CL2018000740A1 (es) 2015-09-24 2018-03-21 Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden

Country Status (18)

Country Link
US (3) US10512675B2 (cg-RX-API-DMAC7.html)
EP (1) EP3352787A1 (cg-RX-API-DMAC7.html)
JP (3) JP2018535929A (cg-RX-API-DMAC7.html)
KR (1) KR102805910B1 (cg-RX-API-DMAC7.html)
CN (1) CN108778323A (cg-RX-API-DMAC7.html)
AU (2) AU2016326602A1 (cg-RX-API-DMAC7.html)
BR (1) BR112018006074A2 (cg-RX-API-DMAC7.html)
CA (1) CA2999297A1 (cg-RX-API-DMAC7.html)
CL (1) CL2018000740A1 (cg-RX-API-DMAC7.html)
IL (2) IL297353B1 (cg-RX-API-DMAC7.html)
MA (1) MA42934A (cg-RX-API-DMAC7.html)
MX (1) MX2018003702A (cg-RX-API-DMAC7.html)
PE (2) PE20241346A1 (cg-RX-API-DMAC7.html)
PH (1) PH12018500594A1 (cg-RX-API-DMAC7.html)
RU (2) RU2742352C2 (cg-RX-API-DMAC7.html)
TW (3) TWI756185B (cg-RX-API-DMAC7.html)
WO (1) WO2017053677A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201801657B (cg-RX-API-DMAC7.html)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
EP3352787A1 (en) * 2015-09-24 2018-08-01 BioMarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
KR20230161535A (ko) 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
AU2018299865A1 (en) * 2017-07-10 2020-01-30 Uniqure Ip B.V. Means and methods for AAV gene therapy in humans
AU2018312565A1 (en) * 2017-08-01 2020-02-27 Spark Therapeutics, Inc. Factor VIII (FVIII) gene therapy methods
EP3749756A4 (en) * 2018-02-05 2021-12-08 Audentes Therapeutics, Inc. ELEMENTS OF TRANSCRIPTION REGULATION AND ASSOCIATED USES
WO2019210187A1 (en) * 2018-04-26 2019-10-31 The University Of North Carolina At Chapel Hill Methods and compositions for treatment of hemophilia
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
AR117427A1 (es) * 2018-05-14 2021-08-04 Biomarin Pharm Inc Expresión estable de vectores de aav en sujetos jóvenes
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
CA3106590A1 (en) * 2018-07-16 2020-01-23 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
MX2021001375A (es) * 2018-08-03 2021-04-19 Sangamo Therapeutics Inc Parametros clinicos mejorados por la expresion del factor viii.
EP3840775A4 (en) * 2018-08-24 2022-07-06 Spark Therapeutics, Inc. OPTIMIZED PROMOTER SEQUENCES, INTRON-FREE EXPRESSION CONSTRUCTS AND METHODS OF USE
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3129532A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
SG11202109850SA (en) 2019-03-13 2021-10-28 Generation Bio Co Non-viral dna vectors and uses thereof for expressing fviii therapeutics
WO2020247814A1 (en) * 2019-06-05 2020-12-10 Orchard Therapeutics (Europe) Limited Compositions and methods for modifying eukaryotic cells
JP2022537555A (ja) * 2019-06-20 2022-08-26 武田薬品工業株式会社 ウイルスベースの遺伝子療法による治療方法
WO2021084277A2 (en) 2019-11-01 2021-05-06 Freeline Therapeutics Limited Transcription regulatory elements
AU2020395323A1 (en) * 2019-12-06 2022-06-30 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor VIII function
EP4073106A2 (en) * 2019-12-12 2022-10-19 Takeda Pharmaceutical Company Limited Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
KR102167829B1 (ko) * 2020-02-10 2020-10-20 주식회사 이노테라피 아데노연관바이러스용 안정화제 및 이를 이용한 아데노연관바이러스의 안정화 방법
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
MX2023005041A (es) 2020-11-02 2023-05-17 Biomarin Pharm Inc Proceso para enriquecer virus adenoasociado.
CN115554418B (zh) 2022-11-22 2023-04-14 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途
CN119015447A (zh) * 2023-09-01 2024-11-26 康霖生物科技（杭州）有限公司 一种缓冲系统及其相应液体制剂及其用途

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
EP0127839B1 (en) 1983-05-27 1992-07-15 THE TEXAS A&M UNIVERSITY SYSTEM Method for producing a recombinant baculovirus expression vector
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
CA2329143A1 (en) 1998-05-27 1999-12-02 Cell Genesys, Inc. Adeno-associated viral vector-mediated expression of factor viii activity
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
ES2381796T3 (es) 2004-09-22 2012-05-31 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
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
CN104152489A (zh) 2007-11-01 2014-11-19 罗切斯特大学 具有增加的稳定性的重组因子viii
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
GB0911870D0 (en) * 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
KR101337797B1 (ko) * 2010-07-14 2013-12-06 한미사이언스 주식회사 지속형 인간 성장 호르몬 결합체 액상 제제
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
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
US20140003880A1 (en) * 2012-06-30 2014-01-02 General Electric Company Ceramic matrix composite and metal attachment configurations
WO2014064277A1 (en) * 2012-10-26 2014-05-01 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
RS61039B1 (sr) * 2013-09-12 2020-12-31 Biomarin Pharm Inc Aav vektori koji sadrže gen koji kodira faktor viii
KR102863734B1 (ko) 2015-02-10 2025-09-25 젠자임 코포레이션 선조체 및 피질로의 바이러스 입자의 향상된 전달
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
EP3352787A1 (en) * 2015-09-24 2018-08-01 BioMarin Pharmaceutical Inc. Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same

Also Published As

Publication number Publication date
CA2999297A1 (en) 2017-03-30
JP2024147801A (ja) 2024-10-16
IL257939A (en) 2018-05-31
IL257939B (en) 2022-11-01
RU2742352C2 (ru) 2021-02-05
KR102805910B1 (ko) 2025-05-13
EP3352787A1 (en) 2018-08-01
US20170087219A1 (en) 2017-03-30
PH12018500594A1 (en) 2018-09-24
JP2022010141A (ja) 2022-01-14
MA42934A (fr) 2018-08-01
PE20241346A1 (es) 2024-07-03
KR20180053335A (ko) 2018-05-21
AU2021261832B2 (en) 2025-01-30
TW202446416A (zh) 2024-12-01
TWI756185B (zh) 2022-03-01
BR112018006074A2 (pt) 2018-10-09
TW202220690A (zh) 2022-06-01
RU2020126034A (ru) 2020-09-02
AU2016326602A1 (en) 2018-03-29
IL297353A (en) 2022-12-01
RU2018114888A3 (cg-RX-API-DMAC7.html) 2019-10-24
US11690898B2 (en) 2023-07-04
JP2018535929A (ja) 2018-12-06
IL257939B2 (en) 2023-03-01
IL297353B1 (en) 2025-10-01
NZ780570A (en) 2024-08-30
NZ740521A (en) 2023-11-24
WO2017053677A1 (en) 2017-03-30
RU2018114888A (ru) 2019-10-24
US20240058423A1 (en) 2024-02-22
CN108778323A (zh) 2018-11-09
PE20181338A1 (es) 2018-08-21
TW201717999A (zh) 2017-06-01
AU2021261832A1 (en) 2021-12-02
ZA201801657B (en) 2021-07-28
TWI841908B (zh) 2024-05-11
US20200061161A1 (en) 2020-02-27
MX2018003702A (es) 2018-04-30
US10512675B2 (en) 2019-12-24

Similar Documents

Publication Publication Date Title
CL2018000740A1 (es) Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden
CL2023003001A1 (es) Novedosas proteínas de la cápside del virus adenoasociado
CL2023000264A1 (es) Nuevos vectores virales adeno-asociados dirigidos al hígado
CL2018001522A1 (es) Focalización de péptidos para dirigir virus adenoasociados (aavs)
PE20190563A1 (es) Genes de mini-distrofina optimizados y casetes de expresion y su uso
CL2020000422A1 (es) Composiciones de glp-1 y sus usos.
CL2024001716A1 (es) Anticuerpo aislado que se une a la proteína e del virus del dengue.
CL2017003096A1 (es) Vectores virales adenoasociados para el tratamiento de mucopolisacaridosis
EA201792500A1 (ru) Aav-опосредованная экспрессия антител против гриппа и способы их использования
MX2015007755A (es) Vacunas contra virus de la influenza y sus usos.
CR20170139A (es) Proteínas de fusión, bacterias recombinantes y métodos de uso de las bacterias recombinantes
EA201891415A1 (ru) Антигены вируса иммунодефицита человека, векторы, композиции и способы их применения
UY36458A (es) Anticuerpos humanos para hemaglutinina influenza.
CL2017001089A1 (es) Vacunas terapéuticas contra el vph16
CL2021000938A1 (es) Composiciones estables de semaglutida y usos de las mismas
CL2017003341A1 (es) Vectores virales recombinantes que contienen la proteína menor del virus del síndrome reproductor y respiratorio porcino (prrsv) y sus métodos de elaboración y uso
CL2017003144A1 (es) Péptidos lipidados resistentes a proteasas
CO2020013557A2 (es) Variantes de lfa3 y composiciones y usos de las mismas
MX2017003949A (es) Derivados peptidicos novedosos y sus usos.
EA201790771A1 (ru) Фармацевтические композиции длительного действия для лечения гепатита c
CL2015003242A1 (es) Virus aav/upr-plus, proteína de fusión upr-plus, método de tratamiento genético y su uso en el tratamiento enfermedades neurodegenerativas, tales como las enfermedades de parkinson y huntington, entre otras.
EA202191862A2 (ru) Антигены вируса иммунодефицита человека, векторы, композиции и способы их применения
AR106131A1 (es) Vectores del factor viii del virus adenoasociado, partículas víricas asociadas y formulaciones terapéuticas que las comprenden, método