[go: up one dir, main page]

PE20181338A1 - Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden - Google Patents

Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden

Info

Publication number
PE20181338A1
PE20181338A1 PE2018000419A PE2018000419A PE20181338A1 PE 20181338 A1 PE20181338 A1 PE 20181338A1 PE 2018000419 A PE2018000419 A PE 2018000419A PE 2018000419 A PE2018000419 A PE 2018000419A PE 20181338 A1 PE20181338 A1 PE 20181338A1
Authority
PE
Peru
Prior art keywords
vectors
fviii
adeno
factor viii
therapeutic formulations
Prior art date
Application number
PE2018000419A
Other languages
English (en)
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 PE20181338A1 publication Critical patent/PE20181338A1/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)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (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)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)

Abstract

Referido a vectores y virus que codifican/expresan un Factor VIII (FVIII) de virus adenoasociado (VAA): AAV5-FVIII-SQ. Ademas, incluye vectores FVIIIVAA con una actividad de expresion elevada y vectores FVIII VAA que expresan la proteina FVIII funcional de longitud completa o truncada. Tambien se refiere a los metodos de preparacion de dichos vectores, a las particulas viricas FVIII VAA recombinantes que comprenden o expresan dichos vectores y formulaciones farmaceuticas que los comprenden, siendo utiles en el tratamiento de la hemofilia A.
PE2018000419A 2015-09-24 2016-09-23 Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden PE20181338A1 (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
PE20181338A1 true PE20181338A1 (es) 2018-08-21

Family

ID=57113749

Family Applications (2)

Application Number Title Priority Date Filing Date
PE2023002034A PE20241346A1 (es) 2015-09-24 2016-09-23 Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprenden
PE2018000419A PE20181338A1 (es) 2015-09-24 2016-09-23 Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PE2023002034A PE20241346A1 (es) 2015-09-24 2016-09-23 Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones que los comprenden

Country Status (18)

Country Link
US (3) US10512675B2 (es)
EP (1) EP3352787A1 (es)
JP (3) JP2018535929A (es)
KR (1) KR102805910B1 (es)
CN (1) CN108778323A (es)
AU (2) AU2016326602A1 (es)
BR (1) BR112018006074A2 (es)
CA (1) CA2999297A1 (es)
CL (1) CL2018000740A1 (es)
IL (2) IL257939B2 (es)
MA (1) MA42934A (es)
MX (1) MX2018003702A (es)
PE (2) PE20241346A1 (es)
PH (1) PH12018500594A1 (es)
RU (2) RU2742352C2 (es)
TW (3) TWI841908B (es)
WO (1) WO2017053677A1 (es)
ZA (1) ZA201801657B (es)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
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
CN108473976B (zh) 2015-10-28 2022-07-19 桑格摩生物治疗股份有限公司 肝特异性构建体、因子viii表达盒及其使用方法
EP3491008A2 (en) 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
DK3652326T3 (da) * 2017-07-10 2024-10-28 Uniqure Ip Bv Midler og fremgangsmåder til AAV-genterapi i mennesker
JP7499174B2 (ja) * 2017-08-01 2024-06-13 スパーク セラピューティクス インコーポレイテッド 第ｖｉｉｉ因子（ｆｖｉｉｉ）遺伝子治療法
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
EP3794112A1 (en) * 2018-05-14 2021-03-24 BioMarin Pharmaceutical Inc. Stable expression of aav vectors in juvenile subjects
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
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
BR112021000695A2 (pt) * 2018-07-16 2021-04-20 Baxalta Incorporated métodos para tratar hemofilia a e para monitorar a eficácia da terapia gênica de fator viii de hemofilia a.
EP3829650A4 (en) * 2018-08-03 2022-05-04 Sangamo Therapeutics, Inc. IMPROVED CLINICAL PARAMETERS BY EXPRESSION OF FACTOR VIII
BR112021003399A2 (pt) * 2018-08-24 2021-05-18 Spark Therapeutics, Inc. sequências de promotores otimizadas, construtos de expressão livres de íntron e métodos de uso
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
CA3129532A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
MX2021011039A (es) 2019-03-13 2021-12-15 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos con fviii.
CN114450412A (zh) * 2019-06-05 2022-05-06 奥查德疗法(欧洲)有限公司 用于修饰真核细胞的组合物和方法
CN114375205A (zh) * 2019-06-20 2022-04-19 武田药品工业株式会社 用基于病毒的基因疗法进行治疗的方法
CN114901686A (zh) 2019-11-01 2022-08-12 自由行疗法有限公司 因子viii多肽
AU2020395323A1 (en) * 2019-12-06 2022-06-30 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor VIII function
CN114981299A (zh) * 2019-12-12 2022-08-30 武田药品工业株式会社 使用编码具有增加的表达的重组fviii变体的病毒载体的a型血友病的基因疗法
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
IL302128A (en) 2020-11-02 2023-06-01 Biomarin Pharm Inc A process for the enrichment of adeno-associated virus
CN116350801A (zh) 2022-11-22 2023-06-30 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
WO2025045082A1 (zh) * 2023-09-01 2025-03-06 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
ATE78293T1 (de) 1983-05-27 1992-08-15 Texas A & M Univ Sys Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors.
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
WO1998009524A1 (en) 1996-09-06 1998-03-12 Chiron Corporation Methods and compositions for liver specific delivery of therapeutic molecules using recombinant aav vectors
AU4185699A (en) * 1998-05-27 1999-12-13 Avigen, 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
EP1804839B1 (en) 2004-09-22 2012-03-14 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
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
MX2010004813A (es) 2007-11-01 2010-10-04 Univ Rochester Factor viii recombinante que tiene estabilidad incrementada.
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
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
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
JP6454643B2 (ja) * 2012-10-26 2019-01-16 フリーイェ・ユニヴェルシテイト・ブリュッセルＶｒｉｊｅ Ｕｎｉｖｅｒｓｉｅｉｔ Ｂｒｕｓｓｅｌ 血友病の肝臓指向性遺伝子治療のためのベクター並びにその方法及び使用
JP6735672B2 (ja) * 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 アデノ随伴ウイルス第ｖｉｉｉ因子ベクター
SG11201706445SA (en) 2015-02-10 2017-09-28 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
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
AU2021261832B2 (en) 2025-01-30
MA42934A (fr) 2018-08-01
US10512675B2 (en) 2019-12-24
NZ780570A (en) 2024-08-30
IL257939B (en) 2022-11-01
CA2999297A1 (en) 2017-03-30
CL2018000740A1 (es) 2018-06-08
CN108778323A (zh) 2018-11-09
JP2024147801A (ja) 2024-10-16
JP2018535929A (ja) 2018-12-06
TW201717999A (zh) 2017-06-01
RU2742352C2 (ru) 2021-02-05
WO2017053677A1 (en) 2017-03-30
KR102805910B1 (ko) 2025-05-13
TWI756185B (zh) 2022-03-01
AU2016326602A1 (en) 2018-03-29
US20240058423A1 (en) 2024-02-22
TWI841908B (zh) 2024-05-11
RU2020126034A (ru) 2020-09-02
JP2022010141A (ja) 2022-01-14
IL257939A (en) 2018-05-31
IL297353B1 (en) 2025-10-01
TW202220690A (zh) 2022-06-01
MX2018003702A (es) 2018-04-30
TW202446416A (zh) 2024-12-01
RU2018114888A (ru) 2019-10-24
EP3352787A1 (en) 2018-08-01
KR20180053335A (ko) 2018-05-21
IL297353A (en) 2022-12-01
IL257939B2 (en) 2023-03-01
US20200061161A1 (en) 2020-02-27
PE20241346A1 (es) 2024-07-03
AU2021261832A1 (en) 2021-12-02
US20170087219A1 (en) 2017-03-30
ZA201801657B (en) 2021-07-28
RU2018114888A3 (es) 2019-10-24
BR112018006074A2 (pt) 2018-10-09
NZ740521A (en) 2023-11-24
US11690898B2 (en) 2023-07-04
PH12018500594A1 (en) 2018-09-24

Similar Documents

Publication Publication Date Title
PE20181338A1 (es) Vectores del factor viii del virus adeno-asociado, particulas viricas asociadas y formulaciones terapeuticas que los comprenden
CL2023003001A1 (es) Novedosas proteínas de la cápside del virus adenoasociado
CL2018001522A1 (es) Focalización de péptidos para dirigir virus adenoasociados (aavs)
UY36021A (es) Proteìnas fc multimèricas
MX391208B (es) Vectores del virus adenoasociado de b-sarcoglicano y microarn-29 y el uso de los mismos en la distrofia muscular.
MX2021004294A (es) Suministro mejorado de particulas virales al cuerpo estriado y al cortex.
PE20160769A1 (es) Vectores del factor viii del virus adeno-asociado
PE20181922A1 (es) Terapia genica para tratar la hemofilia a
CL2024001715A1 (es) Anticuerpo aislado que se une a la proteína e del virus del dengue; y uso.
CY1124143T1 (el) Οστικες μορφογενετικες πρωτεϊνες
AR115097A1 (es) Vectores virales adenoasociados dirigidos al hígado
CL2017001920A1 (es) Anticuerpos humanos contra glicoproteína del virus ébola
CO2017006580A2 (es) Anticuerpos humanos para hemaglutinina de influenza
MX2017001062A (es) Proteinas de fusion de interleucina 2/receptor de interleucina-2 alfa y metodos de uso.
EA201890725A1 (ru) Агонисты фарнезоидного х-рецептора и их применение
GEP20227447B (en) Constructs having sirp-alpha domain or variant thereof
EA201792500A1 (ru) Aav-опосредованная экспрессия антител против гриппа и способы их использования
PE20171133A1 (es) Particulas tipo virus modificadas de cmv
MX381286B (es) Adenovirus que comprende una fracción de unión a albúmina.
MX2017011732A (es) Proteínas de fusión que comprenden glicoproteínas de la superficie del virus alfa modificado y antígeno asociado al tumor y métodos de los mismos.
EA201690826A1 (ru) Рекомбинантные гликопротеиды и их применения
MX390927B (es) Partículas semejantes a virus recombinantes que utilizan la proteína gag del virus de inmunodeficiencia bovina.
AR095431A1 (es) Formulaciones de fviii recombinante
CY1124859T1 (el) Ομοδιμερικο πεπτιδιο για τη θεραπεια του διαβητη και σχετικων νοσων
AR106131A1 (es) Vectores del factor viii del virus adenoasociado, partículas víricas asociadas y formulaciones terapéuticas que las comprenden, método