CL2020000295A1 - Métodos de terapia génica del factor viii (fviii) - Google Patents

Métodos de terapia génica del factor viii (fviii)

Info

Publication number
CL2020000295A1
CL2020000295A1 CL2020000295A CL2020000295A CL2020000295A1 CL 2020000295 A1 CL2020000295 A1 CL 2020000295A1 CL 2020000295 A CL2020000295 A CL 2020000295A CL 2020000295 A CL2020000295 A CL 2020000295A CL 2020000295 A1 CL2020000295 A1 CL 2020000295A1
Authority
CL
Chile
Prior art keywords
nucleic acid
fviii
factor viii
vector
gene therapy
Prior art date
Application number
CL2020000295A
Other languages
English (en)
Inventor
Xavier Anguela
Original Assignee
Spark Therapeutics 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 Spark Therapeutics Inc filed Critical Spark Therapeutics Inc
Publication of CL2020000295A1 publication Critical patent/CL2020000295A1/es

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Classifications

    • 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
    • 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
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—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 administration regime
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5184—Virus capsids or envelopes enclosing drugs
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Hematology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se divulgan métodos de uso de vectores que comprenden ácido nucleico y variantes de ácido nucleico que codifican la proteína de FVIII. En realizaciones particulares, un método para tratar a un ser humano que tiene hemofilia A incluye administrar un vector de virus adeno-asociado recombinante (rAAV) que comprende un ácido nucleico que codifica el Factor VIII (FVIII) o variante de ácido nucleico que codifica el Factor VIII (FVIII) que tiene una eliminación del dominio B (hFVIII-BDD). En algunos aspectos, una variante de ácido nucleico tiene una identidad del 95 % o más con la SEQ ID NO:7 y/o una variante de ácido nucleico no tiene más de 2 dinucleótidos de citosina-guanina (CpGs). En otros aspectos, se administra un vector de rAAV al ser humano a una dosis de menos de aproximadamente 6x10<sup>12</sup> genomas de vector por kilogramo (vg/kg).
CL2020000295A 2017-08-01 2020-01-31 Métodos de terapia génica del factor viii (fviii) CL2020000295A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762540053P 2017-08-01 2017-08-01
US201762583890P 2017-11-09 2017-11-09
US201762596535P 2017-12-08 2017-12-08
US201762596670P 2017-12-08 2017-12-08

Publications (1)

Publication Number Publication Date
CL2020000295A1 true CL2020000295A1 (es) 2020-12-04

Family

ID=65234171

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020000295A CL2020000295A1 (es) 2017-08-01 2020-01-31 Métodos de terapia génica del factor viii (fviii)

Country Status (17)

Country Link
US (1) US20200237930A1 (es)
EP (1) EP3661541A4 (es)
JP (1) JP7499174B2 (es)
KR (2) KR20240160648A (es)
CN (1) CN111163796A (es)
AU (2) AU2018312565A1 (es)
BR (1) BR112020001979A2 (es)
CA (1) CA3071519A1 (es)
CL (1) CL2020000295A1 (es)
CO (1) CO2020002283A2 (es)
IL (1) IL272373A (es)
MX (1) MX2020001402A (es)
MY (1) MY208862A (es)
PE (1) PE20200722A1 (es)
PH (1) PH12020500239A1 (es)
SG (1) SG11202000650YA (es)
WO (1) WO2019028192A1 (es)

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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
JP2021509812A (ja) 2018-01-05 2021-04-08 プレートレット バイオジェネシス， インコーポレイテッド 巨核球を産生するための組成物および方法
EP3813853A4 (en) 2018-06-29 2022-04-06 Platelet Biogenesis, Inc. DELIVERY COMPOSITIONS AND METHODS OF USE
US12497632B2 (en) * 2018-08-03 2025-12-16 Sangamo Therapeutics, Inc. Clinical parameters by expression of factor VIII
SG11202108357PA (en) 2019-02-15 2021-08-30 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
CN110684798A (zh) * 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CN114929735A (zh) * 2019-11-01 2022-08-19 自由行疗法有限公司 因子viii构建体
AU2020395323A1 (en) * 2019-12-06 2022-06-30 The Children's Hospital Of Philadelphia Compositions and methods for modulating factor VIII function
CN114989307B (zh) * 2022-05-11 2023-08-01 华兰生物工程股份有限公司 一种重组人凝血因子Ⅷ-Fc融合蛋白及制备方法
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用

Family Cites Families (9)

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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
JP2003511082A (ja) * 1999-10-12 2003-03-25 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 第ｖｉｉｉ因子をコードするアデノ随伴ウィルスベクターとその使用法
EP3044231B1 (en) * 2013-09-12 2020-08-05 BioMarin Pharmaceutical Inc. Aav vectors comprising a gene encoding factor viii
CN107427557B (zh) * 2014-08-13 2022-01-04 费城儿童医院 用于包装和表达变体因子viii以治疗血友病的改良表达组件
EP3283126B1 (en) * 2015-04-16 2019-11-06 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
TW202446416A (zh) * 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
MY189674A (en) * 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
WO2017123961A1 (en) * 2016-01-14 2017-07-20 The Children's Hospital Of Philadelphia Factor viii variants, nucleic acid sequences, and methods and uses for treatment of hemostasis disorders

Also Published As

Publication number Publication date
RU2020108209A (ru) 2021-09-02
AU2018312565A1 (en) 2020-02-27
MY208862A (en) 2025-06-04
CA3071519A1 (en) 2019-02-07
US20200237930A1 (en) 2020-07-30
WO2019028192A1 (en) 2019-02-07
IL272373A (en) 2020-03-31
CN111163796A (zh) 2020-05-15
KR20200066289A (ko) 2020-06-09
EP3661541A1 (en) 2020-06-10
JP7499174B2 (ja) 2024-06-13
PE20200722A1 (es) 2020-07-21
SG11202000650YA (en) 2020-02-27
MX2020001402A (es) 2021-01-29
BR112020001979A2 (pt) 2020-08-18
KR20240160648A (ko) 2024-11-11
CO2020002283A2 (es) 2020-04-24
JP2020533276A (ja) 2020-11-19
PH12020500239A1 (en) 2021-01-11
AU2024219336A1 (en) 2024-09-19
EP3661541A4 (en) 2021-09-01

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