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PE20250736A1 - Terapia genica para tratar la hemofilia a - Google Patents

Terapia genica para tratar la hemofilia a

Info

Publication number
PE20250736A1
PE20250736A1 PE2023002954A PE2023002954A PE20250736A1 PE 20250736 A1 PE20250736 A1 PE 20250736A1 PE 2023002954 A PE2023002954 A PE 2023002954A PE 2023002954 A PE2023002954 A PE 2023002954A PE 20250736 A1 PE20250736 A1 PE 20250736A1
Authority
PE
Peru
Prior art keywords
transthyretin
seq
set forth
enhancer
nucleotide sequence
Prior art date
Application number
PE2023002954A
Other languages
English (en)
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 PE20250736A1 publication Critical patent/PE20250736A1/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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporcionan composiciones y regimenes utiles en el tratamiento de la hemofilia A. Las composiciones incluyen el virus adenoasociado recombinante (rAAV) con un potenciador y promotor de transtiretina que impulsan la expresion de un Factor VIII humano. Se refiere a un virus adenoasociado recombinante (rAAV), donde dicho rAAV comprende una capside AAV, y un genoma del vector incluido en el, donde dicho genoma del vector comprende: (a) una secuencia de repeticion terminal invertida (ITR) 5' del AAV; (b) un potenciador de transtiretina (enTTR); (c) un promotor de transtiretina (TTR); (d) una secuencia de codificacion que codifica un Factor VIII humano con funcion de coagulacion; y (e) una 3' ITR del AAV; en donde la secuencia de codificacion comprende una secuencia de nucleotidos como se establece en SEQ ID NO:2, el promotor de transtiretina (TTR) comprende la secuencia de nucleotidos como se establece en SEQ ID NO:7, el potenciador de transtiretina (enTTR) comprende la secuencia de nucleotidos como se establece en SEQ ID NO: 5.
PE2023002954A 2016-04-15 2017-04-13 Terapia genica para tratar la hemofilia a PE20250736A1 (es)

Applications Claiming Priority (4)

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
PCT/US2017/027396 WO2017180857A1 (en) 2016-04-15 2017-04-13 Gene therapy for treating hemophilia a

Publications (1)

Publication Number Publication Date
PE20250736A1 true PE20250736A1 (es) 2025-03-10

Family

ID=59253992

Family Applications (2)

Application Number Title Priority Date Filing Date
PE2023002954A PE20250736A1 (es) 2016-04-15 2017-04-13 Terapia genica para tratar la hemofilia a
PE2018001994A PE20181922A1 (es) 2016-04-15 2017-04-13 Terapia genica para tratar la hemofilia a

Family Applications After (1)

Application Number Title Priority Date Filing Date
PE2018001994A PE20181922A1 (es) 2016-04-15 2017-04-13 Terapia genica para tratar la hemofilia a

Country Status (17)

Country Link
US (3) US11779656B2 (es)
EP (1) EP3452102B9 (es)
JP (2) JP7347933B2 (es)
KR (1) KR102450833B1 (es)
CN (1) CN109562191A (es)
AU (1) AU2017248659B2 (es)
BR (1) BR112018071200A2 (es)
CA (1) CA3019425A1 (es)
IL (1) IL262215B2 (es)
MX (1) MX2018012537A (es)
PE (2) PE20250736A1 (es)
RU (1) RU2762257C2 (es)
SA (1) SA518400233B1 (es)
SG (2) SG10202009849WA (es)
TW (1) TWI791433B (es)
WO (1) WO2017180857A1 (es)
ZA (1) ZA201806477B (es)

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CN116949052A (zh) * 2015-11-13 2023-10-27 武田药品工业株式会社 用于血友病a的基因治疗的具有增加的表达的编码重组fviii变体的病毒载体
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
AU2017248659B2 (en) * 2016-04-15 2022-08-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia A

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CN109562191A (zh) 2019-04-02
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