PE20181922A1 - Terapia genica para tratar la hemofilia a - Google Patents

Terapia genica para tratar la hemofilia a

Info

Publication number
PE20181922A1
PE20181922A1 PE2018001994A PE2018001994A PE20181922A1 PE 20181922 A1 PE20181922 A1 PE 20181922A1 PE 2018001994 A PE2018001994 A PE 2018001994A PE 2018001994 A PE2018001994 A PE 2018001994A PE 20181922 A1 PE20181922 A1 PE 20181922A1
Authority
PE
Peru
Prior art keywords
gene therapy
treat hemophilia
compositions
hemophilia
treat
Prior art date
Application number
PE2018001994A
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 PE20181922A1 publication Critical patent/PE20181922A1/es

Links

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
    • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporciona composiciones y regimenes utiles en el tratamiento de la hemoglobina A. Las composiciones incluyen el virus adenoasociado recombinante (rAAV) con un potenciador y promotor de trastiretina que impulsan la expresion de un Factor VIII humano.
PE2018001994A 2016-04-15 2017-04-13 Terapia genica para tratar la hemofilia a PE20181922A1 (es)

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
PE20181922A1 true PE20181922A1 (es) 2018-12-11

Family

ID=59253992

Family Applications (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 (1) PE20181922A1 (es)
RU (1) RU2762257C2 (es)
SA (1) SA518400233B1 (es)
SG (2) SG11201808422QA (es)
TW (1) TWI791433B (es)
WO (1) WO2017180857A1 (es)
ZA (1) ZA201806477B (es)

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CN112912518A (zh) * 2018-10-15 2021-06-04 再生生物股份有限公司 用于测量复制缺陷型病毒载体和病毒的感染性的方法
KR20210133242A (ko) * 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
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JP2023501263A (ja) * 2019-11-01 2023-01-18 フリーライン セラピューティクス リミテッド 第ｖｉｉｉ因子ポリペプチド
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AU2021270447A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
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AU2022214529A1 (en) * 2021-02-01 2023-08-03 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of niemann pick type a disease
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WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
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Also Published As

Publication number Publication date
AU2017248659A1 (en) 2018-10-18
IL262215A (en) 2018-11-29
TWI791433B (zh) 2023-02-11
RU2018136611A3 (es) 2020-08-11
US10888628B2 (en) 2021-01-12
US20240000974A1 (en) 2024-01-04
AU2017248659B2 (en) 2022-08-11
US11779656B2 (en) 2023-10-10
JP2022062121A (ja) 2022-04-19
SG10202009849WA (en) 2020-11-27
EP3452102B1 (en) 2024-06-12
CN109562191A (zh) 2019-04-02
IL262215B2 (en) 2023-07-01
US20190343966A1 (en) 2019-11-14
JP7347933B2 (ja) 2023-09-20
WO2017180857A1 (en) 2017-10-19
RU2018136611A (ru) 2020-05-15
US20200085975A1 (en) 2020-03-19
IL262215B1 (en) 2023-03-01
MX2018012537A (es) 2019-02-25
TW201741458A (zh) 2017-12-01
JP2019513794A (ja) 2019-05-30
KR20190032274A (ko) 2019-03-27
SG11201808422QA (en) 2018-10-30
ZA201806477B (en) 2024-09-25
EP3452102A1 (en) 2019-03-13
EP3452102B9 (en) 2024-09-04
BR112018071200A2 (pt) 2019-04-09
KR102450833B1 (ko) 2022-10-05
RU2762257C2 (ru) 2021-12-17
CA3019425A1 (en) 2017-10-19
SA518400233B1 (ar) 2022-08-23

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