AR102234A1 - Vector adenoviral que codifica atonal homólogo-1 humano (hath1) - Google Patents

Vector adenoviral que codifica atonal homólogo-1 humano (hath1)

Info

Publication number
AR102234A1
AR102234A1 ARP150103277A ARP150103277A AR102234A1 AR 102234 A1 AR102234 A1 AR 102234A1 AR P150103277 A ARP150103277 A AR P150103277A AR P150103277 A ARP150103277 A AR P150103277A AR 102234 A1 AR102234 A1 AR 102234A1
Authority
AR
Argentina
Prior art keywords
region
human
adenoviral
promoter
hath1
Prior art date
Application number
ARP150103277A
Other languages
English (en)
Inventor
R Ettyreddy Damodar
E Brough Douglas
Original Assignee
Genvec 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 Genvec Inc filed Critical Genvec Inc
Publication of AR102234A1 publication Critical patent/AR102234A1/es

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    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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

Abstract

La solicitud se refiere a un vector adenoviral de replicación deficiente que comprende una secuencia de ácido nucleico que codifica la proteína atonal homóloga-1 humana (Hath1) unida operativamente a un promotor de la proteína fibrilar acídica de la glia humana (GFAP). La solicitud también se refiere a una composición y un método que utiliza el vector adenoviral para generar células sensoriales en el oído interno de un ser humano. Reivindicación 1: Un vector adenoviral que comprende: (a) un genoma adenoviral serotipo 5, excepto que se eliminen dos pares de bases en la región VA RNA I, uno o más nucleótidos endógenos de la región E1 se eliminen para generar un gen endógeno en la región E1 funcionalmente deficiente, uno o más nucleótidos endógenos se eliminen de la región E3, y se eliminen uno o más nucleótidos endógenos de la región E4 para generar un gen endógeno en la región E4 funcionalmente deficiente, caracterizado porque se retiene una repetición terminal invertida (ITR) de lado derecho, una secuencia de poliadenilación de la región E4 y una región de promotor E4, (b) una secuencia de poliadenilación temprana SV40, (c) una secuencia de ácido nucleico que codifica una proteína atonal homóloga-1 humana (Hath1) que comprende la SEQ ID Nº 1, (d) un promotor de la proteína fibrilar acídica de la glia humana (GFAP), y (e) un espaciador transcripcionalmente inerte (TIS), donde los elementos (b), (c) y (d) se encuentran en secuencia de 3 a 5 en relación con el genoma adenoviral en la región E1 del genoma adenoviral, y donde el elemento (e) se encuentra en la región E4 del genoma adenoviral entre la secuencia de poliadenilación de E4 y el promotor E4.
ARP150103277A 2014-10-09 2015-10-09 Vector adenoviral que codifica atonal homólogo-1 humano (hath1) AR102234A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462061748P 2014-10-09 2014-10-09

Publications (1)

Publication Number Publication Date
AR102234A1 true AR102234A1 (es) 2017-02-15

Family

ID=54365364

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150103277A AR102234A1 (es) 2014-10-09 2015-10-09 Vector adenoviral que codifica atonal homólogo-1 humano (hath1)

Country Status (19)

Country Link
US (2) US9951351B2 (es)
EP (1) EP3204505B1 (es)
JP (2) JP6863890B2 (es)
KR (1) KR20170082507A (es)
CN (1) CN106795526A (es)
AR (1) AR102234A1 (es)
AU (1) AU2015330793B2 (es)
BR (1) BR112017006450A2 (es)
CA (1) CA2958780A1 (es)
CO (1) CO2017003318A2 (es)
EA (1) EA201790623A1 (es)
IL (1) IL250572A0 (es)
MX (1) MX2017004565A (es)
MY (1) MY179637A (es)
RU (1) RU2707131C2 (es)
SG (1) SG11201701422YA (es)
TW (1) TWI710635B (es)
WO (1) WO2016057868A1 (es)
ZA (1) ZA201701908B (es)

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* Cited by examiner, † Cited by third party
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US9657637B2 (en) 2014-10-13 2017-05-23 Ford Global Technologies, Llc Method for controlling transitions in a variable displacement engine
WO2023091696A1 (en) 2021-11-19 2023-05-25 Christiana Care Gene Editing Institute, Inc. Adenovirus delivery system for cancer treatment

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TW201629230A (zh) 2016-08-16
RU2017112853A (ru) 2018-11-13
WO2016057868A1 (en) 2016-04-14
EA201790623A1 (ru) 2017-08-31
US11279951B2 (en) 2022-03-22
KR20170082507A (ko) 2017-07-14
JP6863890B2 (ja) 2021-04-21
RU2707131C2 (ru) 2019-11-22
EP3204505B1 (en) 2020-05-06
US20160102320A1 (en) 2016-04-14
US20180208944A1 (en) 2018-07-26
AU2015330793B2 (en) 2021-10-07
AU2015330793A1 (en) 2017-03-09
BR112017006450A2 (pt) 2017-12-12
EP3204505A1 (en) 2017-08-16
JP2021050219A (ja) 2021-04-01
MY179637A (en) 2020-11-11
CO2017003318A2 (es) 2017-06-30
JP2017532034A (ja) 2017-11-02
SG11201701422YA (en) 2017-04-27
ZA201701908B (en) 2019-06-26
RU2017112853A3 (es) 2019-04-25
CA2958780A1 (en) 2016-04-14
MX2017004565A (es) 2017-12-14
CN106795526A (zh) 2017-05-31
IL250572A0 (en) 2017-03-30
TWI710635B (zh) 2020-11-21
US9951351B2 (en) 2018-04-24

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