AR124489A1 - Vectores adenovirales para genoterapia - Google Patents

Vectores adenovirales para genoterapia

Info

Publication number
AR124489A1
AR124489A1 ARP210103644A ARP210103644A AR124489A1 AR 124489 A1 AR124489 A1 AR 124489A1 AR P210103644 A ARP210103644 A AR P210103644A AR P210103644 A ARP210103644 A AR P210103644A AR 124489 A1 AR124489 A1 AR 124489A1
Authority
AR
Argentina
Prior art keywords
itr
serotype
adenoviral
fiber
viral polypeptides
Prior art date
Application number
ARP210103644A
Other languages
English (en)
Inventor
Soumitra Roy
Ashvin Reddy Bashyam
Original Assignee
Ensoma 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 Ensoma Inc filed Critical Ensoma Inc
Publication of AR124489A1 publication Critical patent/AR124489A1/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
    • A61K48/0008—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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774—Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • 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
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/10071—Demonstrated in vivo effect
    • 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
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/10332—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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Cell Biology (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)
  • Peptides Or Proteins (AREA)

Abstract

La presente descripción incluye vectores adenovirales que se caracterizan por la transducción eficiente de los HSC, por ejemplo, para la genoterapia in vivo. La presente descripción incluye, entre otras cosas, los genomas y vectores Ad3, Ad7, Ad11, Ad14, Ad16, Ad21, Ad34, Ad37 y Ad50. Los genomas y vectores Ad3, Ad7, Ad11, Ad14, Ad16, Ad21, Ad34, Ad37 y Ad50 de la presente descripción pueden incluir cargas activas terapéuticas. Reivindicación 1: Un método de genoterapia in vivo en un sujeto mamífero, donde el método comprende administrarle al sujeto un vector adenoviral, en donde el vector adenoviral comprende: (a) una cápside que comprende uno o más polipéptidos virales de un serotipo Ad3, Ad7, Ad11, Ad14, Ad16, Ad21, Ad34, Ad37 o Ad50, en donde uno o más de los polipéptidos virales comprende uno o más de: (i) una fracción globular de fibra; (ii) un cuerpo de fibra; (iii) una cola de fibra; (iv) un pentón; y (v) un hexón; y (b) un genoma de ADN bicatenario que comprende una carga activa de ácido nucleico heterólogo. Reivindicación 7: Un vector donante adenoviral que comprende: (a) una cápside que comprende uno o más polipéptidos virales de un serotipo Ad3, Ad7, Ad11, Ad14, Ad16, Ad21, Ad34, Ad37 o Ad50, en donde uno o más de los polipéptidos virales comprende uno o más de: (i) una fracción globular de fibra; (ii) un cuerpo de fibra; (iii) una cola de fibra; (iv) un pentón; y (v) un hexón; y (b) un genoma de ADN bicatenario que comprende una carga activa de ácido nucleico heterólogo. Reivindicación 20: Un genoma de vector donante adenoviral que comprende: (a) una ITR en 3’ y una ITR en 5’, en donde la ITR en 3’ y la ITR en 5’ son cada una del mismo serotipo que se selecciona de un serotipo Ad3, Ad7, Ad11, Ad14, Ad16, Ad21, Ad34, Ad37 o Ad50; (b) una secuencia de encapsidación, en donde la secuencia de encapsidación es del serotipo de la ITR; y (c) una carga activa de ácido nucleico heterólogo.
ARP210103644A 2020-12-22 2021-12-22 Vectores adenovirales para genoterapia AR124489A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063129233P 2020-12-22 2020-12-22

Publications (1)

Publication Number Publication Date
AR124489A1 true AR124489A1 (es) 2023-04-05

Family

ID=82158454

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210103644A AR124489A1 (es) 2020-12-22 2021-12-22 Vectores adenovirales para genoterapia

Country Status (12)

Country Link
US (1) US20240108752A1 (es)
EP (1) EP4267200A1 (es)
JP (1) JP2024500191A (es)
KR (1) KR20230145051A (es)
CN (1) CN117157109A (es)
AR (1) AR124489A1 (es)
AU (1) AU2021410765A1 (es)
CA (1) CA3204826A1 (es)
IL (1) IL303899A (es)
MX (1) MX2023007505A (es)
TW (1) TW202242123A (es)
WO (1) WO2022140618A1 (es)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2002332736A1 (en) * 2001-08-30 2003-03-18 The Government Of The United States Of America, As Represented By The Secretary Of The Department Of New adenovirus type 7 vectors
WO2003093455A2 (en) * 2002-04-30 2003-11-13 Avior Therapeutics, Inc. Adenovirus vectors for immunotherapy
RS57043B1 (sr) * 2013-10-25 2018-05-31 Psioxus Therapeutics Ltd Onkolitički adenovirusi snabdeveni heterologus genima
EP3137120A4 (en) * 2014-05-01 2018-03-14 University Of Washington In vivo gene engineering with adenoviral vectors

Also Published As

Publication number Publication date
IL303899A (en) 2023-08-01
EP4267200A1 (en) 2023-11-01
WO2022140618A8 (en) 2022-08-04
US20240108752A1 (en) 2024-04-04
WO2022140618A1 (en) 2022-06-30
AU2021410765A1 (en) 2023-07-13
CA3204826A1 (en) 2022-06-30
CN117157109A (zh) 2023-12-01
MX2023007505A (es) 2023-09-08
TW202242123A (zh) 2022-11-01
KR20230145051A (ko) 2023-10-17
JP2024500191A (ja) 2024-01-04

Similar Documents

Publication Publication Date Title
Tilemann et al. Gene therapy for heart failure
ES2401608T3 (es) Blastocitos CD34 mesenquimales para uso en terapia génica de diabetes
Hajjar Potential of gene therapy as a treatment for heart failure
Hall et al. Unity and diversity in the human adenoviruses: exploiting alternative entry pathways for gene therapy
Shen et al. Effect of hypoxia on Ad5 infection, transgene expression and replication
MX2022015079A (es) Variantes de hsd17b13 y usos de las mismas.
CN107405411A (zh) 使用腺病毒载体的体内基因改造
Kaur et al. Modified mRNA as a Therapeutic Tool for the Heart
de Macedo Braga et al. In situ delivery of bone marrow cells and mesenchymal stem cells improves cardiovascular function in hypertensive rats submitted to myocardial infarction
Dakin et al. Efficient transduction of primary vascular cells by the rare adenovirus serotype 49 vector
AR125368A1 (es) Vectores adenovirales para genoterapia
Pankajakshan et al. Successful transfection of genes using AAV-2/9 vector in swine coronary and peripheral arteries
Renaud-Gabardos et al. Therapeutic benefit and gene network regulation by combined gene transfer of apelin, FGF2, and SERCA2a into ischemic heart
JP6162224B2 (ja) 血管組織の形質導入のためのアデノウイルスベクター
JP2017526341A (ja) 複数のレトロウイルス株における非対称標的部位を組み換えるための、十分に寛容化され高度に特異的なテーラーメイドリコンビナーゼ
AR124489A1 (es) Vectores adenovirales para genoterapia
Naim et al. Gene therapy for heart failure: where do we stand?
Kim et al. The effectiveness of the oncolytic activity induced by Ad5/F35 adenoviral vector is dependent on the cumulative cellular conditions of survival and autophagy
Fouletier-Dilling et al. Novel compound enables high-level adenovirus transduction in the absence of an adenovirus-specific receptor
Su et al. The increase of VEGF secretion from endothelial progenitor cells post ultrasonic VEGF gene delivery enhances the proliferation and migration of endothelial cells
Rust et al. Gene transfer into mouse embryonic stem cell-derived cardiac myocytes mediated by recombinant adenovirus
Fujiwara et al. Synergistic interaction of telomerase-specific oncolytic virotherapy and chemotherapeutic agents for human cancer
Wu et al. Retrovirus-mediated transfection of the tissue-type plasminogen activator gene results in increased thrombolysis of blood clots
CO6241172A2 (es) Poxvirus de mapache que expresa genes de antígenos de felino
Lee et al. Enhanced proliferation of endothelial progenitor cells post-ultrasonic microbubble transfection is plasmid DNA size dependent and contributed by interleukin-6 generation