[go: up one dir, main page]

AR123756A1 - Construcciones de ácidos nucléicos, vectores virales y partículas virales - Google Patents

Construcciones de ácidos nucléicos, vectores virales y partículas virales

Info

Publication number
AR123756A1
AR123756A1 ARP210102783A ARP210102783A AR123756A1 AR 123756 A1 AR123756 A1 AR 123756A1 AR P210102783 A ARP210102783 A AR P210102783A AR P210102783 A ARP210102783 A AR P210102783A AR 123756 A1 AR123756 A1 AR 123756A1
Authority
AR
Argentina
Prior art keywords
viral
constructions
nucleic acids
viral particles
particles
Prior art date
Application number
ARP210102783A
Other languages
English (en)
Inventor
Stefanie Marie Dedeurwaerdere
Tal Kramer
Csilla Sipeky
Brittany Nicole Vallette
Meiyu Xu
Original Assignee
UCB Biopharma SRL
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 UCB Biopharma SRL filed Critical UCB Biopharma SRL
Publication of AR123756A1 publication Critical patent/AR123756A1/es

Links

Classifications

    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0356—Animal model for processes and diseases of the central nervous system, e.g. stress, learning, schizophrenia, pain, epilepsy
    • 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

Landscapes

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

Abstract

La presente invención se refiere a construcciones de ácido nucleico, vectores virales y partículas virales que comprenden un transgén que codifica GAT-1; y uso de dichas partículas virales para tratar enfermedades mediadas por deterioro de SLC6A1.
ARP210102783A 2020-10-09 2021-10-07 Construcciones de ácidos nucléicos, vectores virales y partículas virales AR123756A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063089817P 2020-10-09 2020-10-09

Publications (1)

Publication Number Publication Date
AR123756A1 true AR123756A1 (es) 2023-01-11

Family

ID=78332750

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210102783A AR123756A1 (es) 2020-10-09 2021-10-07 Construcciones de ácidos nucléicos, vectores virales y partículas virales

Country Status (18)

Country Link
US (1) US20230365652A1 (es)
EP (1) EP4225782A1 (es)
JP (1) JP2023544264A (es)
KR (1) KR20230083335A (es)
CN (1) CN116390935A (es)
AR (1) AR123756A1 (es)
AU (1) AU2021358413A1 (es)
BR (1) BR112023004874A2 (es)
CA (1) CA3195052A1 (es)
CL (2) CL2023000785A1 (es)
CO (1) CO2023002727A2 (es)
EC (1) ECSP23024245A (es)
IL (1) IL301255A (es)
MX (1) MX2023003994A (es)
PE (1) PE20230987A1 (es)
TW (1) TW202229321A (es)
WO (1) WO2022074105A1 (es)
ZA (1) ZA202304290B (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
WO2025228976A1 (en) 2024-04-29 2025-11-06 UCB Biopharma SRL Gene therapy

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
AU663725B2 (en) 1991-08-20 1995-10-19 United States Of America, Represented By The Secretary, Department Of Health And Human Services, The Adenovirus mediated transfer of genes to the gastrointestinal tract
US6225115B1 (en) * 1992-03-04 2001-05-01 Synaptic Pharmaceutical Corporation DNA encoding taurine and GABA transporters and uses thereof
EP1103610A1 (en) 1999-11-26 2001-05-30 Introgene B.V. Production of vaccines from immortalised mammalian cell lines
FR2808803B1 (fr) 2000-05-11 2004-12-10 Agronomique Inst Nat Rech Cellules es modifiees et gene specifique de cellules es
FR2836924B1 (fr) 2002-03-08 2005-01-14 Vivalis Lignees de cellules aviaires utiles pour la production de substances d'interet
EP1528101A1 (en) 2003-11-03 2005-05-04 ProBioGen AG Immortalized avian cell lines for virus production
US20080213765A1 (en) * 2005-02-28 2008-09-04 Integragen Human Autism Susceptibility Genes Encoding a Neurotransmitter Transporter and Uses Thereof
FR2884255B1 (fr) 2005-04-11 2010-11-05 Vivalis Utilisation de lignees de cellules souches aviaires ebx pour la production de vaccin contre la grippe
EP1985305A1 (en) 2007-04-24 2008-10-29 Vivalis Duck embryonic derived stem cell lines for the production of viral vaccines
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2020097395A1 (en) * 2018-11-08 2020-05-14 The Regents Of The University Of California Systems and methods for targeting cancer cells
EP4055174A4 (en) * 2019-11-08 2024-01-03 The Board Of Regents Of The University Of Texas System TRANSGENIC CASSETTES, AAV VECTORS AND AAV VECTORS FOR EXPRESSING HUMAN CODON-OPTIMIZED SLCA1

Also Published As

Publication number Publication date
CL2023000785A1 (es) 2023-10-13
EP4225782A1 (en) 2023-08-16
AU2021358413A1 (en) 2023-05-25
PE20230987A1 (es) 2023-06-21
KR20230083335A (ko) 2023-06-09
CN116390935A (zh) 2023-07-04
CO2023002727A2 (es) 2023-03-27
JP2023544264A (ja) 2023-10-23
ECSP23024245A (es) 2023-05-31
WO2022074105A1 (en) 2022-04-14
US20230365652A1 (en) 2023-11-16
IL301255A (en) 2023-05-01
ZA202304290B (en) 2024-09-25
AU2021358413A9 (en) 2024-10-24
BR112023004874A2 (pt) 2023-05-02
TW202229321A (zh) 2022-08-01
CL2025001325A1 (es) 2025-07-25
CA3195052A1 (en) 2022-04-14
MX2023003994A (es) 2023-04-24

Similar Documents

Publication Publication Date Title
CL2025001325A1 (es) Construcciones de ácido nucleico, vectores virales y partículas virales
CL2024000409A1 (es) Ácidos nucleicos recombinantes que codifican una o más proteínas cosméticas para usos estéticos.
MX2022015081A (es) Variantes de hsd17b13 y usos de las mismas.
CO2019014840A2 (es) Vectores aav auto reguladores para la expresion segura de mecp2 en sidrome de rett
CL2018000740A1 (es) Vectores del factor viii del virus adeno-asociado, partículas víricas asociadas y formulaciones terapéuticas que los comprenden
MX2021006784A (es) Constructos de arni para inhibir la expresion de pnpla3 y metodos de uso de estos.
MX388739B (es) Casetes optimizados de expresión del gen humano del factor viii de coagulación y su uso.
UY38003A (es) Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas
PE20190433A1 (es) Vacuna contra vrs
BR112018011881A2 (pt) proteínas capsidiais modificadas para liberação aumentada de vetores de parvovírus
CO2021007916A2 (es) Ácido ribonucleico (arn) que codifica para una proteína
BR112019004711A2 (pt) receptores de antígeno quiméricos compreendendo domínios de fibronectina tipo iii específicos para bcma e usos dos mesmos
MX2021001292A (es) Metodos para la modificacion genica de celulas hematopoyeticas.
EA202190454A1 (ru) Новый crispr-ассоциированный белок и его применение
MX2021014465A (es) Construcciones de arni para inhibir la expresion de scap y metodos de uso de las mismas.
UY39916A (es) Partículas de aav que comprenden una proteína de la cápside hepatotrópica y alfa-galactosidasa y su uso para tratar la enfermedad de fabry
CO2024005920A2 (es) Constructos de ácido nucleico, vectores víricos y partículas víricas
MX2019002134A (es) Nuevo paramixovirus y sus usos.
EA201790241A1 (ru) Эффективная селективность в отношении рекомбинантных белков
BR112022020942A2 (pt) Métodos de tratamento de infecções virais usando arginase
CO2019008755A2 (es) Vacuna recombinante contra helmintos en pichia pastoris y procesos de purificación y producción de proteínas como vacuna contra helmintos
MX2025001858A (es) Virus de la gripe modificados
EA201991797A1 (ru) Высокоактивный и короткий промотор, предназначенный для экспрессии гетерологичных генов
CO2024001768A2 (es) Método para obtener una cápside de virus adenoasociado modificada
MX2019012106A (es) Metodo de sintesis y uso de bionanoparticulas con actividad asparaginasa utilizando capsides virales para tratar enfermedades asociadas con la dependencia de asparagina.