MX2023001615A - Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. - Google Patents

Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

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Publication number
MX2023001615A
MX2023001615A MX2023001615A MX2023001615A MX2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A MX 2023001615 A MX2023001615 A MX 2023001615A
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Mexico
Prior art keywords
pkp2
plakophilin
gene therapy
vector
promoter
Prior art date
Application number
MX2023001615A
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English (en)
Inventor
Raj Prabhakar
David Ricks
Christopher Dean Herzog
Chester Bittencort Sacramento
Original Assignee
Spacecraft Seven Llc
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Publication date
Application filed by Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of MX2023001615A publication Critical patent/MX2023001615A/es

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
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    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Microbiology (AREA)
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  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

En la presente se proporciona una genoterapia para PKP2 (Placofilina-2), por ejemplo, mediante el uso de un vector de virus adenoasociado (AAV). El promotor del vector puede ser un promotor MHCK7 o un promotor de troponina cardíaca T (HTNNT2). La cápside puede ser una cápside de AAV9 o AAVrh74 o variante funcional de esta. Pueden usarse otros promotores o cápsides. Se proporcionan además métodos de tratamiento, tales como por administración intravenosa, intracoronaria, intracarótida o intracardíaca del vector de rAAV, y otras composiciones y métodos.
MX2023001615A 2020-08-07 2021-08-09 Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav. MX2023001615A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063063032P 2020-08-07 2020-08-07
PCT/US2021/045220 WO2022032226A1 (en) 2020-08-07 2021-08-09 Plakophilin-2 (pkp2) gene therapy using aav vector

Publications (1)

Publication Number Publication Date
MX2023001615A true MX2023001615A (es) 2023-03-08

Family

ID=80118582

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023001615A MX2023001615A (es) 2020-08-07 2021-08-09 Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

Country Status (11)

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US (2) US20220168447A1 (es)
EP (1) EP4192487A1 (es)
JP (1) JP2023538519A (es)
KR (1) KR20230043869A (es)
CN (1) CN116194154A (es)
AU (1) AU2021320902A1 (es)
BR (1) BR112023001852A2 (es)
CA (1) CA3185267A1 (es)
IL (1) IL300263A (es)
MX (1) MX2023001615A (es)
WO (1) WO2022032226A1 (es)

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US20220168446A1 (en) 2022-06-02
AU2021320902A1 (en) 2023-04-06
KR20230043869A (ko) 2023-03-31
BR112023001852A2 (pt) 2023-02-23
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