CO2023014900A2 - Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho - Google Patents

Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho

Info

Publication number
CO2023014900A2
CO2023014900A2 CONC2023/0014900A CO2023014900A CO2023014900A2 CO 2023014900 A2 CO2023014900 A2 CO 2023014900A2 CO 2023014900 A CO2023014900 A CO 2023014900A CO 2023014900 A2 CO2023014900 A2 CO 2023014900A2
Authority
CO
Colombia
Prior art keywords
gene
right ventricular
treating
aav
treatment
Prior art date
Application number
CONC2023/0014900A
Other languages
English (en)
Inventor
Farah Sheikh
William Bradford
Original Assignee
Univ California
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 California filed Critical Univ California
Publication of CO2023014900A2 publication Critical patent/CO2023014900A2/es

Links

Classifications

    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • 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
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Cardiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (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

Composiciones y métodos para prevenir y tratar arritmia cardíaca. Métodos para prevenir o tratar miocardiopatía arritmogénica del ventrículo derecho (ARVC), que comprenden administrar a un sujeto que lo necesite una cantidad profiláctica o eficaz para tratamiento de una composición que comprende un gen placofilina-2 (PKP2). La composición comprende además un vector de virus adenoasociado (AAV) para administrar el gen PKP-2. En formas de realización, la invención proporciona que el AAV es un serotipo de AAV cardiotrópico y contiene un promotor cardíaco específico. Método para tratar una enfermedad cardiovascular caracterizada por un complejo de unión célula-célula cardíaca anormal que comprende administrar a un sujeto que lo necesite una cantidad profiláctica o eficaz para tratamiento de una composición que comprende un gen placofilina-2 (PKP2).
CONC2023/0014900A 2021-04-12 2023-10-31 Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho CO2023014900A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163173527P 2021-04-12 2021-04-12
PCT/US2022/024478 WO2022221320A1 (en) 2021-04-12 2022-04-12 Gene therapy for arrhythmogenic right ventricular cardiomyopathy

Publications (1)

Publication Number Publication Date
CO2023014900A2 true CO2023014900A2 (es) 2023-11-20

Family

ID=83640995

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0014900A CO2023014900A2 (es) 2021-04-12 2023-10-31 Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho

Country Status (10)

Country Link
EP (1) EP4323534A1 (es)
JP (1) JP2024514110A (es)
KR (1) KR20230170686A (es)
CN (1) CN117178057A (es)
AU (1) AU2022258329A1 (es)
BR (1) BR112023021037A2 (es)
CA (1) CA3213231A1 (es)
CO (1) CO2023014900A2 (es)
IL (1) IL307506A (es)
WO (1) WO2022221320A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023001615A (es) 2020-08-07 2023-03-08 Spacecraft Seven Llc Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040142325A1 (en) * 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
US7960100B1 (en) * 2004-04-30 2011-06-14 Celera Corporation Colon cancer targets and uses thereof
US20070072175A1 (en) * 2005-05-13 2007-03-29 Biogen Idec Ma Inc. Nucleotide array containing polynucleotide probes complementary to, or fragments of, cynomolgus monkey genes and the use thereof
AU2013243953A1 (en) * 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of nuclear proteins
EP3808844A1 (en) * 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
WO2021053222A1 (en) * 2019-09-20 2021-03-25 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy

Also Published As

Publication number Publication date
EP4323534A1 (en) 2024-02-21
AU2022258329A1 (en) 2023-10-12
JP2024514110A (ja) 2024-03-28
BR112023021037A2 (pt) 2023-12-19
CN117178057A (zh) 2023-12-05
IL307506A (en) 2023-12-01
CA3213231A1 (en) 2022-10-20
KR20230170686A (ko) 2023-12-19
WO2022221320A1 (en) 2022-10-20
AU2022258329A9 (en) 2023-10-26

Similar Documents

Publication Publication Date Title
CO2023014900A2 (es) Tratamiento génico para miocardiopatía arritmogénica del ventrículo derecho
BR112021011143A2 (pt) Composições para redução específica de drg da expressão de transgene
AR118850A1 (es) Composiciones útiles en el tratamiento de leucodistrofia metacromática
CO2017006494A2 (es) Bacteriófago clo-pep-1 de clostridium perfringens para inhibir la proliferación de clostridium perfringens
ECSP19001018A (es) Moduladores del receptor adrenérgico beta -3 útiles para el tratamiento o prevención de trastornos relacionados con los mismos
MX2017010369A (es) Arni variante.
CO2022012917A2 (es) Vectores de genoterapia para tratar enfermedades cardíacas
CL2018001868A1 (es) Análogos del egf(a) con sustituyentes de ácidos grasos.
BR112023001852A2 (pt) Terapia gênica com placofilina-2 (pkp2) usando vetor de aav
CO6640236A2 (es) Compisicones de ciclasa de maav guanulato y métodos para tratar la amaurosis congénita de leber 1(lca1)
CR20200224A (es) Composiciones y métodos de tratamiento de la insuficiencia cardiaca con fracción de eyección reducida con dapagliflozina
CO2019010062A2 (es) Novedoso bacteriófago clo-pep-2 de clostridium perfringens y su uso para inhibir la proliferación de clostridium perfringens
BR112021023952A2 (pt) Métodos de rejuvenescimento de tecido envelhecido pela inibição de 15-hidroxiprostaglandina desidrogenase (15-pgdh)
AR105752A1 (es) Métodos para el tratamiento de enfermedades cardiovasculares
CO2021012074A2 (es) Suministro de β-sarcoglicano mediante vectores de virus adenoasociado y el tratamiento de la distrofia muscular
CL2018001548A1 (es) Tratamiento de colestasis intrahepática y enfermedades del hígado relacionadas
MX2020013313A (es) Terapia genica cardiaca con aav para cardiomiopatia.
BR112022008214A2 (pt) Composições e métodos para tratar perda auditiva neurossensorial usando sistemas de vetor duplo de otoferlina
EA202290001A1 (ru) Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута
ECSP20055797A (es) Métodos y composiciones para tratar enfermedades pulmonares crónicas
CL2022002067A1 (es) Composición para prevenir o tratar enfermedades pulmonares que comprende proteína 1 de unión de hialuronano
CL2023001650A1 (es) Tratamiento de la enfermedad de danon
BR112018017240A2 (pt) ?polinucleotídeo, vetor de ácido nucleico, preparação farmacêutica, kit, e, método para fabricar um vetor viral adenoassociado recombinante?
AR122289A1 (es) Terapia genética cardíaca con aav para la cardiomiopatía en humanos
BR112017024065A2 (pt) método para tratar insuficiência cardíaca avançada em um indivíduo humano