MX421375B - Terapia genica cardiaca con aav para cardiomiopatia - Google Patents

Terapia genica cardiaca con aav para cardiomiopatia

Info

Publication number
MX421375B
MX421375B MX2020013313A MX2020013313A MX421375B MX 421375 B MX421375 B MX 421375B MX 2020013313 A MX2020013313 A MX 2020013313A MX 2020013313 A MX2020013313 A MX 2020013313A MX 421375 B MX421375 B MX 421375B
Authority
MX
Mexico
Prior art keywords
aav
cardiomyopathy
gene therapy
cardiac gene
transgenes
Prior art date
Application number
MX2020013313A
Other languages
English (en)
Spanish (es)
Other versions
MX2020013313A (es
Inventor
Hugh Lee Sweeney
Margaret Mary Sleeper
Original Assignee
Univ Florida
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 Florida filed Critical Univ Florida
Publication of MX2020013313A publication Critical patent/MX2020013313A/es
Publication of MX421375B publication Critical patent/MX421375B/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/04Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • 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
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • 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
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • 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
    • A01K2267/035Animal model for multifactorial diseases
    • A01K2267/0375Animal model for cardiovascular diseases
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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

Landscapes

  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
MX2020013313A 2018-06-08 2019-06-07 Terapia genica cardiaca con aav para cardiomiopatia MX421375B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862682772P 2018-06-08 2018-06-08
US201962822015P 2019-03-21 2019-03-21
PCT/US2019/036157 WO2019237067A1 (en) 2018-06-08 2019-06-07 Aav cardiac gene therapy for cardiomyopathy

Publications (2)

Publication Number Publication Date
MX2020013313A MX2020013313A (es) 2021-02-22
MX421375B true MX421375B (es) 2025-03-14

Family

ID=68770676

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020013313A MX421375B (es) 2018-06-08 2019-06-07 Terapia genica cardiaca con aav para cardiomiopatia

Country Status (14)

Country Link
US (1) US20210260215A1 (https=)
EP (1) EP3814512A4 (https=)
JP (2) JP2021526818A (https=)
KR (1) KR20210018902A (https=)
CN (1) CN112272705A (https=)
AU (1) AU2019282822B2 (https=)
BR (1) BR112020024935A2 (https=)
CA (1) CA3100280A1 (https=)
CL (1) CL2020003190A1 (https=)
CO (1) CO2020016718A2 (https=)
IL (1) IL279225B2 (https=)
MX (1) MX421375B (https=)
SG (1) SG11202011061SA (https=)
WO (1) WO2019237067A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112022000898A2 (pt) * 2019-07-19 2022-06-07 Univ Florida Terapia de gene cardíaco aav para cardiomiopatia em seres humanos
BR112023001336A2 (pt) * 2020-08-05 2023-02-14 Spacecraft Seven Llc Terapia gênica de csrp3 (proteína 3 rica em cisteína e glicina)
MX2023007602A (es) * 2020-12-23 2023-09-21 Univ Florida Mayor eficiencia del empaquetamiento de vectores para terapia genica cardiaca.
CA3252359A1 (en) * 2022-03-02 2023-09-07 Cevec Pharmaceuticals Gmbh IMPROVED CELL LINES AND PROCESSES FOR THE PRODUCTION OF ADENO-ASSOCIATED VECTORS
EP4239063A1 (en) * 2022-03-02 2023-09-06 CEVEC Pharmaceuticals GmbH Improved cell lines and methods for the production of adeno-associated vectors

Family Cites Families (7)

* 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
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
CN105452458A (zh) * 2013-06-07 2016-03-30 加利福尼亚大学董事会 用于治疗心力衰竭和增加心脏功能的不能产生环腺苷酸的腺苷酸环化酶和组合物和方法
DK3405215T3 (da) * 2016-01-19 2022-09-19 Univ California Fremgangsmåder til behandling af danon-sygdom og andre autofagilidelser
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy

Also Published As

Publication number Publication date
MX2020013313A (es) 2021-02-22
WO2019237067A1 (en) 2019-12-12
JP2021526818A (ja) 2021-10-11
CO2020016718A2 (es) 2021-04-08
CL2020003190A1 (es) 2021-04-30
EP3814512A1 (en) 2021-05-05
EP3814512A4 (en) 2022-03-09
CA3100280A1 (en) 2019-12-12
JP2024097779A (ja) 2024-07-19
CN112272705A (zh) 2021-01-26
AU2019282822B2 (en) 2025-09-04
IL279225A (en) 2021-01-31
IL279225B1 (en) 2024-07-01
KR20210018902A (ko) 2021-02-18
US20210260215A1 (en) 2021-08-26
AU2019282822A1 (en) 2020-11-26
IL279225B2 (en) 2024-11-01
SG11202011061SA (en) 2020-12-30
BR112020024935A2 (pt) 2021-03-09

Similar Documents

Publication Publication Date Title
CO2020016718A2 (es) Terapia génica cardíaca con aav para cardiomiopatía
CO2022001686A2 (es) Terapia genética cardíaca con aav para la cardiomiopatía en humanos
MX2022003063A (es) Composiciones terapeuticas, combinaciones y metodos de uso.
MX2020011786A (es) Oligomeros antisentido para tratamiento de afecciones y enfermedades.
UY38999A (es) Degradadores de proteína tirosina fosfatasa y métodos de uso de los mismos
PE20211419A1 (es) Vectores virales recombinantes y acidos nucleicos para producirlos
MX2019007873A (es) Terapia génica para tratar la enfermedad de wilson.
BR112022002905A2 (pt) Composições e métodos para modular splicing e expressão de proteína
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
CL2022003147A1 (es) Composiciones y métodos para tratar pérdida auditiva asociada con gjb2
BR112022007158A2 (pt) Agentes imunomodulatórios de il-2 em combinação com inibidores do ponto de verificação imunológico
MX2024008461A (es) Metodos para tratar trastornos linfoproliferativos malignos.
MX2022003758A (es) Composiciones para el cuidado bucal que comprenden acido beta de lupulo y aminoacido.
CO2021004934A2 (es) Composiciones y métodos para tratar retinitis pigmentosa
MX2021013901A (es) Composiciones y métodos para el tratamiento del cáncer.
BR112022009279A2 (pt) Tratamento de angioedema hereditário com vetores de terapia gênica específicos do fígado
ECSP20055797A (es) Métodos y composiciones para tratar enfermedades pulmonares crónicas
BR112022002794A2 (pt) Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
DOP2023000132A (es) Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1
MX2022000232A (es) Composiciones y metodos para tratar o evitar infecciones oculares con filociclovir.
EA202092654A1 (ru) Генная терапия сердца с помощью aav при кардиомиопатии
ZA202102023B (en) Potentiation of helminth treatment
CL2023001731A1 (es) Mayor eficiencia del empaquetamiento de vectores para terapia génica cardíaca.
MX2022000349A (es) Cardioterápico.
BR112022020846A2 (pt) Agentes imunoestimuladores em combinação com inibidores de angiogênese