AR122289A1 - Terapia genética cardíaca con aav para la cardiomiopatía en humanos - Google Patents

Terapia genética cardíaca con aav para la cardiomiopatía en humanos

Info

Publication number
AR122289A1
AR122289A1 ARP200102021A ARP200102021A AR122289A1 AR 122289 A1 AR122289 A1 AR 122289A1 AR P200102021 A ARP200102021 A AR P200102021A AR P200102021 A ARP200102021 A AR P200102021A AR 122289 A1 AR122289 A1 AR 122289A1
Authority
AR
Argentina
Prior art keywords
raav
compositions
vector
heart
aav
Prior art date
Application number
ARP200102021A
Other languages
English (en)
Inventor
Hugh Lee Sweeney
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 AR122289A1 publication Critical patent/AR122289A1/es

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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
    • A61K48/005—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • 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
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—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
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4728—Calcium binding proteins, e.g. calmodulin
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6472—Cysteine endopeptidases (3.4.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/22—Cysteine endopeptidases (3.4.22)
    • C12Y304/22061—Caspase-8 (3.4.22.61)
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

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

Abstract

La presente divulgación se relaciona con composiciones y métodos útiles en el tratamiento de afecciones cardíacas. Las composiciones y métodos divulgados se basan en terapias génicas que comprenden un vector de AAV recombinante para el suministro de dos o más transgenes en el corazón de un sujeto humano, en donde los transgenes comprenden una proteína S100A1 y un Represor de la Apoptosis cardíaca con un inhibidor apoptótico del Dominio de Reclutamiento de Caspasas (cARC). En diversas realizaciones, las composiciones y los métodos divulgados en la presente comprenden vectores que constan de secuencias de ADNc de cARC y/o S100A1 que son optimizados con codones para la expresión en humanos. En algunos aspectos, la focalización en múltiples fuentes de una o más afecciones cardíacas puede proporcionar beneficios sinérgicos durante el tratamiento. Reivindicación 1: Un vector de virus adenoasociado recombinante (rAAV) que comprende un polinucleótido que consta de una secuencia que es al menos 90%, al menos 95%, o al menos 99,5% idéntica a cualquiera de las secuencias de nucleótidos de SEQ ID Nº 6 - 8, 16, y 21. Reivindicación 16: Una partícula de rAAV que comprende el vector de rAAV de cualquiera de las reivindicaciones 1 - 15 encapsidado en una cápside de AAV. Reivindicación 19: Una composición que comprende la partícula de rAAV de cualquiera de las reivindicaciones 16 - 18. Reivindicación 20: Un método de tratamiento de un sujeto humano que padece de una enfermedad cardíaca que comprende la administración al sujeto de la composición de la reivindicación 19 o de la partícula de rAAV de cualquiera de las reivindicaciones 16 - 18.
ARP200102021A 2019-07-19 2020-07-17 Terapia genética cardíaca con aav para la cardiomiopatía en humanos AR122289A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962876540P 2019-07-19 2019-07-19

Publications (1)

Publication Number Publication Date
AR122289A1 true AR122289A1 (es) 2022-08-31

Family

ID=74193792

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP200102021A AR122289A1 (es) 2019-07-19 2020-07-17 Terapia genética cardíaca con aav para la cardiomiopatía en humanos

Country Status (16)

Country Link
US (1) US20220265858A1 (es)
EP (1) EP3999074A4 (es)
JP (1) JP2022541793A (es)
KR (1) KR20220034801A (es)
CN (1) CN114206351A (es)
AR (1) AR122289A1 (es)
AU (1) AU2020316339A1 (es)
BR (1) BR112022000898A2 (es)
CA (1) CA3142534A1 (es)
CL (1) CL2022000115A1 (es)
CO (1) CO2022001686A2 (es)
IL (1) IL289667A (es)
MX (1) MX2022000767A (es)
SG (1) SG11202112140SA (es)
TW (1) TW202117017A (es)
WO (1) WO2021016126A1 (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3205116A1 (en) * 2020-12-23 2022-06-30 University Of Florida Research Foundation, Incorporated Increased packaging efficiency of vector for cardiac gene therapy
WO2023147304A1 (en) 2022-01-25 2023-08-03 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002002148A2 (en) * 2000-06-30 2002-01-10 Collateral Therapeutics, Inc. Dual recombinant gene therapy compositions and methods of use
US8383601B2 (en) * 2006-10-30 2013-02-26 Thomas Jefferson University Tissue specific gene therapy treatment
EP2295072A1 (en) * 2009-09-15 2011-03-16 Max-Delbrück-Centrum für Molekulare Medizin (MDC) Use of ARC for inhibiting cell death during liver failure
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US20200308582A1 (en) * 2017-10-12 2020-10-01 Universität Heidelberg Kcnk3-based gene therapy of cardiac arrhythmia
KR20210018902A (ko) * 2018-06-08 2021-02-18 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 심근병증에 대한 aav 심장 유전자 요법

Also Published As

Publication number Publication date
WO2021016126A8 (en) 2021-09-10
SG11202112140SA (en) 2021-11-29
KR20220034801A (ko) 2022-03-18
CL2022000115A1 (es) 2022-10-07
MX2022000767A (es) 2022-04-25
US20220265858A1 (en) 2022-08-25
AU2020316339A1 (en) 2021-11-18
JP2022541793A (ja) 2022-09-27
TW202117017A (zh) 2021-05-01
CN114206351A (zh) 2022-03-18
AU2020316339A2 (en) 2021-11-25
BR112022000898A2 (pt) 2022-06-07
IL289667A (en) 2022-03-01
EP3999074A4 (en) 2023-08-16
EP3999074A1 (en) 2022-05-25
CA3142534A1 (en) 2021-01-28
CO2022001686A2 (es) 2022-03-29
WO2021016126A1 (en) 2021-01-28

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