BR112022000898A2 - Terapia de gene cardíaco aav para cardiomiopatia em seres humanos - Google Patents

Terapia de gene cardíaco aav para cardiomiopatia em seres humanos

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Publication number
BR112022000898A2
BR112022000898A2 BR112022000898A BR112022000898A BR112022000898A2 BR 112022000898 A2 BR112022000898 A2 BR 112022000898A2 BR 112022000898 A BR112022000898 A BR 112022000898A BR 112022000898 A BR112022000898 A BR 112022000898A BR 112022000898 A2 BR112022000898 A2 BR 112022000898A2
Authority
BR
Brazil
Prior art keywords
cardiac
humans
compositions
cardiomyopathy
gene therapy
Prior art date
Application number
BR112022000898A
Other languages
English (en)
Inventor
Lee Sweeney Hugh
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 BR112022000898A2 publication Critical patent/BR112022000898A2/pt

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    • 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
    • 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
    • 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)
    • 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
    • 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)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (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

terapia de gene cardíaco aav para cardiomiopatia em seres humanos. a presente invenção refere-se a composições e métodos úteis no tratamento de condições cardíacas. as composições e métodos descritos são baseados em terapias de gene compreendendo um vetor aav recombinante para a liberação de dois ou mais transgenes no coração de um ser humano, em que os transgenes compreendem uma proteína s100a1 e um repressor de apoptose cardíaca com inibidor apoptótico de domínio de recrutamento de caspase (carc). em várias modalidades, as composições e métodos descritos no presente documento compreendem vetores compreendendo sequências de cdna de s100a1 e/ou carc com códons otimizados para expressão em seres humanos. em alguns aspectos, atingir múltiplas fontes de uma ou mais doenças cardíacas pode fornecer benefícios sinérgicos durante o tratamento.
BR112022000898A 2019-07-19 2020-07-17 Terapia de gene cardíaco aav para cardiomiopatia em seres humanos BR112022000898A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962876540P 2019-07-19 2019-07-19
PCT/US2020/042663 WO2021016126A1 (en) 2019-07-19 2020-07-17 Aav cardiac gene therapy for cardiomyopathy in humans

Publications (1)

Publication Number Publication Date
BR112022000898A2 true BR112022000898A2 (pt) 2022-06-07

Family

ID=74193792

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022000898A BR112022000898A2 (pt) 2019-07-19 2020-07-17 Terapia de gene cardíaco aav para cardiomiopatia em seres humanos

Country Status (16)

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

Families Citing this family (4)

* 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
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1294407A2 (en) * 2000-06-30 2003-03-26 Collateral Therapeutics Dual recombinant gene therapy compositions and methods of use
WO2008054713A2 (en) * 2006-10-30 2008-05-08 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
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
US20200308582A1 (en) * 2017-10-12 2020-10-01 Universität Heidelberg Kcnk3-based gene therapy of cardiac arrhythmia
BR112020024935A2 (pt) * 2018-06-08 2021-03-09 University Of Florida Research Foundation, Incorporated Terapia gênica cardíaca com aav para cardiomiopatia

Also Published As

Publication number Publication date
CL2022000115A1 (es) 2022-10-07
KR20220034801A (ko) 2022-03-18
US20220265858A1 (en) 2022-08-25
WO2021016126A1 (en) 2021-01-28
SG11202112140SA (en) 2021-11-29
AR122289A1 (es) 2022-08-31
JP2022541793A (ja) 2022-09-27
MX2022000767A (es) 2022-04-25
AU2020316339A1 (en) 2021-11-18
EP3999074A1 (en) 2022-05-25
IL289667A (en) 2022-03-01
CA3142534A1 (en) 2021-01-28
CN114206351A (zh) 2022-03-18
EP3999074A4 (en) 2023-08-16
TW202117017A (zh) 2021-05-01
CO2022001686A2 (es) 2022-03-29
WO2021016126A8 (en) 2021-09-10
AU2020316339A2 (en) 2021-11-25

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