JP2022541793A - ヒトにおける心筋症のためのａａｖ心臓遺伝子治療 - Google Patents

ヒトにおける心筋症のためのａａｖ心臓遺伝子治療 Download PDF

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JP2022541793A
JP2022541793A JP2022503802A JP2022503802A JP2022541793A JP 2022541793 A JP2022541793 A JP 2022541793A JP 2022503802 A JP2022503802 A JP 2022503802A JP 2022503802 A JP2022503802 A JP 2022503802A JP 2022541793 A JP2022541793 A JP 2022541793A
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raav
vector
seq
transgene
raav vector
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JPWO2021016126A5 (https=
JP2022541793A5 (https=
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リー スウィニー，ヒュー
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University of Florida Research Foundation Inc
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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
    • 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
    • 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
    • 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

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  • 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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Marine Sciences & Fisheries (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)
JP2022503802A 2019-07-19 2020-07-17 ヒトにおける心筋症のためのａａｖ心臓遺伝子治療 Pending JP2022541793A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962876540P 2019-07-19 2019-07-19
US62/876,540 2019-07-19
PCT/US2020/042663 WO2021016126A1 (en) 2019-07-19 2020-07-17 Aav cardiac gene therapy for cardiomyopathy in humans

Publications (3)

Publication Number Publication Date
JP2022541793A true JP2022541793A (ja) 2022-09-27
JPWO2021016126A5 JPWO2021016126A5 (https=) 2023-07-26
JP2022541793A5 JP2022541793A5 (https=) 2023-07-26

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JP2022503802A Pending JP2022541793A (ja) 2019-07-19 2020-07-17 ヒトにおける心筋症のためのａａｖ心臓遺伝子治療

Country Status (16)

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2023007602A (es) * 2020-12-23 2023-09-21 Univ Florida Mayor eficiencia del empaquetamiento de vectores para terapia genica cardiaca.
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
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
KR20250135916A (ko) 2022-12-17 2025-09-15 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심장 및 골격근 특이적 표적화 모티프를 갖는 재조합 aav 돌연변이체 벡터 및 이를 포함하는 조성물
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016534029A (ja) * 2013-10-01 2016-11-04 ルプレヒト−カールス−ウニヴェルジテート ハイデルベルクＲｕｐｒｅｃｈｔ−Ｋａｒｌｓ−Ｕｎｉｖｅｒｓｉｔａｅｔ Ｈｅｉｄｅｌｂｅｒｇ Ｓ１００に基づく心臓パワー不全の処置
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein

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DE19915485A1 (de) * 1999-04-07 2000-10-19 Hugo A Katus Therapie der Herzinsuffizienz
CA2412847A1 (en) * 2000-06-30 2002-01-10 Collateral Therapeutics, Inc. 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
EP3694996A1 (en) * 2017-10-12 2020-08-19 Universität Heidelberg Kcnk3-based gene therapy of cardiac arrhythmia
US20210260215A1 (en) * 2018-06-08 2021-08-26 University Of Florida Research Foundation, Incorporated Aav cardiac gene therapy for cardiomyopathy

Patent Citations (2)

* Cited by examiner, † Cited by third party
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JP2016534029A (ja) * 2013-10-01 2016-11-04 ルプレヒト−カールス−ウニヴェルジテート ハイデルベルクＲｕｐｒｅｃｈｔ−Ｋａｒｌｓ−Ｕｎｉｖｅｒｓｉｔａｅｔ Ｈｅｉｄｅｌｂｅｒｇ Ｓ１００に基づく心臓パワー不全の処置
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein

Non-Patent Citations (4)

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Title
AKC CANINE HEALTH FOUNDATION: ""Therapeutic gene transfer abrogates canine dilated cardiomyopathy."", [ONLINE], INTERNET, JPN6024029909, 4 April 2019 (2019-04-04), ISSN: 0005511090 *
SLEEPER, M.M.: ""Status of Therapeutic Gene Transfer to Treat Cardiovascular Disease in Dogs and Cats."", VET CLIN SMALL ANIM, vol. 47, JPN6024029910, 2017, pages 1113 - 1121, ISSN: 0005511089 *
SUBHASIS CHATTERJEE, MD ET AL.: "Blocking the development of postischemic cardiomyopathy with viral gene transfer of the apoptosis re", THE JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY, vol. 125, no. 6, JPN7024003187, 2003, pages 1461 - 1469, ISSN: 0005380541 *
WANG XIANG-HAI ET AL.: "Construction of recombinant adeno−associated virus vector expressing hs100a1 gene", 中国臨床薬理学与治療学, vol. 18, no. 8, JPN7024003188, 2013, pages 853 - 857, ISSN: 0005380540 *

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Publication number Publication date
WO2021016126A1 (en) 2021-01-28
BR112022000898A2 (pt) 2022-06-07
AU2020316339A2 (en) 2021-11-25
US20220265858A1 (en) 2022-08-25
CO2022001686A2 (es) 2022-03-29
WO2021016126A8 (en) 2021-09-10
SG11202112140SA (en) 2021-11-29
KR20220034801A (ko) 2022-03-18
CA3142534A1 (en) 2021-01-28
AU2020316339A1 (en) 2021-11-18
AR122289A1 (es) 2022-08-31
EP3999074A1 (en) 2022-05-25
CL2022000115A1 (es) 2022-10-07
EP3999074A4 (en) 2023-08-16
TW202117017A (zh) 2021-05-01
CN114206351A (zh) 2022-03-18
IL289667A (en) 2022-03-01
MX2022000767A (es) 2022-04-25

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