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
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transgene
raav vector
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JP2022541793A5 (enExample
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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)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (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 (2)

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JP2022541793A true JP2022541793A (ja) 2022-09-27
JP2022541793A5 JP2022541793A5 (enExample) 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

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

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116867512A (zh) * 2020-12-23 2023-10-10 佛罗里达州大学研究基金会 包装效率提高的用于心脏基因治疗的载体
CN119317718A (zh) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 用于改善心脏转导和肝脏去靶向的aav衣壳
EP4634394A2 (en) 2022-12-17 2025-10-22 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
WO2025007046A1 (en) 2023-06-29 2025-01-02 The Trustees Of The University Of Pennsylvania Mutant aav with central nervous system targeting motifs and compositions containing same
KR20260114566A (ko) 2023-11-14 2026-07-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 심근 및 골격근-특이적 표적화 모티프를 갖는 조성물 및 이의 용도
WO2026132516A1 (en) * 2024-12-20 2026-06-25 Patrick Most S100a1 protein-derived peptide for use in treating cancer or in treating or preventing antineoplastic agent-mediated cytotoxicity, cancer therapy-related cardiac dysfunction or cancer therapy related heart failure
CN121674484A (zh) * 2025-11-14 2026-03-17 广州派真生物技术有限公司 一种高产细胞毒性基因的rAAV质粒系统及应用

Citations (2)

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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

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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
US20200308582A1 (en) * 2017-10-12 2020-10-01 Universität Heidelberg Kcnk3-based gene therapy of cardiac arrhythmia
CN112272705A (zh) * 2018-06-08 2021-01-26 佛罗里达大学研究基金会有限公司 用于心肌病的aav心脏基因治疗

Patent Citations (2)

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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

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

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