CN114206351A - 用于人中心肌病的aav心脏基因治疗 - Google Patents

用于人中心肌病的aav心脏基因治疗 Download PDF

Info

Publication number
CN114206351A
CN114206351A CN202080052456.6A CN202080052456A CN114206351A CN 114206351 A CN114206351 A CN 114206351A CN 202080052456 A CN202080052456 A CN 202080052456A CN 114206351 A CN114206351 A CN 114206351A
Authority
CN
China
Prior art keywords
raav
cardiac
vector
transgene
seq
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202080052456.6A
Other languages
English (en)
Chinese (zh)
Inventor
休·利·斯威尼
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Florida Research Foundation Inc
Original Assignee
University of Florida Research Foundation Inc
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 University of Florida Research Foundation Inc filed Critical University of Florida Research Foundation Inc
Publication of CN114206351A publication Critical patent/CN114206351A/zh
Pending legal-status Critical Current

Links

Images

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)
  • 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)
CN202080052456.6A 2019-07-19 2020-07-17 用于人中心肌病的aav心脏基因治疗 Pending CN114206351A (zh)

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 (1)

Publication Number Publication Date
CN114206351A true CN114206351A (zh) 2022-03-18

Family

ID=74193792

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202080052456.6A Pending CN114206351A (zh) 2019-07-19 2020-07-17 用于人中心肌病的aav心脏基因治疗

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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100190840A1 (en) * 2006-10-30 2010-07-29 Thomas Jefferson University Tissue specific gene therapy treatment
US20130012455A1 (en) * 2009-09-15 2013-01-10 Max-Delbruck-Centrum Fur Molekulare Medizin (Mdc) Berlin-Buch 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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CA2925691A1 (en) * 2013-10-01 2015-04-09 Ruprecht-Karls-Univeristat Heidelberg S100 based treatment of cardiac power 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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100190840A1 (en) * 2006-10-30 2010-07-29 Thomas Jefferson University Tissue specific gene therapy treatment
US20130012455A1 (en) * 2009-09-15 2013-01-10 Max-Delbruck-Centrum Fur Molekulare Medizin (Mdc) Berlin-Buch 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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SUBHASIS CHATTERJEE等: "Blocking the development of postischemic cardiomyopathy with viral gene transfer of the apoptosis repressor with caspase recruitment domain", THE JOURNAL OF THORACIC AND CARDIOVASCULAR SURGERY》, vol. 125, no. 6, pages 1461 - 1469, XP055660674, DOI: 10.1016/S0022-5223(02)73229-7 *

Also Published As

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
JP2022541793A (ja) 2022-09-27
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
IL289667A (en) 2022-03-01
MX2022000767A (es) 2022-04-25

Similar Documents

Publication Publication Date Title
CN114206351A (zh) 用于人中心肌病的aav心脏基因治疗
AU2019282822B2 (en) AAV cardiac gene therapy for cardiomyopathy
CN109069672A (zh) 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症
US20250269065A1 (en) B-cell lymphoma 2-associated anthanogene 3 (bag3) gene therapy using aav vector
CN114231532B (zh) 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用
CN116867512A (zh) 包装效率提高的用于心脏基因治疗的载体
EA049034B1 (ru) Генная терапия сердца с применением aav для кардиомиопатии у людей
EA046740B1 (ru) Генная терапия сердца с применением aav для кардиомиопатии у людей
EA046532B1 (ru) Генная терапия сердца с помощью aav при кардиомиопатии
EA048262B1 (ru) Повышенная эффективность упаковки вектора для генотерапии сердечных заболеваний
US20230220391A1 (en) CILP-1 Inhibitors for Use in the Treatment of Dilated Cardiomyopathies
US20230250450A1 (en) NRF2 Activator for Use in Treating Dilated Cardiomyopathies
WO2026060422A2 (en) Materials and method for enhancing muscle contraction and mitochondrial function

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
WD01 Invention patent application deemed withdrawn after publication

Application publication date: 20220318

WD01 Invention patent application deemed withdrawn after publication