AU2018352855B2 - Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof - Google Patents

Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof Download PDF

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Publication number
AU2018352855B2
AU2018352855B2 AU2018352855A AU2018352855A AU2018352855B2 AU 2018352855 B2 AU2018352855 B2 AU 2018352855B2 AU 2018352855 A AU2018352855 A AU 2018352855A AU 2018352855 A AU2018352855 A AU 2018352855A AU 2018352855 B2 AU2018352855 B2 AU 2018352855B2
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amino acid
seq
gly
capsid protein
asn
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AU2018352855A
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AU2018352855A1 (en
Inventor
Marwa BENHAJSALAH
Saswati Chatterjee
Serena Nicole DOLLIVE
James Anthony Mcswiggen
Jaime PROUT
Albert Barnes Seymour
Laura Jane Smith
Thia Baboval ST. MARTIN
Kamehameha K. WONG
Jason Boke WRIGHT
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City of Hope
Homology Medicines Inc
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City of Hope
Homology Medicines Inc
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    • 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
    • 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
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/075—Adenoviridae
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2018352855A 2017-10-18 2018-10-17 Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof Active AU2018352855B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762574163P 2017-10-18 2017-10-18
US62/574,163 2017-10-18
US201862621102P 2018-01-24 2018-01-24
US62/621,102 2018-01-24
PCT/US2018/056271 WO2019079437A1 (en) 2017-10-18 2018-10-17 ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RESTORING THE FUNCTION OF THE HBB GENE AND METHODS OF USE

Publications (2)

Publication Number Publication Date
AU2018352855A1 AU2018352855A1 (en) 2020-04-02
AU2018352855B2 true AU2018352855B2 (en) 2022-07-14

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ID=64332161

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018352855A Active AU2018352855B2 (en) 2017-10-18 2018-10-17 Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof

Country Status (12)

Country Link
US (1) US12201657B2 (https=)
EP (1) EP3697450A1 (https=)
JP (1) JP2021500070A (https=)
KR (1) KR20200095462A (https=)
CN (1) CN111447954A (https=)
AU (1) AU2018352855B2 (https=)
BR (1) BR112020007765A2 (https=)
CA (1) CA3079437A1 (https=)
IL (1) IL273877A (https=)
MX (1) MX2020004092A (https=)
SG (1) SG11202002737PA (https=)
WO (1) WO2019079437A1 (https=)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
CN109536494A (zh) * 2017-10-31 2019-03-29 广东赤萌医疗科技有限公司 一种用于修复HBB1基因点突变的gRNA、基因编辑系统、表达载体和基因编辑试剂盒
EP4085143B1 (en) * 2019-12-31 2025-08-13 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
CN111363756B (zh) * 2020-04-02 2023-01-17 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种珠蛋白基因双表达慢病毒载体及其应用
WO2021207077A1 (en) * 2020-04-06 2021-10-14 Homology Medicines, Inc. Adeno-associated virus compositions for ids gene transfer and methods of use thereof
WO2022221319A2 (en) * 2021-04-12 2022-10-20 Graphite Bio, Inc. Methods and compositions for production of genetically modified primary cells
US20250127812A1 (en) * 2021-08-10 2025-04-24 GentiBio. Inc. Compositions and methods for engineering stable tregs

Citations (1)

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WO2016049230A1 (en) * 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

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US5449759A (en) * 1987-05-16 1995-09-12 Somatogen, Inc. Hemoglobins with intersubunit desulfide bonds
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
DE69727492T2 (de) * 1996-12-05 2004-12-16 Crucell Holland B.V. Genetische modifikation der hämatopoietischen repopulation von stammzellen in primaten
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
CN101348786B (zh) * 2007-07-20 2011-05-11 谭孟群 一种人β珠蛋白基因及其重组腺相关病毒载体
WO2009013559A1 (en) 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
US8628966B2 (en) * 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
US11242525B2 (en) * 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
CA2960209C (en) 2014-09-04 2023-08-29 Memorial Sloan-Kettering Cancer Center Globin gene therapy for treating hemoglobinopathies
US20180135074A1 (en) * 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
US11136597B2 (en) * 2016-02-16 2021-10-05 Yale University Compositions for enhancing targeted gene editing and methods of use thereof

Patent Citations (1)

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Publication number Priority date Publication date Assignee Title
WO2016049230A1 (en) * 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

Also Published As

Publication number Publication date
AU2018352855A1 (en) 2020-04-02
JP2021500070A (ja) 2021-01-07
MX2020004092A (es) 2021-01-08
IL273877A (en) 2020-05-31
KR20200095462A (ko) 2020-08-10
CA3079437A1 (en) 2019-04-25
BR112020007765A2 (pt) 2020-10-20
WO2019079437A1 (en) 2019-04-25
US20190134118A1 (en) 2019-05-09
CN111447954A (zh) 2020-07-24
US12201657B2 (en) 2025-01-21
EP3697450A1 (en) 2020-08-26
SG11202002737PA (en) 2020-05-28

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