KR20200095462A - Hbb 유전자 기능 회복을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 - Google Patents

Hbb 유전자 기능 회복을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Download PDF

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KR20200095462A
KR20200095462A KR1020207012240A KR20207012240A KR20200095462A KR 20200095462 A KR20200095462 A KR 20200095462A KR 1020207012240 A KR1020207012240 A KR 1020207012240A KR 20207012240 A KR20207012240 A KR 20207012240A KR 20200095462 A KR20200095462 A KR 20200095462A
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amino acid
seq
capsid protein
protein corresponding
gly
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Korean (ko)
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사스와티 채터지
카메하메하 케이. 웡
마르와 벤하지살라
로라 제인 스미스
알버트 반스 시무어
제이슨 보케 라이트
제임스 안토니 맥위겐
세레나 니콜 돌라이브
마틴 티아 바보발 세인트
제이미 프라우트
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시티 오브 호프
호몰로지 메디슨, 인크.
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
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    • 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
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    • 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
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    • C12N15/86—Viral vectors
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    • 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
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    • A01K2207/00—Modified animals
    • A01K2207/12—Animals modified by administration of exogenous cells
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    • 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
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    • 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
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    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0381—Animal model for diseases of the hematopoietic system
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    • 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)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Developmental Biology & Embryology (AREA)
  • Immunology (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)
KR1020207012240A 2017-10-18 2018-10-17 Hbb 유전자 기능 회복을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법 Ceased KR20200095462A (ko)

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 (1)

Publication Number Publication Date
KR20200095462A true KR20200095462A (ko) 2020-08-10

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KR1020207012240A Ceased KR20200095462A (ko) 2017-10-18 2018-10-17 Hbb 유전자 기능 회복을 위한 아데노-연관 바이러스 조성물 및 이의 사용 방법

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

Families Citing this family (6)

* Cited by examiner, † Cited by third party
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
CA3166374A1 (en) * 2019-12-31 2021-07-08 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
BR112022019182A2 (pt) * 2020-04-06 2022-11-29 Homology Medicines Inc Composições de vírus adeno-associadas para transferência do gene ids e métodos de uso da mesma
EP4323513A4 (en) * 2021-04-12 2025-09-17 Kamau Therapeutics Inc METHODS AND COMPOSITIONS FOR THE PRODUCTION OF GENETICALLY MODIFIED PRIMARY CELLS
WO2023019185A1 (en) * 2021-08-10 2023-02-16 Gentibio, Inc. Compositions and methods for engineering stable tregs

Family Cites Families (12)

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Publication number Priority date Publication date Assignee Title
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
EP0938578B1 (en) * 1996-12-05 2004-02-04 Crucell Holland B.V. Genetic modification of primate hemopoietic repopulating stem cells
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
ES2746529T3 (es) 2014-09-04 2020-03-06 Memorial Sloan Kettering Cancer Center Terapia génica de globina para tratar hemoglobinopatías
PL3198018T3 (pl) 2014-09-24 2021-07-19 City Of Hope Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby
US20180135074A1 (en) * 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
AU2017221405A1 (en) * 2016-02-16 2018-09-20 Carnegie Mellon University Compositions for enhancing targeted gene editing and methods of use thereof

Also Published As

Publication number Publication date
US20190134118A1 (en) 2019-05-09
CA3079437A1 (en) 2019-04-25
MX2020004092A (es) 2021-01-08
EP3697450A1 (en) 2020-08-26
AU2018352855B2 (en) 2022-07-14
IL273877A (en) 2020-05-31
WO2019079437A1 (en) 2019-04-25
US12201657B2 (en) 2025-01-21
CN111447954A (zh) 2020-07-24
AU2018352855A1 (en) 2020-04-02
SG11202002737PA (en) 2020-05-28
BR112020007765A2 (pt) 2020-10-20
JP2021500070A (ja) 2021-01-07

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