EA202191977A1 - Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии а - Google Patents

Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии а

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Publication number
EA202191977A1
EA202191977A1 EA202191977A EA202191977A EA202191977A1 EA 202191977 A1 EA202191977 A1 EA 202191977A1 EA 202191977 A EA202191977 A EA 202191977A EA 202191977 A EA202191977 A EA 202191977A EA 202191977 A1 EA202191977 A1 EA 202191977A1
Authority
EA
Eurasian Patent Office
Prior art keywords
gene therapy
hemophilia
viral vectors
increased expression
vectors encoding
Prior art date
Application number
EA202191977A
Other languages
English (en)
Inventor
Ханспетер Роттенстайнер
Фридрих Шайфлингер
Original Assignee
Баксалта Инкорпорейтед
Баксалта Гмбх
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 Баксалта Инкорпорейтед, Баксалта Гмбх filed Critical Баксалта Инкорпорейтед
Publication of EA202191977A1 publication Critical patent/EA202191977A1/ru

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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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Abstract

Изобретение относится, среди других аспектов, к кодон-модифицированным полинуклеотидам, кодирующим варианты фактора VIII, для экспрессии в клетках млекопитающих. В некоторых вариантах осуществления изобретение также относится к векторам для генной терапии млекопитающих и к способам лечения гемофилии A.
EA202191977A 2019-01-16 2020-01-15 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии а EA202191977A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962793058P 2019-01-16 2019-01-16
PCT/US2020/013722 WO2020150375A1 (en) 2019-01-16 2020-01-15 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (1)

Publication Number Publication Date
EA202191977A1 true EA202191977A1 (ru) 2021-12-15

Family

ID=69593774

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202191977A EA202191977A1 (ru) 2019-01-16 2020-01-15 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии а

Country Status (19)

Country Link
US (2) US11707536B2 (ru)
EP (1) EP3911672A1 (ru)
JP (1) JP2022517267A (ru)
KR (1) KR20210117291A (ru)
CN (1) CN113614104A (ru)
AR (1) AR117825A1 (ru)
AU (1) AU2020208375A1 (ru)
BR (1) BR112021013874A2 (ru)
CA (1) CA3127065A1 (ru)
CL (1) CL2021001880A1 (ru)
CO (1) CO2021009338A2 (ru)
EA (1) EA202191977A1 (ru)
EC (1) ECSP21060605A (ru)
IL (1) IL284648A (ru)
MX (1) MX2021008550A (ru)
PE (1) PE20220429A1 (ru)
SG (1) SG11202107421YA (ru)
TW (1) TW202039546A (ru)
WO (1) WO2020150375A1 (ru)

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CN116710554A (zh) * 2021-11-25 2023-09-05 四川至善唯新生物科技有限公司 基因工程改造获得的分泌能力和凝血活性增强的人类八因子
US20230374546A1 (en) * 2022-03-11 2023-11-23 Homology Medicines, Inc. Bidirectional dual promoter expression vectors and uses thereof

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Also Published As

Publication number Publication date
CN113614104A (zh) 2021-11-05
PE20220429A1 (es) 2022-03-29
US20200289672A1 (en) 2020-09-17
CL2021001880A1 (es) 2022-01-14
KR20210117291A (ko) 2021-09-28
BR112021013874A2 (pt) 2021-09-21
SG11202107421YA (en) 2021-08-30
MX2021008550A (es) 2021-08-19
TW202039546A (zh) 2020-11-01
US11707536B2 (en) 2023-07-25
CA3127065A1 (en) 2020-07-23
WO2020150375A1 (en) 2020-07-23
EP3911672A1 (en) 2021-11-24
US20230211017A1 (en) 2023-07-06
IL284648A (en) 2021-08-31
JP2022517267A (ja) 2022-03-07
CO2021009338A2 (es) 2021-07-30
AR117825A1 (es) 2021-08-25
AU2020208375A1 (en) 2021-07-29
ECSP21060605A (es) 2022-01-31

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