EA201891137A1 - Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a - Google Patents

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

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Publication number
EA201891137A1
EA201891137A1 EA201891137A EA201891137A EA201891137A1 EA 201891137 A1 EA201891137 A1 EA 201891137A1 EA 201891137 A EA201891137 A EA 201891137A EA 201891137 A EA201891137 A EA 201891137A EA 201891137 A1 EA201891137 A1 EA 201891137A1
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Prior art keywords
gene therapy
hemophilia
options
viral vectors
increased expression
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EA201891137A
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English (en)
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EA038288B1 (ru
Inventor
Фалько-Гюнтер Фалькнер
Франциска Хорлинг
Йоханнес Ленглер
Ханспетер Роттенштайнер
Фридрих Шайфлингер
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Баксалта Инкорпорейтед
Баксалта Гмбх
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Publication of EA201891137A1 publication Critical patent/EA201891137A1/ru
Publication of EA038288B1 publication Critical patent/EA038288B1/ru

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    • C—CHEMISTRY; METALLURGY
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    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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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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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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Abstract

Данное описание предлагает, среди прочего, полинуклеотиды с измененными кодонами, кодирующие варианты фактора VIII для экспрессии в клетках млекопитающих. В некоторых вариантах реализации изобретения в описании также представлены векторы для генной терапии млекопитающих и способы лечения гемофилии A.
EA201891137A 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a EA038288B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562255317P 2015-11-13 2015-11-13
PCT/US2016/061684 WO2017083762A1 (en) 2015-11-13 2016-11-11 Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a

Publications (2)

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EA201891137A1 true EA201891137A1 (ru) 2018-12-28
EA038288B1 EA038288B1 (ru) 2021-08-05

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EA202190827A EA202190827A1 (ru) 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a
EA201891137A EA038288B1 (ru) 2015-11-13 2016-11-11 Вирусные векторы, кодирующие рекомбинантные варианты fviii с повышенной экспрессией для генной терапии гемофилии a

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US (4) US10189888B2 (ru)
EP (1) EP3374387A1 (ru)
JP (4) JP6768798B2 (ru)
KR (2) KR20230008256A (ru)
CN (3) CN108884145B (ru)
AU (4) AU2016353353B2 (ru)
BR (1) BR112018009717B1 (ru)
CA (1) CA3005334A1 (ru)
CL (1) CL2018001299A1 (ru)
CO (1) CO2018005377A2 (ru)
EA (2) EA202190827A1 (ru)
IL (3) IL305526A (ru)
MX (1) MX2018005982A (ru)
MY (1) MY190067A (ru)
SG (2) SG10202106307UA (ru)
TW (3) TW202302627A (ru)
WO (1) WO2017083762A1 (ru)
ZA (2) ZA201803443B (ru)

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