CO2021002965A2 - Moléculas de ácido nucleico y sus usos para la terapia génica no viral - Google Patents

Moléculas de ácido nucleico y sus usos para la terapia génica no viral

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Publication number
CO2021002965A2
CO2021002965A2 CONC2021/0002965A CO2021002965A CO2021002965A2 CO 2021002965 A2 CO2021002965 A2 CO 2021002965A2 CO 2021002965 A CO2021002965 A CO 2021002965A CO 2021002965 A2 CO2021002965 A2 CO 2021002965A2
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Colombia
Prior art keywords
itr
nucleic acid
acid molecules
gene therapy
viral gene
Prior art date
Application number
CONC2021/0002965A
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English (en)
Inventor
Douglas Drager
Susannah Patarroyo-White
Jiayun Liu
Tongyao Liu
Robert T Peters
Alexey Seregin
Philip Zakas
Original Assignee
Bioverativ Therapeutics Inc
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Filing date
Publication date
Application filed by Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of CO2021002965A2 publication Critical patent/CO2021002965A2/es

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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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    • 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
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Abstract

La presente divulgación proporciona moléculas de ácido nucleico que comprenden una primera repetición terminal invertida (ITR), una segunda ITR, y un casete genético que codifica una secuencia diana. En algunas realizaciones, la secuencia diana codifica un miARN y/o una proteína terapéutica. En ciertas realizaciones, la proteína terapéutica comprende un factor de coagulación, un factor de crecimiento, una hormona, una citocina, un anticuerpo, un fragmento del mismo, y una combinación de los mismos. En algunas realizaciones, la primera ITR y/o la segunda ITR son una ITR de un virus no adenoasociado (AAV). La presente divulgación también proporciona métodos de tratamiento de un trastorno metabólico del hígado en un sujeto que comprenden administrar al sujeto la molécula de ácido nucleico o un polipéptido codificado de ese modo.
CONC2021/0002965A 2018-08-09 2021-03-04 Moléculas de ácido nucleico y sus usos para la terapia génica no viral CO2021002965A2 (es)

Applications Claiming Priority (2)

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US201862716826P 2018-08-09 2018-08-09
PCT/US2019/045957 WO2020033863A1 (en) 2018-08-09 2019-08-09 Nucleic acid molecules and uses thereof for non-viral gene therapy

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CO2021002965A2 true CO2021002965A2 (es) 2021-03-19

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US (1) US20200069817A1 (es)
EP (1) EP3833766A1 (es)
JP (1) JP2021533753A (es)
KR (1) KR20210042128A (es)
CN (1) CN113227385A (es)
AU (1) AU2019319984A1 (es)
BR (1) BR112021002017A2 (es)
CA (1) CA3108799A1 (es)
CO (1) CO2021002965A2 (es)
IL (1) IL280652A (es)
MX (1) MX2021001599A (es)
PH (1) PH12021500010A1 (es)
SG (1) SG11202101157VA (es)
TW (1) TW202023630A (es)
WO (1) WO2020033863A1 (es)

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WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022355088A1 (en) * 2021-09-30 2024-05-16 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity
CN114250224B (zh) * 2021-12-16 2024-02-27 福建和瑞基因科技有限公司 一种用于提取或检测样本中小分子rna的核酸组合物及其试剂盒和方法
WO2023135273A2 (en) * 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
CN116064673A (zh) * 2022-10-19 2023-05-05 河南省人民医院 表达rs1的重组腺相关病毒载体及其应用

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