AU2016232146B2 - Optimized liver-specific expression systems for FVIII and FIX - Google Patents

Optimized liver-specific expression systems for FVIII and FIX Download PDF

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AU2016232146B2
AU2016232146B2 AU2016232146A AU2016232146A AU2016232146B2 AU 2016232146 B2 AU2016232146 B2 AU 2016232146B2 AU 2016232146 A AU2016232146 A AU 2016232146A AU 2016232146 A AU2016232146 A AU 2016232146A AU 2016232146 B2 AU2016232146 B2 AU 2016232146B2
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seq
promoter
nucleic acid
vector
mvm
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AU2016232146A1 (en
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Marinee Chuah
Thierry Vandendriessche
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Vrije Universiteit Brussel VUB
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    • 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
    • A—HUMAN NECESSITIES
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    • 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
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AU2016232146A 2015-03-17 2016-03-17 Optimized liver-specific expression systems for FVIII and FIX Ceased AU2016232146B2 (en)

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EP15159395.1 2015-03-17
EP15159395 2015-03-17
PCT/EP2016/055825 WO2016146757A1 (en) 2015-03-17 2016-03-17 Optimized liver-specific expression systems for fviii and fix

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AU2016232146A1 AU2016232146A1 (en) 2017-10-12
AU2016232146B2 true AU2016232146B2 (en) 2021-11-04

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US (2) US11007280B2 (cg-RX-API-DMAC7.html)
EP (1) EP3270944B1 (cg-RX-API-DMAC7.html)
JP (1) JP6879486B2 (cg-RX-API-DMAC7.html)
AU (1) AU2016232146B2 (cg-RX-API-DMAC7.html)
CA (1) CA2979495A1 (cg-RX-API-DMAC7.html)
DK (1) DK3270944T3 (cg-RX-API-DMAC7.html)
ES (1) ES2764453T3 (cg-RX-API-DMAC7.html)
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US20180071406A1 (en) 2018-03-15
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AU2016232146A1 (en) 2017-10-12
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