HRP20200930T1 - Lentivirusni vektori - Google Patents

Lentivirusni vektori Download PDF

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Publication number
HRP20200930T1
HRP20200930T1 HRP20200930TT HRP20200930T HRP20200930T1 HR P20200930 T1 HRP20200930 T1 HR P20200930T1 HR P20200930T T HRP20200930T T HR P20200930TT HR P20200930 T HRP20200930 T HR P20200930T HR P20200930 T1 HRP20200930 T1 HR P20200930T1
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Croatia
Prior art keywords
lentiviral vector
disease
factor
transgene
virus
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HRP20200930TT
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English (en)
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Eric Walter Frederick Wolfgang ALTON
Uta Griesenbach
Kamila Malgorzata PYTEL
Michael Christian PAUL-SMITH
Ian Andrew PRINGLE
Stephen Charles Hyde
Deborah Rebecca GILL
Lee Adrian Davies
Alan Christopher BOYD
Gerard Mc Lachlan
Makoto Inoue
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Ip2Ipo Innovations Limited
Id Pharma Co., Ltd.
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Application filed by Ip2Ipo Innovations Limited, Id Pharma Co., Ltd. filed Critical Ip2Ipo Innovations Limited
Publication of HRP20200930T1 publication Critical patent/HRP20200930T1/hr

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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
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    • A61K48/0066Manipulation 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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    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
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Claims (13)

1. Lentivirusni vektor, naznačen time, da je pseudotipiziran s hemaglutinin-neuraminidazom (HN) i proteinima fuzije (F) iz respiratornog paramiksovirusa, pri čemu navedeni lentivirusni vektor obuhvaća hibridni ljudski CMV aktivator/EF1a (hCEF)-promotor i jedan transgen, te time, da lentivirusnom vektoru nedostaje jedan (bilo koji) intron koji je pozicioniran između navedenog promotora i navedenog transgena.
2. Lentivirusni vektor prema patentnom zahtjevu 1, naznačen time, da se lentivirusni vektor odabire iz skupine koja se sastoji od vektora ljudskog virusa imunodeficijencije (HIV- Human immunodeficiency virus), vektora majmunskog virusa imunodeficijencije (SIV-Simian immunodeficiency virus), vektora mačjeg virusa imunodeficijencije (FIV-Feline immunodeficiency virus), vektora virusa infektivne anemije porodice konja (EIAV-Equine infectious anaemia virus), i vektora virusa Visna/Maedi; pri čemu je lentivirusni vektor poželjno SIV-vektor.
3. Lentivirusni vektor prema patentnom zahtjevu 1 ili 2, naznačen time, da respiratorni paramiksovirus je virus Sendai.
4. Lentivirusni vektor prema bilo kojem od prethodnih patentnih zahtjeva, naznačen time, da se transgen odabire od sljedećih: (a) sekrecijski terapeutski protein, opcionalno, alfa-1-antitripsin (A1AT), faktor VIII, tenzidni protein B (SFTPB), faktor VII, faktor IX, faktor X, faktor XI, faktor von Willebrand, faktor stimuliranja kolonije granulocita i makrofaga (GM-CSF), ili monoklonsko protutijelo protiv infektivnog agensa; ili (b) CFTR, DNAH5, DNAH11, DNAI1 ili DNAI2.
5. Lentivirusni vektor prema bilo kojem od patentnih zahtjeva 1 do 4, naznačen time, da: (a) transgen kodira CFTR; (b) transgen kodira A1AT; ili (c) transgen kodira FVIII.
6. Lentivirusni vektor prema bilo kojem od patentnih zahtjeva 1 do 5, naznačen time, da transgen kodira CFTR.
7. Postupak proizvodnje lentivirusa definiranog prema bilo kojem od patentnih zahtjeva 1 do 6, naznačen time, da postupak obuhvaća sljedeće korake: (a) uzgajanje stanica u suspenziji; (b) transficiranje stanica s jednim ili više plazmida; (c) dodavanje nukleaze; (d) sakupljanje lentivirusa; (e) dodavanje tripsina; i (f) pročišćavanje.
8. Postupak prema patentnom zahtjevu 7, naznačen time, da: (i) koraci (a) do (f) se provode slijedom uzastopno; (ii) stanice su HEK293T-stanice ili 293T/17-stanice; (iii) dodavanje nukleaze slijedi u stadiju prije sakupljanja; (iv) dodavanje tripsina slijedi u stadiju nakon sakupljanja; i/ili (v) korak pročišćavanja obuhvaća korak kromatografije.
9. Lentivirusni vektor prema bilo kojem od patentnih zahtjeva 1 do 6, naznačen time, da se upotrebljava u postupku liječenja bolesti.
10. Lentivirusni vektor za uporabu prema patentnom zahtjevu 9, naznačen time, da se kod bolesti radi o plućnoj bolesti koja je odabrana od sljedećih: cistična fibroza (CF); primarna cilijarna diskinezija (PCD); pomanjkanje tenzidnog proteina B (SP-B-deficijencija); alfa-1-antitripsin-deficijencija (A1AD); plućna alveolska proteinoza (PAP); i kronična opstruktivna plućna bolest (KOPB).
11. Lentivirusni vektor za uporabu prema patentnom zahtjevu 9, naznačen time, da: (a) bolest koja se treba liječiti je CF; (b) bolest koja se treba liječiti je A1AT-deficijencija; ili (c) bolest koja se treba liječiti je hemofilija, pri čemu, opcionalno, transgen je faktor VIII.
12. Stanica domaćina, naznačena time, da sadrži vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 6.
13. Pripravak, naznačen time, da sadrži vektor u skladu s bilo kojim od patentnih zahtjeva 1 do 6, i farmaceutski prihvatljiv nosač.
HRP20200930TT 2014-05-21 2020-06-10 Lentivirusni vektori HRP20200930T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GB1409089.8A GB2526339A (en) 2014-05-21 2014-05-21 Lentiviral vectors
EP15720762.2A EP3145949B1 (en) 2014-05-21 2015-04-23 Lentiviral vectors
PCT/GB2015/051201 WO2015177501A1 (en) 2014-05-21 2015-04-23 Lentiviral vectors

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HRP20200930T1 true HRP20200930T1 (hr) 2020-09-18

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US (1) US10704061B2 (hr)
EP (1) EP3145949B1 (hr)
JP (1) JP6637444B2 (hr)
AU (1) AU2015263150B2 (hr)
CA (1) CA2947125C (hr)
CY (1) CY1123413T1 (hr)
DK (1) DK3145949T3 (hr)
EA (1) EA038966B1 (hr)
ES (1) ES2805045T3 (hr)
GB (1) GB2526339A (hr)
HR (1) HRP20200930T1 (hr)
HU (1) HUE051269T2 (hr)
LT (1) LT3145949T (hr)
ME (1) ME03789B (hr)
PL (1) PL3145949T3 (hr)
PT (1) PT3145949T (hr)
RS (1) RS60586B1 (hr)
SI (1) SI3145949T1 (hr)
UA (1) UA122127C2 (hr)
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