HRP20231183T1 - Optimizirani gen faktora viii - Google Patents

Optimizirani gen faktora viii Download PDF

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Publication number
HRP20231183T1
HRP20231183T1 HRP20231183TT HRP20231183T HRP20231183T1 HR P20231183 T1 HRP20231183 T1 HR P20231183T1 HR P20231183T T HRP20231183T T HR P20231183TT HR P20231183 T HRP20231183 T HR P20231183T HR P20231183 T1 HRP20231183 T1 HR P20231183T1
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HR
Croatia
Prior art keywords
nucleotide sequence
seq
vector
nucleic acid
acid molecule
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Application number
HRP20231183TT
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English (en)
Inventor
Siyuan Tan
Robert T. Peters
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Bioverativ Therapeutics Inc.
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Application filed by Bioverativ Therapeutics Inc. filed Critical Bioverativ Therapeutics Inc.
Publication of HRP20231183T1 publication Critical patent/HRP20231183T1/hr

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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/30—Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/31—Fusion polypeptide fusions, other than Fc, for prolonged plasma life, e.g. albumin

Claims (15)

1. Izolirana molekula nukleinske kiseline koja sadrži nukleotidnu sekvencu koja je najmanje 95%, najmanje 96%, najmanje 97%, najmanje 98%, najmanje 99%, ili 100% identična sa SEQ ID NO:2, pri čemu nukleotidna sekvenca kodira polipeptid s aktivnošću faktora VIII.
2. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 1, naznačena time što nukleotidna sekvenca sadrži SEQ ID NO:2.
3. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 1 ili 2, naznačena time što nukleotidna sekvenca kodira FVIII s izbrisanom B domenom.
4. Izolirana molekula nukleinske kiseline prema bilo kojem od zahtjeva 1 do 3, naznačena time što nukleotidna sekvenca ima jednu ili više od sljedećih karakteristika: (a) nukleotidna sekvenca sadrži veći postotak G/C nukleotida u usporedbi sa SEQ ID NO:3; (b) nukleotidna sekvenca sadrži manje MARS/ARS sekvenci (SEQ ID NO:5 i SEQ ID NO:6) u usporedbi sa SEQ ID NO:3; (c) nukleotidna sekvenca ne sadrži mjesto spajanja GGTGAT (SEQ ID NO:7); (d) nukleotidna sekvenca sadrži manje destabilizirajućih elemenata (SEQ ID NO:8 i SEQ ID NO:9) u odnosu na SEQ ID NO:3; (e) nukleotidna sekvenca ne sadrži poli-T sekvencu (SEQ ID NO:10); (f) nukleotidna sekvenca ne sadrži poli-A sekvencu (SEQ ID NO:11); ili (g) indeks adaptacije humanog kodona je povećan u odnosu na SEQ ID NO:3.
5. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 4, naznačena time što (a) indeks adaptacije humanog kodona je najmanje oko 0,75, najmanje oko 0,76, najmanje oko 0,77, najmanje oko 0,78, najmanje oko 0,79, najmanje oko 0,80, najmanje oko 0,81, najmanje oko 0,82, najmanje oko 0,83, najmanje oko 0,84, najmanje oko 0,85, najmanje oko 0,86, najmanje oko 0,87, ili najmanje oko 0,88; (b) postotak G/C nukleotida je najmanje oko 45%, najmanje oko 46%, najmanje oko 47%, najmanje oko 48%, najmanje oko 49%, ili najmanje oko 50%; (c) nukleotidna sekvenca: (i) sadrži najviše jednu MARS/ARS sekvencu; ili (ii) ne sadrži MARS/ARS sekvencu; i/ili (d) nukleotidna sekvenca: (i) sadrži najviše 4 destabilizirajuća elementa; (ii) sadrži najviše 2 destabilizirajuća elementa; ili (iii) ne sadrži destabilizirajući element.
6. Izolirana molekula nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1-5, naznačena time što nadalje sadrži heterolognu nukleotidnu sekvencu.
7. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 6, naznačena time što je heterologna nukleotidna sekvenca kodira heterolognu aminokiselinsku sekvencu koja produživač vremena poluraspada.
8. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 7, naznačena time što je heterologna aminokiselinska sekvenca konstantna regija imunoglobulina ili njezin dio, transferin, albumin, ili PAS sekvenca; izborno pri čemu heterologna aminokiselinska sekvenca je Fc regija.
9. Izolirana molekula nukleinske kiseline prema patentnom zahtjevu 8, naznačena time što je heterologna aminokiselinska sekvenca povezana s N-terminalnim krajem ili C-terminalnim krajem aminokiselinske sekvence kodirane nukleotidnom sekvencom ili umetnuta između dvije aminokiseline u aminokiselinskoj sekvenci kodiranoj nukleotidnom sekvencom.
10. Vektor naznačen time što sadrži molekulu nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1-9.
11. Vektor prema patentnom zahtjevu 10, naznačen time što je vektor adenovirusni vektor, lentivirusni vektor, bakulovirusni vektor, vektor virusa Epstein Barr, papovavirusni vektor, vektor vaccinia virusa, vektor virusa herpes simplex, ili vektor adeno povezanog virusa (AAV).
12. Stanica domaćina koja sadrži molekulu nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1-9 ili vektor prema zahtjevu 10.
13. Polipeptid kodiran molekulom nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1-9 ili vektorom iz zahtjeva 10 ili proizveden pomoću stanice domaćina prema zahtjevu 12.
14. Postupak za proizvodnju polipeptida s aktivnošću faktora VIII, naznačen time što obuhvaća: uzgoj stanice domaćina prema zahtjevu 12 pod uvjetima u kojima se proizvodi polipeptid s aktivnošću faktora VIII; te ponovno dobivanje polipeptida s aktivnošću faktora VIII.
15. Izolirana molekula nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1-9, vektor iz zahtjeva 10, ili polipeptid iz zahtjeva 13 naznačen time što se koristi u liječenju poremećaja krvarenja kod subjekta kojem je to potrebno; izborno pri čemu je poremećaj krvarenja hemofilija.
HRP20231183TT 2013-02-15 2014-02-14 Optimizirani gen faktora viii HRP20231183T1 (hr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361765626P 2013-02-15 2013-02-15
EP20204866.6A EP3889173B1 (en) 2013-02-15 2014-02-14 Optimized factor viii gene

Publications (1)

Publication Number Publication Date
HRP20231183T1 true HRP20231183T1 (hr) 2024-01-05

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ID=51354568

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HRP20231183TT HRP20231183T1 (hr) 2013-02-15 2014-02-14 Optimizirani gen faktora viii
HRP20210185TT HRP20210185T1 (hr) 2013-02-15 2021-02-02 Optimizirani gen faktora viii

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Country Status (13)

Country Link
US (3) US10370431B2 (hr)
EP (3) EP4223772A3 (hr)
DK (2) DK2956477T4 (hr)
ES (2) ES2959747T3 (hr)
FI (2) FI2956477T4 (hr)
HR (2) HRP20231183T1 (hr)
HU (2) HUE063051T2 (hr)
LT (2) LT3889173T (hr)
PL (2) PL2956477T3 (hr)
PT (2) PT2956477T (hr)
RS (2) RS64664B1 (hr)
SI (2) SI2956477T1 (hr)
WO (1) WO2014127215A1 (hr)

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