HRP20210185T1 - Optimizirani gen faktora viii - Google Patents

Optimizirani gen faktora viii Download PDF

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Publication number
HRP20210185T1
HRP20210185T1 HRP20210185TT HRP20210185T HRP20210185T1 HR P20210185 T1 HRP20210185 T1 HR P20210185T1 HR P20210185T T HRP20210185T T HR P20210185TT HR P20210185 T HRP20210185 T HR P20210185T HR P20210185 T1 HRP20210185 T1 HR P20210185T1
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HR
Croatia
Prior art keywords
nucleotide sequence
vector
nucleic acid
acid molecule
seq
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HRP20210185TT
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English (en)
Inventor
Siyuan Tan
Robert T. Peters
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Bioverativ Therapeutics Inc.
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=51354568&utm_source=***_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20210185(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Bioverativ Therapeutics Inc. filed Critical Bioverativ Therapeutics Inc.
Publication of HRP20210185T1 publication Critical patent/HRP20210185T1/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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Claims (15)

1. Molekula nukleinske kiseline koja sadrži nukleotidnu sekvencu koja je najmanje 85%, 90%, 95%, 96%, 97%, 98%, 99%, ili 100% identična sa SEQ ID NO:1, pri čemu nukleotidna sekvenca kodira polipeptid s aktivnošću faktora VIII.
2. Molekula nukleinske kiseline prema patentnom zahtjevu 1, naznačena time što nukleotidna sekvenca kodira B domenu izbrisanog FVIII.
3. Molekula nukleinske kiseline prema patentnim zahtjevima 1 ili 2, 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 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.
4. Molekula nukleinske kiseline prema patentnom zahtjevu 3, 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 sadrži najviše jednu MARS/ARS sekvencu; ili (d) nukleotidna sekvenca sadrži najviše 4 destabilizirajuća elementa.
5. Molekula nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1 do 4, naznačena time što nadalje sadrži heterolognu nukleotidnu sekvencu.
6. Molekula nukleinske kiseline prema patentnom zahtjevu 5, naznačena time što je heterologna nukleotidna sekvenca kodira heterolognu aminokiselinsku sekvencu koja produživač vremena poluraspada.
7. Molekula nukleinske kiseline prema patentnom zahtjevu 6, naznačena time što je heterologna aminokiselinska sekvenca konstantna regija imunoglobulina ili njezin dio, transferin, albumin, ili PAS sekvenca.
8. Molekula nukleinske kiseline prema patentnom zahtjevu 7, naznačena time što je heterologna aminokiselinska sekvenca Fc regija.
9. Molekula nukleinske kiseline prema patentnom zahtjevu 1, naznačena time što nukleotidna sekvenca sadrži SEQ ID NO:1.
10. Vektor naznačen time što sadrži molekulu nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1 do 9.
11. Vektor prema patentnom zahtjevu 10, naznačen time što je vektor odabran od adenovirusnog vektora, lentivirusnog vektora, bakulovirusnog vektora, Epstein Barr virusnog vektora, papovavirusnog vektora, virusnog vektora vaccinia, virusnog vektora herpes simplex, i vektora adeno povezanog virusa (AAV).
12. Stanica domaćina koja sadrži molekulu nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1 do 9 ili vektor prema zahtjevu 10 ili 11.
13. Polipeptid kodiran molekulom nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1 do 9 ili vektorom iz zahtjeva 10 ili 11 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. Molekula nukleinske kiseline prema bilo kojem od patentnih zahtjeva 1 do 9, vektor iz zahtjeva 10 ili 11, ili polipeptid iz zahtjeva 13 naznačen time što se koristi u liječenju poremećaja krvarenja kod subjekta kojem je to potrebno.
HRP20210185TT 2013-02-15 2021-02-02 Optimizirani gen faktora viii HRP20210185T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361765626P 2013-02-15 2013-02-15
PCT/US2014/016441 WO2014127215A1 (en) 2013-02-15 2014-02-14 Optimized factor viii gene
EP14751254.5A EP2956477B2 (en) 2013-02-15 2014-02-14 Optimized factor viii gene

Publications (1)

Publication Number Publication Date
HRP20210185T1 true HRP20210185T1 (hr) 2021-03-19

Family

ID=51354568

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications Before (1)

Application Number Title Priority Date Filing Date
HRP20231183TT HRP20231183T1 (hr) 2013-02-15 2014-02-14 Optimizirani gen faktora viii

Country Status (13)

Country Link
US (3) US10370431B2 (hr)
EP (3) EP2956477B2 (hr)
DK (2) DK2956477T4 (hr)
ES (2) ES2959747T3 (hr)
FI (2) FI3889173T3 (hr)
HR (2) HRP20231183T1 (hr)
HU (2) HUE063051T2 (hr)
LT (2) LT2956477T (hr)
PL (2) PL3889173T3 (hr)
PT (2) PT2956477T (hr)
RS (2) RS61387B1 (hr)
SI (2) SI2956477T1 (hr)
WO (1) WO2014127215A1 (hr)

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