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US 12,728,171 B2Granted
Complement factor I and complement factor I cofactor, vectors encoding therefor and therapeutic use
AssigneeNovartis Pharmaceuticals UK Limited
InventorsAnna Dreismann, Scott Ellis, Josephine Heather Lucienne Joel
Life of this patent
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Abstract
A product comprising (i) a Complement Factor I (CFI) cofactor; and (ii) Complement Factor I (CFI), or nucleotide sequences encoding therefor, as a combined preparation for simultaneous, separate or sequential use in therapy.
Claims13
An isolated polynucleotide comprising nucleotide sequences encoding (i) a Complement Factor I (CFI) cofactor;
- and (ii) Complement Factor I (CFI), wherein the polynucleotide comprises:
- a) a 5′ AAV ITR;
- b) a CMV promoter;
- c) a codon-optimized nucleotide sequence encoding Complement Factor H Like Protein 1 (FHL1);
- d) a linker comprising a self-cleaving 2A peptide sequence;
- e) a codon-optimized nucleotide sequence encoding CFI;
- f) a WPRE3 regulatory element;
- g) a Bovine Growth Hormone poly-A signal;
- and
- h) a 3′ AAV ITR;
- wherein the polynucleotide comprises the nucleotide sequence of SEQ ID NO:
- 22 or 23, or a nucleotide sequence that has at least 95% sequence identity thereto.
9 more elements
Dependencies are inferred from the claim text.