US 12,257,319 B2
Optimised coding sequence and promoter
Amit Nathwani, London (GB); Natalie Ward, London (GB); Adrian Thrasher, London (GB); Edward Tuddenham, London (GB); John McVey, London (GB); John Gray, Memphis, TN (US); and Andrew Davidoff, Memphis, TN (US)
Assigned to UCL BUSINESS LTD, London (GB); ST. JUDE CHILDREN'S RESEARCH HOSPITAL, Memphis, TN (US); and THROMBOSIS RESEARCH INSTITUTE, London (GB)
Filed by UCL BUSINESS LTD, London (GB); THROMBOSIS RESEARCH INSTITUTE, London (GB); and ST. JUDE CHILDREN'S RESEARCH HOSPITAL, Memphis, TN (US)
Filed on Jun. 10, 2020, as Appl. No. 16/897,900.
Application 14/680,836 is a division of application No. 13/382,953, granted, now 9,393,323, previously published as PCT/US2010/041378, filed on Jul. 8, 2010.
Application 16/897,900 is a continuation of application No. 15/651,698, filed on Jul. 17, 2017, granted, now 10,709,796.
Application 15/651,698 is a continuation of application No. 14/680,836, filed on Apr. 7, 2015, granted, now 9,764,045.
Claims priority of application No. 0911870 (GB), filed on Jul. 8, 2009.
Prior Publication US 2021/0000978 A1, Jan. 7, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. C07H 21/04 (2006.01); A61K 31/70 (2006.01); A61K 38/37 (2006.01); A61K 48/00 (2006.01); C07K 14/755 (2006.01); C12N 7/00 (2006.01); C12N 15/63 (2006.01); C12N 15/86 (2006.01); A01K 67/00 (2006.01); A61K 38/00 (2006.01)
CPC A61K 48/0058 (2013.01) [A61K 38/37 (2013.01); C07K 14/755 (2013.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 38/00 (2013.01); C12N 2710/10041 (2013.01); C12N 2710/14143 (2013.01); C12N 2750/14143 (2013.01); C12N 2800/22 (2013.01); C12N 2830/008 (2013.01)] 20 Claims
 
1. An isolated nucleic acid molecule comprising a functional FVIII coding nucleotide sequence having at least 80% homology to the nucleotide sequence of SEQ ID NO: 5 that is operably linked to a liver specific promoter comprising a nucleotide sequence having at least 95% identity to the nucleotide sequence of SEQ ID NO: 3, and wherein in respect to the amino acid sequence encoded by SEQ ID NO: 5 any amino acid changes encoded by the isolated nucleic acid molecule is in the portion of the amino acid sequence which replaced the B domain of the FVIII protein.