Peter Cameron Colosi, Novato, CA (US); Amit Nathwani, London (GB); Jenny McIntosh, London (GB); Edward Tuddenham, London (GB); and Andrew Davidoff, Memphis, TN (US)
Assigned to BIOMARIN PHARMACEUTICAL INC., Novato, CA (US); UCL BUSINESS LTD, London (GB); and ST. JUDE CHILDREN'S RESEARCH HOSPITAL, Memphis, TN (US)
Filed by BIOMARIN PHARMACEUTICAL INC., Novato, CA (US); UCL BUSINESS LTD, London (GB); and ST. JUDE CHILDREN'S RESEARCH HOSPITAL, Memphis, TN (US)
Filed on Jun. 30, 2022, as Appl. No. 17/854,286.
Application 17/854,286 is a continuation of application No. 16/588,130, filed on Sep. 30, 2019, granted, now 11,406,690.
Application 16/588,130 is a continuation of application No. 15/294,310, filed on Oct. 14, 2016, granted, now 10,463,718.
Application 15/294,310 is a continuation of application No. 14/482,648, filed on Sep. 10, 2014, granted, now 9,504,762.
Claims priority of provisional application 61/877,042, filed on Sep. 12, 2013.
Prior Publication US 2022/0339262 A1, Oct. 27, 2022
1. An adeno-associated virus (AAV) vector comprising a 5′ inverted terminal repeat (ITR), a liver-specific transcription regulatory region operably linked to a codon-optimized nucleic acid sequence encoding a functionally active Factor VIII (FVIII) protein having the A3 and B domain deleted, and a 3′ ITR, wherein the nucleic acid sequence comprises nucleotides 646-4611 of SEQ ID NO: 5.