US 12,168,776 B2
Nucleic acid molecules and uses thereof
Alexey Seregin, Westford, MA (US); Tongyao Liu, Lexington, MA (US); Susannah Patarroyo-White, Waltham, MA (US); Douglas Drager, Waltham, MA (US); Robert T. Peters, Needham, MA (US); and Jiayun Liu, Wellesley, MA (US)
Assigned to BIOVERATIV THERAPEUTICS INC., Waltham, MA (US)
Appl. No. 16/636,583
Filed by BIOVERATIV THERAPEUTICS INC., Waltham, MA (US)
PCT Filed Aug. 9, 2018, PCT No. PCT/US2018/046110
§ 371(c)(1), (2) Date Feb. 4, 2020,
PCT Pub. No. WO2019/032898, PCT Pub. Date Feb. 14, 2019.
Claims priority of provisional application 62/543,297, filed on Aug. 9, 2017.
Prior Publication US 2021/0163986 A1, Jun. 3, 2021
Int. Cl. C12N 15/00 (2006.01); C07K 14/755 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/86 (2013.01) [C07K 14/755 (2013.01); C12N 2750/14143 (2013.01)] 13 Claims
 
1. A capsid-free system capable of producing closed-end DNA (ceDNA) comprising a first inverted terminal repeat (ITR), a second ITR, and a genetic cassette;
wherein the first ITR and the second ITR are ITRs of an erythrovirus parvovirus B19 or a goose parvovirus (GPV), wherein the genetic cassette is positioned between the first ITR and the second ITR, and wherein the genetic cassette encodes a Factor VIII (FVIII) protein comprising an amino acid sequence at least 90% identical to the amino acid sequence of SEQ ID NO: 106 or 109.