公开(公告)号：US20170216408A1

公开(公告)日：2017-08-03

申请号：US15462660

申请日：2017-03-17

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Xavier ANGUELA ,  Sam Hsien-I SHEN

IPC: A61K38/37 ,  A61K45/06 ,  C07K14/755 ,  A61K48/00 ,  C12N15/86 ,  C12N7/00

CPC classification number: C07K14/755 ,  A61K38/37 ,  A61K45/06 ,  A61K48/00 ,  A61K48/0066 ,  C12N7/00 ,  C12N15/86 ,  C12N2710/10342 ,  C12N2710/10343 ,  C12N2750/14122 ,  C12N2750/14143 ,  C12N2750/14145

Abstract: CpG reduced nucleic acid variants encoding FVIII protein and methods of use thereof are disclosed. In particular embodiments, CpG reduced nucleic acid variants encoding FVIII are expressed more efficiently by cells, are secreted at increased levels by cells over wild-type Factor VIII proteins, exhibit enhanced expression and/or activity over wild-type Factor VIII proteins or are packaged more efficiently into viral vectors.