公开(公告)号：US20210371877A1

公开(公告)日：2021-12-02

申请号：US15733246

申请日：2018-12-18

Applicant: SPARK THERAPEUTICS, INC.

Inventor： Andrew William SHAW ,  Xavier ANGUELA

IPC: C12N15/86 ,  A61K9/08 ,  A61K9/107 ,  A61K9/51 ,  C12N15/88

Abstract: A composition includes an adeno-associated vims (AAV) vector in a lipid nanoparticle (LNP). The AAV vector can include a heterologous nucleic acid sequence, optionally an inverted terminal repeat (ITR) positioned 5′ of the heterologous nucleic acid sequence and an ITR positioned 3′ of the heterologous nucleic acid sequence. The AAV vector can further include expression control elements (e.g., a promoter and/or enhancer), and intron, and/or or a polyadenylation signal.