公开(公告)号：US20240110201A1

公开(公告)日：2024-04-04

申请号：US18262959

申请日：2022-01-27

Applicant: Spark Therapeutics, Inc.

Inventor： Christopher Riling ,  Francis Pankowicz ,  Sean Armour ,  William John Quinn, III ,  Michael Preston ,  Stephen Ioele ,  Daniel Cohen ,  Zhenwei Kelvin See

IPC: C12N15/864 ,  C12N15/62

CPC classification number: C12N15/8645 ,  C12N15/625 ,  C12N2830/001 ,  C12N2830/50

Abstract: Nucleic acids encoding C1 inhibitor are described. Also described are expression cassettes, vectors, cells, and cell lines containing the nucleic acids, as well as methods of using the nucleic acids to treat complement-mediated disorders, such as hereditary angioedema.

公开(公告)号：US20240301380A1

公开(公告)日：2024-09-12

申请号：US18261285

申请日：2022-01-13

Applicant: Spark Therapeutics, Inc.

Inventor： Sean Armour ,  Daniel Cohen ,  Christopher Riling

IPC: C12N9/40 ,  A61K48/00 ,  A61P3/00 ,  C12N15/86

CPC classification number: C12N9/2465 ,  A61K48/0058 ,  A61P3/00 ,  C12N15/86 ,  C07K2319/02 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/15 ,  C12N2830/42 ,  C12N2830/50 ,  C12Y302/01022

Abstract: Polynucleotides including nucleic acids encoding α-galactosidase A (GLA) are described. Also described are expression cassettes, vectors, cells, and cell lines containing the polynucleotides, as well as methods of using the polynucleotides to treat lysosomal storage disorders such as Fabry disease.