公开(公告)号：US20230365946A1

公开(公告)日：2023-11-16

申请号：US18186731

申请日：2023-03-20

Applicant: Genzyme Corporation

Inventor： Thomas M. Wasylenko ,  Kevin Brower ,  Xiaoying Jin

IPC: C12N9/16 ,  A61P3/00 ,  B01D15/36 ,  B01D15/38 ,  B01D15/18 ,  B01D15/20

CPC classification number: C12N9/16 ,  C12Y301/04012 ,  A61P3/00 ,  B01D15/362 ,  B01D15/3828 ,  B01D15/1871 ,  B01D15/203 ,  A61K38/00

Abstract: Disclosed here are compositions comprising recombinant acid sphingomyelinase (rASM) having desired purity, specific activity, and/or rASM isoforms. Also provided are methods for making and purifying such compositions, comprising chromatography steps. Further provided are methods of modulating rASM specific activity in a composition, and methods of modulating rASM isoforms in a composition. The methods disclosed here can be particularly useful for manufacturing pharmaceutical compositions comprising rASM for treating acid sphingomyelinase deficiency (ASMD).