公开(公告)号：US11698377B2

公开(公告)日：2023-07-11

申请号：US16325653

申请日：2017-08-14

申请人： Genzyme Corporation

发明人： Xiaoying Jin ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC分类号： G01N33/68 ,  C12N15/86 ,  G01N30/72 ,  G01N30/02

CPC分类号： G01N33/6848 ,  C12N15/86 ,  G01N30/72 ,  C12N2750/14123 ,  C12N2750/14143 ,  G01N2030/027

摘要： Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

公开(公告)号：US20230365946A1

公开(公告)日：2023-11-16

申请号：US18186731

申请日：2023-03-20

申请人： Genzyme Corporation

发明人： Thomas M. Wasylenko ,  Kevin Brower ,  Xiaoying Jin

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

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

摘要： 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).

公开(公告)号：US12123880B2

公开(公告)日：2024-10-22

申请号：US18321542

申请日：2023-05-22

申请人： Genzyme Corporation

发明人： Xiaoying Jin ,  Catherine O'Riordan ,  Lin Liu ,  Kate Zhang

IPC分类号： G01N33/68 ,  C12N15/86 ,  G01N30/72 ,  G01N30/02

CPC分类号： G01N33/6848 ,  C12N15/86 ,  G01N30/72 ,  C12N2750/14123 ,  C12N2750/14143 ,  G01N2030/027

摘要： Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.

公开(公告)号：US20210261625A1

公开(公告)日：2021-08-26

申请号：US17162356

申请日：2021-01-29

申请人： GENZYME CORPORATION

发明人： Amy Frederick ,  Xiaoying Jin ,  Lin Liu ,  Catherine O'Riordan ,  Jennifer Sullivan

IPC分类号： C07K14/005 ,  C12N15/86

摘要： Provided herein are modified adeno-associated viral (AAV) capsid proteins, compositions (e.g., rAAV) comprising the capsid proteins, and nucleic acids encoding the capsid proteins. The AAV capsids provided herein confer retinal cell tropism and/or corneal cell tropism, and mediate improved transduction efficiency in clinically relevant ocular cell types such as photoreceptors and/or corneal endothelial cells. Also provided are nucleic acids encoding the capsid proteins, and AAV particles comprising the capsid proteins.