公开(公告)号：US20230127775A1

公开(公告)日：2023-04-27

申请号：US17930014

申请日：2022-09-06

申请人： Ultragenyx Pharmaceutical Inc.

发明人： Emil D. KAKKIS ,  Michel Claude Vellard ,  Andrzej Swistowski

IPC分类号： A61K38/48 ,  A61K38/05 ,  A61K38/54 ,  A61K31/198 ,  A61K31/454 ,  A61K31/475 ,  A61K31/573 ,  A61K31/675 ,  A61K31/69 ,  A61K31/704 ,  A61K35/12 ,  A61P25/28 ,  A61K45/06 ,  C12N9/48 ,  C12N9/64

摘要： Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.

公开(公告)号：US20210228694A1

公开(公告)日：2021-07-29

申请号：US17065836

申请日：2020-10-08

申请人： Ultragenyx Pharmaceutical Inc.

发明人： Emil D. Kakkis ,  Michel Claude Vellard ,  Andrzej Swistowski

IPC分类号： A61K38/48 ,  A61K38/05 ,  A61K38/54 ,  A61K31/198 ,  A61K31/454 ,  A61K31/475 ,  A61K31/573 ,  A61K31/675 ,  A61K31/69 ,  A61K31/704 ,  A61K35/12 ,  A61P25/28 ,  A61K45/06 ,  C12N9/48 ,  C12N9/64

摘要： Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.