公开(公告)号：US10682423B2

公开(公告)日：2020-06-16

申请号：US15990463

申请日：2018-05-25

申请人： GENZYME CORPORATION

发明人： Carol A. Nelson ,  Bruce M. Wentworth ,  Ronald K. Scheule ,  Timothy E. Weeden ,  Nicholas P. Clayton

IPC分类号： A61K48/00 ,  C07H21/04 ,  C12N15/113

摘要： The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.

公开(公告)号：US11103587B2

公开(公告)日：2021-08-31

申请号：US16265883

申请日：2019-02-01

申请人： Genzyme Corporation

发明人： Timothy E. Weeden ,  Carol A. Nelson ,  Bruce M. Wentworth ,  Nicholas P. Clayton ,  Andrew Leger

IPC分类号： A61K47/54 ,  C12N15/11

摘要： The present disclosure relates to antisense oligonucleotides (AONs), such as phosphorodiamidate morpholino oligonucleotides (PMOs). The present disclosure further relates to the conjugation of multiple PMOs to cationic cell penetrating peptides (CPPs) to enhance the uptake of PMOs into skeletal and cardiac muscle cells.

公开(公告)号：US11801311B2

公开(公告)日：2023-10-31

申请号：US16867261

申请日：2020-05-05

申请人： Genzyme Corporation

发明人： Carol A. Nelson ,  Bruce M. Wentworth ,  Ronald K. Scheule ,  Timothy E. Weeden ,  Nicholas P. Clayton

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  C12N15/113

CPC分类号： A61K48/005 ,  C12N15/113 ,  C12N15/1137 ,  C12Y204/01011 ,  C12N2310/11 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/33

摘要： The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.