公开(公告)号：EP3146050B1

公开(公告)日：2020-08-12

申请号：EP15727794.8

申请日：2015-05-22

申请人： Genzyme Corporation

发明人： NELSON, Carol A. ,  WENTWORTH, Bruce M. ,  SCHEULE, Ronald K. ,  WEEDEN, Timothy E. ,  CLAYTON, Nicholas P.

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7105

公开(公告)号：EP3795687A1

公开(公告)日：2021-03-24

申请号：EP20190340.8

申请日：2015-05-22

申请人： Genzyme Corporation

发明人： NELSON, Carol A. ,  WENTWORTH, Bruce M. ,  SCHEULE, Ronald K. ,  WEEDEN, Timothy E. ,  CLAYTON, Nicholas P.

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7105

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：EP3146050A1

公开(公告)日：2017-03-29

申请号：EP15727794.8

申请日：2015-05-22

申请人： Genzyme Corporation

发明人： NELSON, Carol A. ,  WENTWORTH, Bruce M. ,  SCHEULE, Ronald K. ,  WEEDEN, Timothy E. ,  CLAYTON, Nicholas P.

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7105

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.

摘要翻译： 本发明涉及反义寡核苷酸（AON），用于调制糖原合成酶的表达。 本发明的AON可用于治疗与酶糖原合酶,:如蓬珀病的表达的调节相关的疾病是有用的。 因此，通过本发明提供了包含AON，以及向下的方法调节mRNA编码的糖原合酶，减少在骨骼肌和心肌糖原合成酶的方法，以及治疗庞贝氏症的方法。

公开(公告)号：EP3151841A1

公开(公告)日：2017-04-12

申请号：EP15727795.5

申请日：2015-05-22

申请人： Genzyme Corporation

发明人： WEEDEN, Timothy E. ,  NELSON, Carol A. ,  WENTWORTH, Bruce M. ,  CLAYTON, Nicholas P. ,  LEGER, Andrew

IPC分类号： A61K31/712 ,  C07K19/00 ,  C12N15/113 ,  A61K47/50

CPC分类号： A61K47/549 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/3145 ,  C12N2310/3233 ,  C12N2310/3513 ,  C12N2320/32

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.

摘要翻译： 本公开涉及反义寡核苷酸（AON），例如磷酰二胺吗啉代寡核苷酸（PMO）。 本公开还涉及多个PMO与阳离子细胞穿透肽（CPP）的缀合，以增强PMO到骨骼和心肌细胞中的摄取。