公开(公告)号：US20240358857A1

公开(公告)日：2024-10-31

申请号：US18559078

申请日：2022-05-11

申请人： REGENXBIO INC.

发明人： Olivier Danos ,  SunJung Kim ,  Nicolas Buss ,  Michele Fiscella ,  Ye Liu ,  Chunping Qiao

IPC分类号： A61K48/00 ,  A61K38/17 ,  A61K45/06 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K38/1709 ,  A61K45/06 ,  C12N15/86 ,  C12N2750/14143

摘要： Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin in combination with a second therapeutic which is effective to treat or ameliorate the symptoms of the dystrophinopathy.

公开(公告)号：US20240216543A1

公开(公告)日：2024-07-04

申请号：US18557155

申请日：2022-04-26

申请人： REGENXBIO INC.

发明人： Olivier Danos ,  SunJung Kim ,  Nicholas Buss ,  Ye Liu ,  Chunping Qiao ,  Michele Fiscella ,  Hiren Patel

IPC分类号： A61K48/00 ,  A61K9/00 ,  A61K31/436 ,  A61K31/573 ,  A61K38/17 ,  A61K39/395 ,  A61P21/00 ,  C12N15/86

CPC分类号： A61K48/0058 ,  A61K9/0019 ,  A61K31/436 ,  A61K31/573 ,  A61K38/177 ,  A61K39/3955 ,  A61K48/0033 ,  A61P21/00 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/008 ,  C12N2830/50

摘要： Provided are methods of treating or ameliorating the symptoms of dystrophinopathies, such as Duchenne muscular dystrophy and Becker muscular dystrophy by administration of therapeutically effective doses of recombinant adeno-associated viruses (rAAV) containing a transgene encoding a microdystrophin.