专利检索 ap:("TRANSLATE BIO, INC.") AND inv:"BARBIER, Ann, J." 第 1 页

1.

发明申请
IMPROVED COMPOSITION AND METHODS FOR TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY 审中-公开

公开(公告)号：WO2019126593A1

公开(公告)日：2019-06-27

申请号：PCT/US2018/066970

申请日：2018-12-20

申请人： TRANSLATE BIO, INC.

发明人： ASKEW, Kim , CHUNG, Jou-ku , DEROSA, Frank , HEARTLEIN, Michael , KARVE, Shrirang , MCCAULEY, Thomas , SMITH, Lianne , BARBIER, Ann, J.

IPC分类号： A61K38/45 , A61P3/00

CPC分类号： A61K48/005 , A61K9/0019 , A61K9/1272 , A61K38/45 , A61K47/6929 , A61K48/0025 , A61K48/0075 , A61K48/0083 , A61P3/00 , C12N15/52 , C12N15/85 , C12Y201/03003

摘要： The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.