专利检索 ap:("Vertex Pharmaceuticals Incorporated") AND inv:"John Patrick Leonard" 第 1 页

1.

发明公开
Compositions and Methods for Treatment of DM1 with SLUCAS9 and SACAS9 审中-公开

公开(公告)号：US20230414648A1

公开(公告)日：2023-12-28

申请号：US18035631

申请日：2021-11-05

申请人： Vertex Pharmaceuticals Incorporated

发明人： John Patrick Leonard , Zachary Michael Detwiler , Peter Thomas Hallock , Eric Gunnar Anderson

IPC分类号： A61K31/7105 , C12N15/11 , C12N9/22 , C12N15/90 , A61K38/46

CPC分类号： A61K31/7105 , C12N15/11 , C12N9/22 , C12N15/907 , A61K38/465 , C12N2310/20 , C12N2800/80

摘要： Compositions and methods for treating myotonic dystrophy type 1 (DM1) with SluCas9 and SaCas9 are encompassed.

2.

发明申请
Compositions and Methods for Treatment of Disorders Associated with Repetitive DNA 有权

公开(公告)号：US20220186216A1

公开(公告)日：2022-06-16

申请号：US17681138

申请日：2022-02-25

申请人： Vertex Pharmaceuticals Incorporated

发明人： Gregoriy Aleksandrovich Dokshin , Matthias Heidenreich , Norzehan Abdul-Manan , Lu Gan , Jianming Liu , Guoxiang Ruan , Jesper Gromada , John Patrick Leonard , Zachary Michael Detwiler , Peter Thomas Hallock , David Esopi , Giselle Dominguez Gutierrez

IPC分类号： C12N15/11 , C12N9/22 , C12N15/113

摘要： Compositions and methods for treating excising trinucleotide repeats, as well as for treating diseases and disorders associated with trinucleotide repeats are encompassed.