- Patent Title: Materials and methods for treatment of Duchenne Muscular Dystrophy
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Application No.: US17748495Application Date: 2022-05-19
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Publication No.: US12053531B2Publication Date: 2024-08-06
- Inventor: Ami Meda Kabadi , Chad Albert Cowan , Ante Sven Lundberg
- Applicant: Vertex Pharmaceuticals Incorporated
- Applicant Address: US MA Boston
- Assignee: Vertex Pharmaceuticals Incorporated
- Current Assignee: Vertex Pharmaceuticals Incorporated
- Current Assignee Address: US MA Boston
- Agency: McNeill Baur PLLC
- The original application number of the division: US15763328
- Main IPC: C12N15/63
- IPC: C12N15/63 ; A61K38/46 ; A61K48/00 ; A61P21/00 ; A61P25/14 ; C12N9/22 ; C12N15/113 ; C12N15/90
Abstract:
The present application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, the present application provides materials and methods for editing a dystrophin gene in a cell by genome editing.
Public/Granted literature
- US20230181766A1 Materials and Methods for Treatment of Duchenne Muscular Dystrophy Public/Granted day:2023-06-15
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