The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Aug. 19, 2025
Filed:
May. 10, 2019
Wave Life Sciences Ltd., Singapore, SG;
Jason Jingxin Zhang, Walpole, MA (US);
Chandra Vargeese, Schwenksville, PA (US);
Naoki Iwamoto, Boston, MA (US);
Chikdu Shakti Shivalila, Woburn, MA (US);
Nayantara Kothari, Newton, MA (US);
Ann Fiegen Durbin, Arlington, MA (US);
Selvi Ramasamy, Wayland, MA (US);
Pachamuthu Kandasamy, Belmont, MA (US);
Jayakanthan Kumarasamy, Belmont, MA (US);
Gopal Reddy Bommineni, Belmont, MA (US);
Subramanian Marappan, Acton, MA (US);
Sethumadhavan Divakaramenon, Lexington, MA (US);
David Charles Donnell Butler, Medford, MA (US);
Genliang Lu, Winchester, MA (US);
Hailin Yang, Brighton, MA (US);
Mamoru Shimizu, Arlington, MA (US);
Prashant Monian, Arlington, MA (US);
WAVE LIFE SCIENCES LTD., Singapore, SG;
Abstract
Among other things, the present disclosure provides designed DMD oligonucleotides, compositions, and methods of use thereof. In some embodiments, the present disclosure provides technologies useful for repairing mutant DMD transcripts by skipping exon 51 or exon 53, so that the transcript can be translated into an internally truncated but at least partially functional Dystrophin protein variant. In some embodiments, the present disclosure provides technologies useful for modulating DMD transcript splicing. In some embodiments, provided technologies can alter splicing of a dystrophin (DMD) DMD transcript. In some embodiments, the present disclosure provides methods for treating diseases, such as muscular dystrophy, including but not limited to Duchenne muscular dystrophy, Becker's muscular dystrophy, etc.