Wave Life Sciences Ltd.

Prashant Monian

Chandra Vargeese

Genliang Lu

Naoki Iwamoto

Subramanian Marappan

David Charles Donnell Butler

Jason Jingxin Zhang

Pachamuthu Kandasamy

Sethumadhavan Divakaramenon

Gopal Reddy Bommineni

Hailin Yang

Mamoru Shimizu

Chikdu Shakti Shivalila

Jayakanthan Kumarasamy

Ann Fiegen Durbin

Selvi Ramasamy

Nayantara Kothari

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.

Oligonucleotide compositions and methods of use thereof

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Arlington, MA, United States of America

Prashant Monian