Wave Life Sciences Ltd.

Hailin Yang

Chandra Vargeese

Maria Frank-Kamenetsky

Genliang Lu

Naoki Iwamoto

Subramanian Marappan

David Charles Donnell Butler

Jason Jingxin Zhang

Sethumadhavan Divakaramenon

Christopher J Francis

Gregory Lawrence Verdine

Meena

Pachamuthu Kandasamy

Gopal Reddy Bommineni

Aaron Jay Morris

Mamoru Shimizu

Chikdu Shakti Shivalila

Jayakanthan Kumarasamy

Ann Fiegen Durbin

Selvi Ramasamy

Nayantara Kothari

Prashant Monian

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

Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

Oligonucleotide compositions and methods thereof

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West Roxbury, MA, United States of America

Hailin Yang