Wave Life Sciences Ltd.

Gopal Reddy Bommineni

David Charles Donnell Butler

Pachamuthu Kandasamy

Chandra Vargeese

Subramanian Marappan

Genliang Lu

Naoki Iwamoto

Sethumadhavan Divakaramenon

Mamoru Shimizu

Christopher P Hencken

Ilia Korboukh

Mohammed Rowshon Alam

Keith Andrew Bowman

Snehlata Tripathi

Alvaro Andres Lanao

Vincent Aduda

Stephany Michelle Standley

Jason Jingxin Zhang

Hailin Yang

Chikdu Shakti Shivalila

Ik-Hyeon Paik

Jayakanthan Kumarasamy

Prashant Monian

Ann Fiegen Durbin

Selvi Ramasamy

Nayantara Kothari

Among other things, the present disclosure provides technologies for oligonucleotide preparation, particularly chirally controlled oligonucleotide preparation, which technologies provide greatly improved crude purity and yield, and significantly reduce manufacturing costs.

Technologies for oligonucleotide preparation

The present disclosure, among other things, provides technologies for synthesis, including reagents and methods for stereoselective synthesis. In some embodiments, the present disclosure provides compounds useful as chiral auxiliaries. In some embodiments, the present disclosure provides reagents and methods for oligonucleotide synthesis. In some embodiments, the present disclosure provides reagents and methods for chirally controlled preparation of oligonucleotides. In some embodiments, technologies of the present disclosure are particularly useful for constructing challenging internucleotidic linkages, providing high yields and stereoselectivity.

Compounds, compositions and methods for synthesis

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

The present disclosure, among other things, provides technologies for oligonucleotide synthesis. In some embodiments, the present disclosure provides phosphoramidites and methods for synthesis thereof. In some embodiments, provided methods provides higher yields and/or purities. In some embodiments, provided methods remove byproducts without contact with an aqueous solution.

Compositions and methods for phosphoramidite and oligonucleotide synthesis

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Belmont, MA, United States of America

Gopal Reddy Bommineni