Invention Grant
- Patent Title: Oligonucleotide compositions and methods thereof
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Application No.: US16755544Application Date: 2018-10-12
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Publication No.: US11596646B2Publication Date: 2023-03-07
- Inventor: Jason Jingxin Zhang , Chikdu Shakti Shivalila , Chandra Vargeese , Naoki Iwamoto
- Applicant: WAVE LIFE SCIENCES LTD.
- Applicant Address: SG Singapore
- Assignee: WAVE LIFE SCIENCES LTD.
- Current Assignee: WAVE LIFE SCIENCES LTD.
- Current Assignee Address: SG Singapore
- Agency: Choate, Hall & Stewart LLP
- Agent Xiaodong Li; Dustin K. Goncharoff
- International Application: PCT/US2018/055653 WO 20181012
- International Announcement: WO2019/075357 WO 20190418
- Main IPC: C07H21/04
- IPC: C07H21/04 ; A61K31/7125 ; C12N15/113

Abstract:
Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).
Public/Granted literature
- US20210228615A1 OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF Public/Granted day:2021-07-29
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