Invention Grant
- Patent Title: Methods of delivering an oligonucleotide to a subject having facioscapulohumeral muscular dystrophy
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Application No.: US17846738Application Date: 2022-06-22
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Publication No.: US11518816B2Publication Date: 2022-12-06
- Inventor: Romesh R. Subramanian , Mohammed T. Qatanani , Timothy Weeden
- Applicant: Dyne Therapeutics, Inc.
- Applicant Address: US MA Waltham
- Assignee: Dyne Therapeutics, Inc.
- Current Assignee: Dyne Therapeutics, Inc.
- Current Assignee Address: US MA Waltham
- Agency: Wolf, Greenfield & Sacks, P.C.
- Main IPC: C07K16/28
- IPC: C07K16/28 ; C12N15/113

Abstract:
Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload inhibits expression or activity of DUX4. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.
Public/Granted literature
- US20220324992A1 MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY Public/Granted day:2022-10-13
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