Invention Application
- Patent Title: MUSCLE TARGETING COMPLEXES AND FORMULATIONS FOR TREATING DYSTROPHINOPATHIES
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Application No.: US18896790Application Date: 2024-09-25
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Publication No.: US20250057972A1Publication Date: 2025-02-20
- Inventor: Timothy Weeden , Scott Hilderbrand , Sean Spring , Peiyi Shen , Cody A. Desjardins , Romesh R. Subramanian , Mohammed T. Qatanani , Brendan Quinn , John Najim
- Applicant: Dyne Therapeutics, Inc.
- Applicant Address: US MA Waltham
- Assignee: Dyne Therapeutics, Inc.
- Current Assignee: Dyne Therapeutics, Inc.
- Current Assignee Address: US MA Waltham
- Main IPC: A61K47/68
- IPC: A61K47/68 ; A61K47/54 ; A61K47/65 ; A61P21/00

Abstract:
Aspects of the disclosure relate to complexes and other aspects relate to formulations (e.g., aqueous, lyophilized forms) comprising such complexes (e.g., wherein each complex is of the exemplary formula shown below) comprising a phosphorodiamidate morpholino oligomer (e.g., useful for targeting DMD) covalently linked to an antibody (e.g., anti-TfR1 antibody). In some embodiments, the complexes are formulated with histidine (e.g., L-histidine) and sucrose at a specified pH (e.g., about 5.0 to 7.0). Also provided are uses of these formulations for treating a subject having a mutated DMD allele associated with Duchenne Muscular Dystrophy.
Information query
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