公开(公告)号：US20240148891A1

公开(公告)日：2024-05-09

申请号：US17769467

申请日：2020-10-16

申请人： Dyne Therapeutics, Inc.

发明人： Romesh R. Subramanian ,  Mohammed T. QATANANI ,  Timothy WEEDEN ,  Cody A. DESJARDINS

IPC分类号： A61K47/68 ,  A61P21/00

CPC分类号： A61K47/6849 ,  A61P21/00

摘要： 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 a DMPK allele comprising a disease-associated-repeat. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide or RNAi oligonucleotide.