Patent ID: 11872287
Assignee: AVIDITY BIOSCIENCES, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 0:
1. A method of treating muscle atrophy or myotonic dystrophy in a subject in need thereof, comprising administering to the subject an siRNA molecule conjugate comprising an anti-transferrin receptor binding moiety conjugated to an siRNA molecule that hybridizes to a target sequence of a human DMPK mRNA and mediates RNA interference against the human DMPK mRNA preferentially in muscle cells in the subject, wherein the target sequence of the human DMPK mRNA comprises exons 1-13 of the human DMPK mRNA excluding CUG repeats, thereby treating muscle atrophy or myotonic dystrophy in the subject.