Compositions and methods of treating muscle atrophy and myotonic dystrophy
AssigneeAvidity Biosciences, Inc.
InventorsAndrew John Geall, Venkata Ramana Doppalapudi, David Sai-Ho Chu, Michael Caramian Cochran, Michael Hood, Beatrice Diana Darimont, Rob Burke, Yunyu Shi, Gulin Erdogan Marelius, Barbora Malecova
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Abstract
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating muscle atrophy or myotonic dystrophy.
Claims16
A siRNA molecule conjugate comprising an anti-transferrin receptor antibody conjugated to a sense strand of a siRNA molecule that hybridizes to a target sequence of human DMPK mRNA and mediates RNA interference against the human DMPK mRNA preferentially in muscle cells in a human subject, wherein the anti-transferrin receptor antibody comprises two light chain variable domains and two heavy chain variable domains.
Dependencies are inferred from the claim text.