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US 11,446,387 B2Granted
Compositions and methods of treating muscle dystrophy
AssigneeAvidity Biosciences, Inc.
InventorsVenkata Ramana Doppalapudi, Michael David Hood, Rob Burke, Michael Caramian Cochran, Beatrice Diana Darimont, 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 dystrophy (DM1).
Claims14
A small interfering RNA (siRNA) molecule conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a siRNA molecule that hybridizes to a target sequence of DMPK mRNA;
- wherein the sense strand of the siRNA comprises the sequence selected from the group consisting of SEQ ID NOs:
- 3, 5, 7, 9, 11, 13, and 15, and the antisense strand of the siRNA comprises the sequence selected from the group consisting of SEQ ID NOs:
- 4, 6, 8, 10, 12, 14, and 16;
- and
- wherein the siRNA molecule conjugate mediates RNA interference against DMPK.
2 more elements
Dependencies are inferred from the claim text.