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US 2023/0398231 A1Published application
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).
Claims20
A polynucleic acid molecule that hybridizes to a target sequence of DMPK mRNA comprising:
- a sense strand comprising a nucleic acid sequence of SEQ ID NO:
- 1;
- and
- an antisense strand comprising a nucleic acid sequence of SEQ ID NO:
- 2, wherein the antisense strand comprises 2′-F modified nucleotides at positions 2, 6, 14, and 16 from the 5′-end.
2 more elements
A polynucleic acid molecule conjugate comprising:
- an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a polynucleic acid molecule comprising a sense strand and an antisense strand;
- wherein the sense strand comprises a nucleic acid sequence of SEQ ID NO:
- 1, and the antisense strand comprises a nucleic acid sequence of SEQ ID NO:
- 2;
- and
- wherein the antisense strand comprises 2′-F modified nucleotides at positions 2, 6, 14, and 16 from the 5′-end.
3 more elements
Dependencies are inferred from the claim text.