Back to results
US 11,999,955 B2Granted
Compositions and methods of treating facioscapulohumeral muscular dystrophy
AssigneeAvidity Biosciences, Inc.
InventorsBarbora Malecova, Rob Burke, David Sala Cano, Beatrice Diana Darimont
Life of this patent
Estimating the term…
Abstract
Disclosed herein are polynucleic acid molecules, pharmaceutical compositions, and methods for treating Facioscapulohumeral muscular dystrophy.
Claims16
A polynucleotide-antibody conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a polynucleotide, wherein the polynucleotide is from 19 to 30 nucleotides in length, wherein the polynucleotide comprises a nucleic acid sequence selected from a group consisting of SEQ ID NOs:
- 72, 76, 126, 131, 132, 134, 135, 136, 212, 216, 266, 271, 272, 274, 275, and 276 and wherein the polynucleotide mediates RNA interference against DUX4 mRNA and downregulation of DUX4 biomarker mRNA level.
Dependencies are inferred from the claim text.