	US 11,912,779 B2
	Compositions and methods of treating facioscapulohumeral muscular dystrophy
Barbora Malecova, San Diego, CA (US); Rob Burke, Carlsbad, CA (US); Beatrice Diana Darimont, San Diego, CA (US); and David Sala Cano, San Diego, CA (US)
Assigned to AVIDITY BIOSCIENCES, INC., San Diego, CA (US)
Filed by Avidity Biosciences, Inc., San Diego, CA (US)
Filed on Sep. 15, 2022, as Appl. No. 17/932,653.
Claims priority of provisional application 63/245,123, filed on Sep. 16, 2021.
Prior Publication US 2023/0091801 A1, Mar. 23, 2023
Int. Cl. C07K 16/28 (2006.01); A61P 21/00 (2006.01); C12N 15/113 (2010.01); A61K 39/00 (2006.01)

CPC C07K 16/2881 (2013.01) [A61P 21/00 (2018.01); C12N 15/113 (2013.01); A61K 2039/505 (2013.01); C12N 2310/141 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01)]

17 Claims

1. A polynucleic acid molecule conjugate comprising:

an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a polynucleic acid molecule that hybridizes to a target sequence of DUX4;

wherein the polynucleic acid molecule comprises a nucleic acid sequence selected from SEQ ID NOs: 72, 76, 126, or 131-136;

wherein the polynucleic acid molecule comprises 2′-F modified nucleotides only at positions 2, 6, 14, and 16; and

wherein the polynucleic acid molecule conjugate mediates RNA interference against the DUX4.