	US 12,071,485 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 Jan. 2, 2024, as Appl. No. 18/402,596.
Application 18/402,596 is a division of application No. 17/932,653, filed on Sep. 15, 2022, granted, now 11,912,779.
Claims priority of provisional application 63/245,123, filed on Sep. 16, 2021.
Prior Publication US 2024/0124600 A1, Apr. 18, 2024
Int. Cl. C07K 16/28 (2006.01); A61K 39/00 (2006.01); A61P 21/00 (2006.01); C12N 15/113 (2010.01)

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)]

13 Claims

1. A method for treating muscular dystrophy in a subject in need thereof, comprising:

providing a polynucleic acid 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: 412-420; and

administering a therapeutic amount of the polynucleic acid conjugate to the subject in need thereof to treat the muscular dystrophy, wherein the polynucleic acid conjugate reduces a quantity of the mRNA transcript of human DUX4, by mediating RNA interference against the human DUX4, and thereby treating muscle dystrophy in the subject.