US 11,999,955 B2
Compositions and methods of treating facioscapulohumeral muscular dystrophy
Barbora Malecova, San Diego, CA (US); Rob Burke, Carlsbad, CA (US); David Sala Cano, San Diego, CA (US); and Beatrice Diana Darimont, San Diego, CA (US)
Assigned to AVIDITY BIOSCIENCES, INC., San Diego, CA (US)
Filed by Avidity Biosciences, Inc., San Diego, CA (US)
Filed on Nov. 4, 2022, as Appl. No. 18/052,898.
Application 18/052,898 is a continuation of application No. 17/200,612, filed on Mar. 12, 2021, granted, now 11,555,190.
Claims priority of provisional application 63/066,655, filed on Aug. 17, 2020.
Claims priority of provisional application 62/992,071, filed on Mar. 19, 2020.
Prior Publication US 2023/0340474 A1, Oct. 26, 2023
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/113 (2010.01); A61K 31/7125 (2006.01); A61K 47/68 (2017.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01); A61P 21/06 (2006.01); C07K 16/28 (2006.01); C07K 19/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 31/7125 (2013.01); A61K 47/6807 (2017.08); A61K 47/6849 (2017.08); A61K 47/6889 (2017.08); A61K 48/005 (2013.01); A61P 21/00 (2018.01); A61P 21/06 (2018.01); C07K 16/2881 (2013.01); C07K 19/00 (2013.01); C07K 2317/55 (2013.01); C12N 2310/11 (2013.01); C12N 2310/14 (2013.01); C12N 2310/31 (2013.01); C12N 2310/313 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/32 (2013.01)] 16 Claims
 
1. 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.