US 11,712,478 B2
Compositions and methods of treating muscle atrophy and myotonic dystrophy
Andrew John Geall, Carlsbad, CA (US); Venkata Ramana Doppalapudi, San Diego, CA (US); David Sai-Ho Chu, La Jolla, CA (US); Michael Caramian Cochran, La Jolla, CA (US); Michael Hood, San Diego, CA (US); Beatrice Diana Darimont, San Diego, CA (US); Rob Burke, Encinitas, CA (US); Yunyu Shi, San Diego, CA (US); Gulin Erdogan Marelius, San Diego, CA (US); and Barbora Malecova, La Jolla, CA (US)
Assigned to AVIDITY BIOSCIENCES, INC., La Jolla, CA (US)
Filed by Avidity Biosciences, Inc., La Jolla, CA (US)
Filed on Oct. 12, 2021, as Appl. No. 17/499,796.
Application 17/499,796 is a division of application No. 17/024,624, filed on Sep. 17, 2020.
Application 17/024,624 is a continuation of application No. 16/435,422, filed on Jun. 7, 2019, granted, now 10,881,743, issued on Jan. 5, 2021.
Application 16/435,422 is a continuation of application No. PCT/US2018/064359, filed on Dec. 6, 2018.
Claims priority of provisional application 62/725,883, filed on Aug. 31, 2018.
Claims priority of provisional application 62/595,545, filed on Dec. 6, 2017.
Prior Publication US 2022/0040323 A1, Feb. 10, 2022
Int. Cl. C07H 21/04 (2006.01); A61K 47/68 (2017.01); A61K 31/712 (2006.01); A61K 31/713 (2006.01); C07K 16/18 (2006.01); C12N 15/113 (2010.01); A61P 21/00 (2006.01); A61K 39/395 (2006.01); A61K 9/51 (2006.01)
CPC A61K 47/6807 (2017.08) [A61K 31/712 (2013.01); A61K 31/713 (2013.01); A61K 39/395 (2013.01); A61K 47/6849 (2017.08); A61P 21/00 (2018.01); C07K 16/18 (2013.01); C12N 15/113 (2013.01); A61K 9/5107 (2013.01); C12N 2310/14 (2013.01); C12N 2310/315 (2013.01); C12N 2310/317 (2013.01); C12N 2310/3513 (2013.01); C12N 2310/3515 (2013.01); C12N 2320/31 (2013.01); C12N 2320/32 (2013.01)] 13 Claims
 
1. A small interfering RNA (siRNA)-antibody conjugate comprising an anti-transferrin receptor antibody or antigen-binding fragment thereof conjugated to an siRNA that hybridizes to a target sequence of human Atrogin-1 mRNA and mediates RNA interference against the human Atrogin-1 mRNA preferentially in a muscle cell, wherein the siRNA comprises a guide strand and a passenger strand, and the guide strand comprises a sequence selected from SEQ ID NO: 318, SEQ ID NO: 321, SEQ ID NO: 325, SEQ ID NO: 327, SEQ ID NO:333, SEQ ID NO: 337, SEQ ID NO:339, SEQ ID NO:342, SEQ ID NO:344, SEQ ID NO: 346, SEQ ID NO:348, SEQ ID NO:350, SEQ ID NO:353, SEQ ID NO:358, SEQ ID NO:359, SEQ ID NO:361, SEQ ID NO:363, SEQ ID NO:365, SEQ ID NO:366 and SEQ ID NO: 368.