US 11,872,287 B2
Compositions and methods of treating muscle atrophy and myotonic dystrophy
Andrew John Geall, Carlsbad, CA (US); Venkata 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., San Diego, CA (US)
Filed by Avidity Biosciences, Inc., San Diego, CA (US)
Filed on Sep. 27, 2022, as Appl. No. 17/935,922.
Application 17/529,207 is a division of application No. 17/024,624, filed on Sep. 17, 2020, granted, now 11,253,607, issued on Feb. 22, 2022.
Application 17/935,922 is a continuation of application No. 17/822,342, filed on Aug. 25, 2022.
Application 17/822,342 is a continuation of application No. 17/529,207, filed on Nov. 17, 2021, granted, now 11,576,980.
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 2023/0201362 A1, Jun. 29, 2023
This patent is subject to a terminal disclaimer.
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)] 17 Claims
 
1. A method of treating muscle atrophy or myotonic dystrophy in a subject in need thereof, comprising administering to the subject an siRNA molecule conjugate comprising an anti-transferrin receptor binding moiety conjugated to an siRNA molecule that hybridizes to a target sequence of a human DMPK mRNA and mediates RNA interference against the human DMPK mRNA preferentially in muscle cells in the subject, wherein the target sequence of the human DMPK mRNA comprises exons 1-13 of the human DMPK mRNA excluding CUG repeats, thereby treating muscle atrophy or myotonic dystrophy in the subject.