US 12,064,483 B2
Nucleic acid-polypeptide compositions and methods of inducing exon skipping
Arthur A. Levin, Del Mar, CA (US); Andrew John Geall, Carlsbad, CA (US); Venkata Ramana Doppalapudi, San Diego, CA (US); Michael Caramian Cochran, La Jolla, CA (US); Hanhua Huang, San Diego, CA (US); and Rob Burke, Encinitas, CA (US)
Assigned to AVIDITY BIOSCIENCES, INC., San Diego, CA (US)
Filed by Avidity Biosciences, Inc., San Diego, CA (US)
Filed on Jun. 17, 2022, as Appl. No. 17/843,705.
Application 17/843,705 is a continuation of application No. 17/463,484, filed on Aug. 31, 2021, granted, now 11,400,163.
Application 17/463,484 is a continuation of application No. 16/129,696, filed on Sep. 12, 2018, granted, now 11,179,472, issued on Nov. 23, 2021.
Application 16/129,696 is a continuation of application No. 16/128,450, filed on Sep. 11, 2018, granted, now 10,994,020, issued on May 4, 2021.
Application 16/128,450 is a continuation of application No. PCT/US2018/012672, filed on Jan. 5, 2018.
Claims priority of provisional application 62/561,939, filed on Sep. 22, 2017.
Claims priority of provisional application 62/443,514, filed on Jan. 6, 2017.
Prior Publication US 2022/0313833 A1, Oct. 6, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. C07H 21/04 (2006.01); A61K 31/7088 (2006.01); A61K 31/713 (2006.01); A61K 47/60 (2017.01); A61K 47/64 (2017.01); A61K 47/68 (2017.01); A61K 48/00 (2006.01); A61P 21/00 (2006.01); C07K 14/00 (2006.01); C07K 16/28 (2006.01); C07K 16/40 (2006.01); C12N 15/113 (2010.01); A61K 38/00 (2006.01)
CPC A61K 47/6803 (2017.08) [A61K 31/7088 (2013.01); A61K 31/713 (2013.01); A61K 47/60 (2017.08); A61K 47/6455 (2017.08); A61K 47/6807 (2017.08); A61K 47/6849 (2017.08); A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61K 48/0083 (2013.01); A61P 21/00 (2018.01); C07K 14/003 (2013.01); C07K 16/2881 (2013.01); C07K 16/40 (2013.01); C12N 15/113 (2013.01); A61K 38/00 (2013.01); C12N 2310/11 (2013.01); C12N 2310/315 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/32 (2013.01); C12N 2320/33 (2013.01)] 24 Claims
 
1. A method of treating muscular dystrophy in a subject in need thereof, comprising:
administering to the subject a single stranded oligonucleotide conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a single stranded oligonucleotide hybridizing to an acceptor splice site, a donor splice site, or an exonic splice enhancer element of a pre-mRNA transcript of the DMD gene;
wherein the single stranded oligonucleotide induces exon skipping in said pre-mRNA transcript to generate an mRNA transcript encoding a truncated dystrophin protein, thereby treating the muscular dystrophy in the subject.