	US 12,071,621 B2
	Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
Beatrice Diana Darimont, San Diego, CA (US); Usue Etxaniz Irigoien, San Diego, CA (US); Venkata Ramana Doppalapudi, San Diego, CA (US); Michael Caramian Cochran, La Jolla, CA (US); Isaac Marks, San Diego, CA (US); and Tyler Albin, San Diego, CA (US)
Assigned to AVIDITY BIOSCIENCES, INC., San Diego, CA (US)
Filed by Avidity Biosciences, Inc., San Diego, CA (US)
Filed on Apr. 4, 2023, as Appl. No. 18/130,757.
Claims priority of provisional application 63/327,725, filed on Apr. 5, 2022.
Prior Publication US 2023/0364256 A1, Nov. 16, 2023
Int. Cl. C07K 16/28 (2006.01); A61K 31/7125 (2006.01); A61K 39/395 (2006.01); A61K 47/68 (2017.01); A61P 21/00 (2006.01); C07K 19/00 (2006.01); C12N 15/113 (2010.01); A61K 39/00 (2006.01)

CPC C12N 15/113 (2013.01) [A61K 31/7125 (2013.01); A61K 39/3955 (2013.01); A61K 47/6807 (2017.08); A61K 47/6849 (2017.08); A61P 21/00 (2018.01); C07K 16/2881 (2013.01); C07K 19/00 (2013.01); A61K 2039/505 (2013.01); A61K 47/68 (2017.08); A61K 47/6811 (2017.08); C12N 2310/314 (2013.01); C12N 2310/3233 (2013.01); C12N 2320/33 (2013.01)]

21 Claims

1. A phosphorodiamidate morpholino oligonucleotide (PMO) conjugate comprising an anti-transferrin receptor antibody or antigen binding fragment thereof conjugated to a PMO molecule, wherein the PMO molecule consists of a sequence selected from the group consisting of SEQ ID NOs: 116-119.