MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES

Abstract: aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. in some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. in some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. in some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mrna expressed from a mutant dmd allele.

IPC:

CPC: A61K47/6807,