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.

Inventor(s): Romesh R. Subramanian, Mohammed T. Qatanani, Timothy Weeden, Cody A. Desjardins, Brendan Quinn, John Najim

CPC Classification: A61K47/6807 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

Search for rejections for patent application number 20250177549