Pipeline

Mechanism of Action of NS-065/NCNP-01

Exon skipping is a potential therapy that is being developed for patients with DMD. Specialized molecules are created to skip over the non-working part of the dystrophin gene and allow pieces of the puzzle to attach. It creates a smaller puzzle, but a puzzle that may produce some of the protein that muscles need to work properly. NS-065/NCNP-01-201 Phase II dose-finding study is evaluating the safety and dosing of an investigational medication called NS-065/NCNP-01, in the treatment of boys with DMD who have specific changes in the dystrophin gene that may be helped with the skipping of exon 53