Casimersen
Overview
Casimersen is a phosphorodiamidate morpholino oligomer designed to bind to exon 45 of dystrophin pre-mRNA, promoting exon skipping in the DMD gene. This mechanism helps patients with Duchenne muscular dystrophy who have mutations amenable to exon 45 skipping to produce functional dystrophin protein.
Patent & exclusivity (US)
Per the FDA Orange Book (1 approved US product), the latest listed US patent on Casimersen expires 12 Nov 2030 and exclusivity 25 Feb 2028. Generic API sourcing typically opens up once these lapse.
Therapeutic areas
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