EDK060

EDK060 is an investigational oligonucleotide drug under development by Novartis for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)[1][3][5][7]. It is being evaluated in first-in-human studies focused on safety, tolerability, and pharmacokinetics. EDK060 is designed to address genetic and neurological aspects of CMT1A, a hereditary neuropathy characterized by demyelination of peripheral nerves. While the specific detailed mechanism of action is not disclosed in public sources, its development as an oligonucleotide suggests a targeted approach toward disease-driving molecules, likely via RNA modulation or gene expression interference[7].