SHC1A-012

SHC1A-012 is an investigational antisense oligonucleotide (ASO) therapy being developed by Shift Pharmaceuticals for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). CMT1A is a hereditary, progressive peripheral neuropathy caused by a duplication in the PMP22 gene, leading to overproduction of PMP22 protein and subsequent demyelination of peripheral nerves. SHC1A-012 works by partially blocking the production of PMP22 protein in peripheral nerves, aiming to restore healthy myelin formation and improve nerve function. The drug is currently in preclinical development with support from organizations such as the Charcot-Marie-Tooth Association (CMTA)[2][3][9].