Similar to ETX101, STK-001 is an antisense oligonucleotide designed to enhance the generation of wild-type SCN1A mRNA transcripts to restore inhibitory function in Dravet Syndrome (Bialer et al., 2020; Wengert et al., 2022). Here, SCN1A is linked to encephalopathy, progressive, early-onset, with brain edema and/or leukoencephalopathy.