Diplotype-based expanded use of individualized allele-specific antisense oligonucleotides

Abstract While an accelerated FDA process enables development and treatment trials of ASOs for individual patients within a year, it does not scale to patient communities. We evaluated a diplotype-based pathway for broad use of individualized ASOs whose pre-mRNA targets contain common single nucleotide variants. Two such ASOs that had shown safety and effectiveness in individual patients with SCN2A-Complex Neurodevelopmental Disorder were compatible with use in seven of fifteen patients evaluated.