individualized antisense oligonucleotide

EveryONE Medicines is developing individualized antisense oligonucleotide (ASO) therapies for pediatric patients with rare, severely debilitating, and life-threatening (SDLT) central nervous system (CNS) disorders caused by unique genetic variants. These 'n-of-1' medicines are customized for each individual patient, targeting specific mutations that typically affect only one or two people globally. The active ingredients are single-stranded, chemically modified, synthetic RNA molecules designed to bind to endogenous RNA sequences through Watson-Crick base pairing, thereby modulating gene expression or correcting splicing errors. The company was co-founded by Julia Vitarello and Dr. Timothy Yu, who previously developed milasen, the first individualized ASO. EveryONE Medicines is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of these patient-specific ASOs in children with SDLT CNS conditions.

Other names

n-of-1 medicinesn-of1 medicinesn-of 1 medicinespatient-specific ASO therapies