nL-ATN1-002

nL-ATN1-002 is a personalized antisense oligonucleotide (ASO) drug developed to inhibit the expression of the ATN1 gene, specifically targeting pathogenic CAG trinucleotide expansions in ATN1. It is designed for ultra-rare, patient-specific treatment and is currently being evaluated in a single-participant clinical trial for dentatorubral-pallidoluysian atrophy (DRPLA), a neurodegenerative disorder caused by mutations in ATN1. The drug acts as an inhibitor of atrophin 1 (the protein product of ATN1), aiming to reduce toxic gain-of-function effects associated with expanded CAG repeats[1][2][4]. The developer and sponsor is n-Lorem Foundation, which specializes in individualized ASO therapies for nano-rare diseases[3][5].