SKG0201

SKG0201 is a novel adeno-associated virus (AAV)-mediated gene replacement therapy developed for the treatment of spinal muscular atrophy (SMA), specifically Type 1. The therapy consists of a codon-optimized human SMN1 cDNA under the control of a central nervous system (CNS)-selective promoter and specific regulatory elements, designed to drive high expression of SMN1 protein in the CNS and appropriate expression in peripheral tissues. By providing a functional copy of the human SMN1 gene, SKG0201 aims to address the root cause of SMA by restoring survival motor neuron (SMN) protein levels. Preclinical studies have shown that intravenous administration leads to rapid and sustained correction of SMN protein deficiency, increased body weight, extended lifespan in animal models, and no acute hepatotoxicity at high doses. Clinical trials are ongoing to evaluate its safety and efficacy in infants with SMA Type 1[1][2][3].