EXG001-307

EXG001-307 is a recombinant adeno-associated virus (rAAV)-based gene therapy in clinical development for the treatment of spinal muscular atrophy (SMA) Type 1. It is designed to deliver a functional copy of the survival motor neuron 1 (SMN1) gene using an engineered AAV vector with a novel pro-NS promoter, which enhances expression in target spinal cord tissue and reduces off-target effects in liver and heart compared to existing therapies. EXG001-307 has demonstrated significant improvements in motor function—including head control and independent sitting—in infants with SMA Type 1 during Phase 1/2 trials, with high tolerability and no dose-limiting toxicities reported. The therapy is being developed by Exegenesis Bio and Hangzhou Jiayin Biotech, with ongoing clinical trials primarily targeting pediatric SMA patients[1][2][4][5].