OAV101 IT for treating spinal muscular atrophy in children aged 2 and older

OAV101 IT is an advanced therapy medicinal product (gene therapy product) in clinical development for the treatment of spinal muscular atrophy (SMA). SMA is a rare genetic condition that affects the motor neurons, specialised nerve cells that are responsible for coordinating movement between the brain and muscles. SMA is caused by having a faulty or missing survival motor neuron 1 (SMN1) gene, which normally produces the survival motor neuron (SMN) protein, which is crucial for motor neuron upkeep. SMA significantly affects both lifespan and quality of life; disease severity is grouped into five subtypes, which correlate generally to the age when symptoms first appear.