01 Recombinant AAV9 Capsid Shell is a non-replicating adeno-associated virus capsid used to deliver a functional copy of the human SMN gene across the blood-brain barrier to cells without modifying the patient’s existing DNA
02 Human SMN Transgene is a stable, functioning SMN gene that is introduced into the cell’s nucleus
03 scAAV ITR (Self-complementary DNA Technology) is a human SMN transgene introduced as a self-complementary double- stranded molecule. The inclusion of this technology enables rapid onset of effect
04 Continuous Promoter activates the transgene and is designed to allow for continuous and sustained SMN expression