RGX-181 is designed to use the NAV® AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the central nervous system (CNS).
CLN2 disease is a rare, recessive genetic disease caused by a deficiency in TPP1, an enzyme required for the breakdown of specific peptides in the lysosomes of cells.
Disease onset is generally between two to four years of age with initial features of recurrent seizures (epilepsy), language delay, and difficulty coordinating movements (ataxia). Following onset, the disease progresses rapidly resulting in loss of language and motor functions, seizures, cognitive decline, vision loss, and premature death by mid-childhood.
There is currently no cure for CLN2 disease or treatment that targets the underlying genetic disorder. We believe that delivery of the gene encoding for the TPP1 enzyme via a one-time administration could provide a durable source of TPP1 activity in the CNS, thereby potentially preventing neurological decline and other disease manifestations.