A single treatment with the potential
for long-lasting results
RGX-181 is our product candidate for the treatment of late-infantile neuronal ceroid lipofuscinosis Type 2 (CLN2 disease), a form of Batten disease.
RGX-181 is designed to use the 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.
We expect to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for RGX-181 to enable initiation of a first-in-human clinical trial for children with CLN2 disease. RGX-181 has received orphan drug product designation and rare pediatric disease designation from the FDA.