The FDA has granted REGENXBIO Inc. a rare pediatric disease designation for RGX-181, for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease.
CLN2 is a rare, autosomal recessive, neurodegenerative lysosomal storage disorder caused by mutations in the TPP1 gene. The disease is characterized by seizures, rapid language and motor function deterioration, cognitive decline, loss of vision and blindness, and premature death by mid-childhood. Symptom onset is usually between the ages of two to four years. There is currently no cure for CLN2 disease.
RGX-181 is being developed as a novel, one-time treatment for CLN2 disease using REGENXBIO’s NAV AAV9 vector to deliver the TPP1 gene directly into the central nervous system (CNS). TPP1 is an enzyme that is deficient in children who have CLN2 disease. RGX-181 treatment is designed to modify cells in the CNS and provide a lasting source of TPP1.
Source: RegenXBio, January 31, 2019