Biotechnology company REGENXBIO Inc (Nasdaq:RGNX) reported on Thursday the receipt of the US Food and Drug Administration's (FDA) Rare Pediatric Disease Designation for RGX-181 for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease.

The company said that RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

According to the company, RGX-181 is designed to use REGENXBIO's NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system (CNS), which may induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease. Following a single administration given by intracisternal injection, RGX-181 treatment is designed to modify cells in the CNS, thereby providing a durable source of TPP1 and allowing for long-term correction of cells throughout the CNS.

In the second half of 2019, the company intends to submit an Investigational New Drug (IND) application for RGX-181 with the US FDA to enable initiation of a first-in-human clinical trial. It has previously received the US FDA's Orphan Drug Designation for the candidate.