Posted by Michael Wonder on 11 Feb 2019
RegenxBio receives rare paediatric disease designation for RGX-181 gene therapy for the treatment of CLN2 form of Batten disease
31 January 2019 - Novel, one-time investigational treatment for CLN2 disease designed to halt progression of this rare, paediatric, neurodegenerative disease.
RegenxBio today announced the U.S. FDA granted rare paediatric disease designation to RGX-181. 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. In addition to the rare paediatric disease designation, RegenxBio previously received orphan drug designation from the FDA for RGX-181.
RGX-181 is designed to use RegenxBio's NAV AAV9 vector to deliver the TPP1 gene directly to the central nervous system, which may induce sustained levels of TPP1, the enzyme deficient in children with CLN2 disease.
