REGENXBIO Announces Pipeline Expansion with RGX-181 Gene Therapy for CLN2 Form of Batten Disease

REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced it is developing a new product candidate, RGX-181, for the treatment of 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.

"Our NAV technology platform holds tremendous promise for advancing this new product candidate in our neurodegenerative disease pipeline," said Olivier Danos, Ph.D., Senior Vice President and Chief Scientific Officer of REGENXBIO. "CLN2 is a rapidly progressing, fatal disease with limited treatment options and no cure. Children with CLN2 disease experience an array of serious symptoms such as seizures, deterioration of language and motor skills, blindness, cognitive decline and premature death. The goal of our RGX-181 clinical program is to develop a single-dose treatment to halt progression of neurological decline and improve a broad range of these devastating symptoms experienced by children with CLN2 disease."

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. Pharmacology studies conducted in an animal model of CLN2 disease demonstrated that a single administration of RGX-181 resulted in widespread distribution and sustained expression of the TPP1 enzyme in the CNS with significant improvements in neurobehavioral function and survival of the animals.

"For children living with Batten disease and their families, the promise of effective gene therapy gives incredible hope," said Margie Frazier, Ph.D., the Executive Director at the Batten Disease Support and Research Association. "A diagnosis of Batten disease is devastating, yet children with the condition, and their families, face the disease with courage. More and widely available treatment options are desperately needed to reduce the cognitive and physical effects of the disease."

REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced it is developing a new product candidate, RGX-181, for the treatment of 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.

"Our NAV technology platform holds tremendous promise for advancing this new product candidate in our neurodegenerative disease pipeline," said Olivier Danos, Ph.D., Senior Vice President and Chief Scientific Officer of REGENXBIO. "CLN2 is a rapidly progressing, fatal disease with limited treatment options and no cure. Children with CLN2 disease experience an array of serious symptoms such as seizures, deterioration of language and motor skills, blindness, cognitive decline and premature death. The goal of our RGX-181 clinical program is to develop a single-dose treatment to halt progression of neurological decline and improve a broad range of these devastating symptoms experienced by children with CLN2 disease."

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. Pharmacology studies conducted in an animal model of CLN2 disease demonstrated that a single administration of RGX-181 resulted in widespread distribution and sustained expression of the TPP1 enzyme in the CNS with significant improvements in neurobehavioral function and survival of the animals.

"For children living with Batten disease and their families, the promise of effective gene therapy gives incredible hope," said Margie Frazier, Ph.D., the Executive Director at the Batten Disease Support and Research Association. "A diagnosis of Batten disease is devastating, yet children with the condition, and their families, face the disease with courage. More and widely available treatment options are desperately needed to reduce the cognitive and physical effects of the disease."

REGENXBIO plans to submit an investigational new drug (IND) application for RGX-181 to the U.S. Food and Drug Administration in 2019 to enable initiation of a global first-in-human clinical trial with clinical centers planned in the United States and Europe. REGENXBIO has two other CNS products, RGX-111 and RGX-121, in clinical trials for the treatment of Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II) diseases, respectively.

"Given the unmet need and the potential for NAV gene therapy, we believe CLN2 disease is a natural addition to our neurodegenerative disease pipeline," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "As we prepare to initiate dosing in our clinical trials of RGX-111 for MPS I and RGX-121 for MPS II, adding RGX-181 to our research pipeline furthers our commitment to finding potential cures for children affected by these extremely serious and life-threatening neurodegenerative conditions."

enable initiation of a global first-in-human clinical trial with clinical centers planned in the United States and Europe. REGENXBIO has two other CNS products, RGX-111 and RGX-121, in clinical trials for the treatment of Mucopolysaccharidosis Type I (MPS I) and Mucopolysaccharidosis Type II (MPS II) diseases, respectively.

"Given the unmet need and the potential for NAV gene therapy, we believe CLN2 disease is a natural addition to our neurodegenerative disease pipeline," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "As we prepare to initiate dosing in our clinical trials of RGX-111 for MPS I and RGX-121 for MPS II, adding RGX-181 to our research pipeline furthers our commitment to finding potential cures for children affected by these extremely serious and life-threatening neurodegenerative conditions."

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