ROCKVILLE, Md., Dec. 8, 2020 /PRNewswire/ — REGENXBIO Inc. (Nasdaq: RGNX), 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 interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II). In addition, REGENXBIO announced plans to evaluate a higher dose of RGX-121 in a third cohort of patients at an increased dose of 2.0×1011 GC/g brain mass. REGENXBIO expects to begin enrolling patients in Cohort 3 in the first quarter of 2021.

RGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. RGX-121 is delivered directly to the central nervous system (CNS) via intracisternal administration.

“We are very pleased to see the encouraging safety profile of RGX-121, as well as additional evidence of long-term biomarker activity and neurocognitive development in patients with MPS II up to two years after RGX-121 administration. The consistent reduction in heparan sulfate in the CSF suggests that the gene therapy may potentially restore intracellular activity of the I2S enzyme, and improve neurocognitive development and outcomes for patients,” said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. “We are also encouraged by the I2S enzyme systemic expression observed at the second dose level. We look forward to presenting additional details from these first two cohorts at the WORLDSymposium in early 2021.”

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