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MPS II is estimated to occur in approximately 1 in 100,000 to 1 in 170,000 births.
The current disease-modifying therapy for MPS II is enzyme replacement therapy with a recombinant form of human I2S administered intravenously.
The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials.Treatment with RGX-121 has been shown to restore I2S enzyme activity in animal models of MPS II to levels equivalent to or greater than those in non-affected animals.The extent of CNS correction in animal studies suggests that RGX-121 has the potential to be an important and suitable therapeutic option for MPS II patients.However, intravenous enzyme therapy does not treat the CNS manifestations of MPS II. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy.REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.