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REGENXBIO Presents Additional Positive Interim Data from Phase I/II Trial of RGX-121 for the Treatment of MPS II (Hunter Syndrome) at American Society of Gene and Cell Therapy's 24th Annual Meeting
ROCKVILLE, Md., May 14, 2021 /PRNewswire/ -- RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related serious adverse
About this update from Regenxbio Inc.
[{"type":"text","content":"ROCKVILLE, Md., May 14, 2021 /PRNewswire/ --\nRGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related serious adverse events Biomarkers and measures of neurodevelopmental function from patients in Cohorts 1 and 2 continue to indicate CNS activity following RGX-121 administration Evidence of systemic enzyme expression and biomarker activity continues to be observed REGENXBIO recently initiated dosing of patients in Cohort 3REGENXBIO Inc. (Nasdaq: RGNX) today announced a safety update and additional positive interim data from 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), also known as Hunter Syndrome. The latest data from this trial will be presented today at the American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting by Dr. Roberto Giugliani, Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil.\n\"I am pleased to report additional data from the Phase I/II trial of RGX-121 in patients with MPS II. The biomarker data from these patients continues to demonstrate that the I2S enzyme is active in the CNS and importantly, continued cognitive development has been observed in the majority of patients who have been followed for more than 6 months. In addition, emerging evidence of systemic enzyme expression and activity has been shown in urine and plasma, including in patients who are naive to enzyme replacement therapy, the current standard of care,\" said Dr. Giugliani. \"The potential to provide therapeutic benefit from a one-time gene therapy would be a meaningful advancement for the treatment of MPS II patients.\"\n\"It is encouraging to see continued efficacy signals up to two years after administration of RGX-121 in our Phase I/II trial. The safety profile we have seen to date, combined with evidence of CNS and systemic effects, are encouraging as we seek to bring a one-time gene therapy treatment to the MPS II community,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"We have begun dosing patients in Cohort 3, and we look forward to providing further program updates later this year.\"\nRGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using ...