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REGENXBIO Announces Continued Progress and Expansion of Clinical Development Program for RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)
- Ongoing Phase I/II trial to enroll up to 6 additional patients in Cohort 2 while planning for next steps in program; further trial updates expected by the
About this update from Regenxbio Inc.
[{"type":"text","content":"- Ongoing Phase I/II trial to enroll up to 6 additional patients in Cohort 2 while planning for next steps in program; further trial updates expected by the end of 2020\n - New IND for Phase I/II trial cleared by FDA to evaluate RGX-121 in patients with severe MPS II ages 5-18 years old and expected to initiate in 2H 2020\n - Initiation of a new prospective observational study expected to provide detailed characterization of neurocognitive development and key biomarkers in patients with severe MPS II\n\n\nROCKVILLE, Md., Sept. 30, 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 the expansion of the RGX-121 program for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome, to gain additional insight into the neurodegenerative manifestations of the disease and evaluate RGX-121 in a broader patient population. RGX-121 is an investigational one-time gene therapy, designed to use the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) through intracisternal administration.\n\n \n \n \n \n \n \n\n \n\"Today's update reflects significant forward progress in our clinical program for the treatment of MPS II, as we expand the program to gain additional insight into the potential treatment effects of RGX-121 in more patients. REGENXBIO is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"In addition, we are announcing the initiation of an important prospective natural history study to provide critical data about the neurocognitive development of young pediatric patients with MPS II. We plan to share the data from this observational study with the patient community and look forward to working closely with researchers and advocates in order to enhance awareness of this study.\" \n\"MPS II is a serious and debilitating lysosomal disease that affects 1 in 100,000 children, and available treatments are inadequate ...