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REGENXBIO Provides Update on Progress of Clinical Programs for Rare Genetic Neurodegenerative Diseases

ROCKVILLE, Md., July 8, 2020 /PRNewswire/ --A 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 that it has completed dosing of three patients in Cohort 2 of the Company's Phase I/II study of RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II) and reported encouraging data under a single-patient investigator-initiated Investigational New Drug (IND) application for RGX-111 for the treatment of Mucopolysaccharidosis Type I (MPS I) conducted at CHOC Children's. "We are pleased to have completed the dosing of three patients with MPS II at the second dose level of our RGX-121 Phase I/II study, which included involvement from new leading centers at UCSF Benioff Children's Hospital Oakland and Hospital de ClA­nicas de Porto Alegre in Brazil. We expect to provide an additional interim data update from the RGX-121 program later this year," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "Further, we are encouraged by the initial data from the first patient dosed with RGX-111, and we look forward to advancing our RGX-111 Phase I/II study."A A Enrollment in Cohort 2 of the Phase I/II study of RGX-121 is now complete, with three patients with MPS II dosed intracisternally with 6.5x1010 genome copies per gram (GC/g) of brain mass. As of June 24, 2020, RGX-121 is reported to be well-tolerated in patients across two dose levels, with no drug-related serious adverse events (SAEs). Additional data from both cohorts in this study and a program update will be available in the second half of 2020.Under a single-patient investigator-initiated IND for RGX-111, Raymond Wang, M.D., a biochemical genetics specialist at CHOC Children's, dosed a patient with severe MPS I intracisternally with 1x1010 GC/g of brain mass at the age of 21 months. MPS I is caused by a deficiency of the enzyme I±-l-iduronidase (IDUA) and subsequent accumulation of heparan sulfate (HS) within the central nervous system (CNS). High levels of HS in the CSF closely correlate with neurocognitive decline and are a key biomarker of enzyme activity. As of June 9, 2020, RGX-111 is reported to be well-tolerated in this patient, with no drug-related SAEs.The IDUA enzyme activity in the patient's cerebrospinal fluid (CSF) was below the limit of quantification prior to the administration of RGX-111. An increase in IDUA enzyme activity was detected at Week 12, the latest timepoint available following administration of RGX-111. High levels of HS in the CSF were measured in this patient at baseline, and initial data demonstrated sustained decreases in total HS in the CSF over time, with a 50% reduction from baseline at Week 12 and a 45% reduction from baseline at Week 33, the latest timepoint available.
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