REGENXBIO(US:RGNX) Prnewswire·2024-08-01 11:03Core Insights - REGENXBIO Inc. reported positive interim safety and efficacy data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 for Duchenne muscular dystrophy, showing robust microdystrophin expression levels in treated patients [1][3][5] - The company plans to initiate a pivotal trial in Q4 2024 and expects to complete enrollment in the dose level 2 expansion cohort early in Q3 2024 [1][7] Company Overview - REGENXBIO is a clinical-stage biotechnology company focused on gene therapy, with a proprietary NAVXpress™ platform for manufacturing [10][12] - RGX-202 is a one-time AAV therapeutic designed to deliver a novel microdystrophin gene, which is the only gene therapy in development for Duchenne that encodes for the C-Terminal domain [2][10] Clinical Trial Details - The AFFINITY DUCHENNE trial is a multicenter, open-label study evaluating the safety, tolerability, and clinical efficacy of RGX-202 in patients aged 1-11 years [8][9] - Initial results showed microdystrophin expression levels of 77.2% and 46.5% in patients aged 5.8 and 8.5 years, respectively, compared to control [4][5] Manufacturing and Capacity - RGX-202 is manufactured at the REGENXBIO Manufacturing Innovation Center, which has the capacity to produce up to 2,500 doses per year using a scalable suspension-based process [6][10] Future Expectations - The company anticipates sharing initial strength and functional assessment data for both dose levels of the AFFINITY DUCHENNE trial in the second half of 2024 [7]