REGENXBIO Inc. provided a year-end 2019 corporate update and anticipated milestones for 2020. Product Candidate Updates: Gene Therapy Using NAV Vectors for AAV-Mediated Antibody Delivery: RGX-314 for the Treatment of Wet AMD. REGENXBIO expects to initiate a pivotal program for the subretinal delivery of RGX-314 for the treatment of wet AMD in the second half of 2020. A pivotal clinical trial is expected to investigate the one-time administration of RGX-314 at a single dose compared to anti-VEGF injections, which is the current standard of care for patients with wet AMD. The primary efficacy endpoint in the trial will be the mean change in visual acuity from baseline assessed at 12 months after treatment with RGX-314. REGENXBIO plans to finalize the design of the trial based on the 12-month assessment of patients in Cohort 5 in the Phase I/IIa trial. This will allow for further characterization of RGX-314-treated patients, enhancements of the trial design and the potential acceleration of the clinical program. REGENXBIO intends to submit the design of the trial to the FDA in mid-2020 and begin dosing patients in the second half of 2020. The U.S. Food and Drug Administration (FDA) has removed the partial clinical hold on the Investigational New Drug (IND) application for the Phase I/IIa trial of the subretinal delivery of RGX-314 without modification to the surgical delivery system, after reviewing information provided by the Company. The partial clinical hold was not related to the gene therapy candidate. As of December 31, 2019, RGX-314 continued to be well-tolerated across all cohorts in the Phase I/IIa trial, with no drug-related serious adverse events (SAEs) reported. All patients in Cohort 5 (2.5 x 1011 GC/eye) have now completed their 6-month follow-up.1 Patients in Cohort 5 continue to demonstrate a meaningful reduction in anti-vascular endothelial growth factor (anti-VEGF) treatment burden at 6 months following administration of RGX-314, with 8 out of 11 (73%) patients remaining anti-VEGF injection-free, and a reduction across the cohort of over 80% from the mean annualized injection rate during the 12 months prior to administration of RGX-314. Importantly, the Cohort 5 patients continued to demonstrate a mean improvement in vision of +3 ETDRS letters and mean improvement in retinal thickness of -83 microns, while the 8 patients who were anti-VEGF injection-free after administration of RGX-314 showed a mean improvement in vision of +5 ETDRS letters and mean improvement in retinal thickness of -83 microns. REGENXBIO plans to initiate the Phase II trial of the suprachoroidal delivery of RGX-314 using the SCS Microinjector™ for the treatment of wet AMD in the first half of 2020. The trial will build upon data from the Phase I/IIa trial of RGX-314 and is expected to evaluate patients in two dose cohorts of RGX-314 versus a control arm. Interim data is expected from Cohort 1 by the end of 2020. RGX-314 for the Treatment of Diabetic Retinopathy (DR) REGENXBIO expects to submit an IND in the first half of 2020 and plans to initiate a Phase II trial of the suprachoroidal delivery of RGX-314 using the SCS Microinjector for the treatment of DR in the second half of 2020. The trial is expected to evaluate patients in up to three dose cohorts of RGX-314 versus a control arm. Enrollment of Cohort 1 is expected to be complete by the end of 2020, with interim data expected in 2021. Research Program for the Treatment of Hereditary Angioedema (HAE). As first announced in July 2019, REGENXBIO is developing a one-time gene therapy candidate to deliver a gene encoding a therapeutic antibody against plasma kallikrein, a key protein of the plasma contact pathway which is left unregulated in patients with HAE. Preclinical animal studies conducted using NAV AAV8 indicate the potential for a sustained and safe delivery of biologically active antibody at therapeutic concentrations. REGENXBIO expects to select a lead product candidate in the first half of 2020 and provide a program update in the second half of 2020.Research Program for the Treatment of Neurodegenerative Diseases. REGENXBIO has expanded its exclusive collaboration program with Neurimmune AG to include therapies targeting alpha synuclein, in addition to tauopathies. The program uses Neurimmune's Reverse Translational Medicine™ platform along with REGENXBIO's NAV Technology Platform to design and develop vectorized antibody therapies for neurodegenerative diseases. REGENXBIO expects to provide a program update in the second half of 2020. Anticipated 2020 Milestones: REGENXBIO expects to meet the following milestones related to the development of internal product candidates in 2020: Gene Therapy Using NAV Vectors for AAV-Mediated Antibody Delivery: RGX-314 for the Treatment of Wet AMD: Subretinal delivery; Complete 12-month assessments of Cohort 5 in the Phase I/IIa trial in the first half of 2020. Initiate a pivotal trial in the second half of 2020. Suprachoroidal delivery; Initiate Phase II trial in the first half of 2020. Report interim data from Cohort 1 of the Phase II trial in the second half of 2020. RGX-314 for the Treatment of DR. Submit IND for a Phase II trial of suprachoroidal delivery in the first half of 2020. Initiate Phase II trial of suprachoroidal delivery in the second half of 2020. Research Program for Treatment of HAE. Select lead product candidate in first half of 2020. Provide program update in the second half of 2020. Research Program for Treatment of Neurodegenerative Diseases. Provide program update in the second half of 2020.