Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — American Society of Retina Specialists (ASRS) 2026
10 slides from Regenxbio's conference presentation, July 2026
Slide 1: Subretinal Delivery of Investigational Sura-vec Gene Therapy for nAMD: Long-Term Follow-Up
Subretinal Delivery of Investigational Sura-vec Gene Therapy for Neovascular Age-Related Macular Title of Presentation, Title Case Degeneration: Long-Term Follow-Up of Phase 1/2a Participants Robert Avery, MD1; Stephen Huddleston, MD2,3; Jeffrey Heier, MD4,5; Peter Campochiaro, MD6; Allen C. Ho, MD7
Slide 2: Disclosures
Disclosures AbbVie and REGENXBIO funded this study and participated in the study design, research, analysis, data collection, interpretation of data, and the review and approval of the publication. All authors had access to relevant data and participated in the drafting, review, and approval of this
Slide 3: Background
Background Surabgene Lomparvovec (Sura-vec; ABBV-RGX-314) Product Candidate • Mechanism of action: reducing leaky blood vessel formation by giving retinal cells the ability to produce an anti-VEGF fab • Subretinal route of administration • NAV® vector: AAV8 • Gene: anti-VEGF fab Phase I/IIa nAMD Stu
Slide 4: Purpose and Methods
Purpose and Methods Phase I/IIa Study Long-Term Follow-Up (LTFU) Study 0 1 2 2.5 3 4 5 Sura-vec Subretinal Year Administration LTFU Visits Post Sura-vec Key Phase I/IIa Eligibility Criteria LTFU Visit Schedule & Assessments • Male or female, 50-89 years Visit Schedule: • nAMD with response to anti-V
Slide 5: Baseline Characteristics of Participants in LTFU
Results: Baseline Characteristics of Participants in LTFU • 37 of the 42 participants from Ph I/IIa (RGX-314-001) across all cohorts enrolled in LTFU • Long history of nAMD treatment Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Total Parameter (n=5)a (n=6) (n=6) (n=12) (n=8)b (N=37) Mean age, years
Slide 6: Safety in LTFU
Results: Safety in LTFU • The most frequent ocular AESI in the study eye was retinal pigmentary changes (26/37, 70.3%) • After Year 2 in the LTFU: • 1 new case of peripheral retinal thinning (Cohort 4) • 1 new case of significant vision decrease (Cohort 5) in a participant who had macular pigmentary
Slide 7: Mean BCVA Through Years in All Cohorts
Results: Mean BCVA Through 5 Years in All 5 Cohorts Note: Error bars represent standard deviation. a1 participant died during the study. b4 participants did not enroll in LTFU (three participants did not enroll and one participant died in the parent study). BCVA, best corrected visual acuity.
Slide 8: Mean BCVA Through Years
Results: Mean BCVA Through 5 Years Without PCV (refractory to anti-VEGF) Participant in Cohort 4a Note: Error bars represent standard deviation. aParticipant with PCV (refractory to anti-VEGF therapy) was excluded from Cohort 4. BCVA, best corrected visual acuity; PCV, polypoidal choroidal vasculopa
Slide 9: Annualized Injection Rate Pre and Post Sura-Vec Through Years
Results: Annualized Injection Rate Pre and Post Sura-Vec Through 5 Years Note: The dashed boxes contain the injection burden reduction in Year 5 compared to the year prior. aPercent reduction is calculated as (annualized anti-VEGF injection rate in the prior year - annualized supplemental anti-VEGF
Slide 10: Conclusions: 5-Year Results of the Phase I/IIa Study
Conclusions: 5-Year Results of the Phase I/IIa Study No new safety signals were identified for participants who completed the study Cohorts 3 and 4 demonstrated stable to improved visual acuity (apart from a PCV outlier in Cohort 4) Cohorts 3 and 4 had > 64% reductions in injection burden after a si
REGENXBIO — American Society of Retina Specialists (ASRS) 2026 on 6ix