Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — Investor Presentation (September 2026)
40 slides from Regenxbio's investor presentation, September 2026
Slide 1: Delivering the promise of gene therapy
Delivering the promise of gene therapy REGENXBIO Corporate Presentation September 2026
Slide 2: Forward-looking statements
Forward-looking statements This presentation includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are gen
Slide 3: Our Vision and Mission
Our Vision A world in which debilitating diseases can be treated with a one-time therapy, resulting in lasting benefits Our Mission Seeking to improve lives through the curative potential of gene therapy 3 © 2026 REGENXBIO INC. • ALL RIGHTS RESERVED.
Slide 4: About REGENXBIO
About REGENXBIO Industry-Leading Late-Stage Rare and Commercial Innovative AAV Platform Retinal Programs Readiness Next-Gen Capsids 100+ NAV® vectors, Multiple potential first- or Near-term catalysts and in-house, Expanding pipeline with 5 licensees, best-in-class candidates in or U.S.-based manufac
Slide 5: Two lead programs approaching regulatory approvals
Two lead programs approaching regulatory approvals Differentiated gene therapies addressing substantial patient populations ANTICIPATED REGULATORY PATIENT POPULATION DIFFERENTIATION RGX-202 MILESTONE 1 Potential DUCHENNE MUSCULAR DYSTROPHY ~15K best-in-class 2027 BLA submission (1Q) and U.S. patient
Slide 6: Our gene therapy franchise for rare and retinal diseases
Our gene therapy franchise for rare and retinal diseases Disease Area Indication Product Candidate Phase 1 Phase 2 Phase 3 Commercial Rights Duchenne Muscular Novel microdystrophin RGX-202 WHOLLY OWNED Dystrophy (DMD) NAV® AAV8 Direct delivery of IDS to Rare Hunter Syndrome RGX-121 CNS Disease (MPS
Slide 7: Our future is now
Our future is now Recent & upcoming milestones demonstrate momentum toward commercial stage 2026 2027 Recent & anticipated milestones RGX-202 • U.S. BLA submission (1Q) Topline pivotal data (2Q) Duchenne Muscular • Initiate AFFINITY RISE® (1H) 1 Complete confirmatory enrollment Dystrophy • Poten
Slide 8: We built next-generation manufacturing, delivering biologics-level scalability and industry-leading vector purity
We built next-generation manufacturing, delivering biologics-level scalability and industry-leading vector purity REGENXBIO Manufacturing Innovation Center in Rockville, MD • Full control of product quality, clinical, and commercial supply • Capacity to supply market o 2,500 RGX-202 doses/year o 350
Slide 9: RGX-202: Potential next and best-in-class opportunity in DMD
RGX-202: Potential next and best-in-class opportunity in Duchenne Muscular Dystrophy (DMD)
Slide 10: RGX-202: Designed to strengthen and preserve muscle long-term
RGX-202: Designed to strengthen and preserve muscle long-term • Unique market opportunity: Potential for approval in 2H 2027 to serve as the next approved gene therapy in Duchenne • Encouraging clinical data: Only gene therapy program to demonstrate the combination of robust microdystrophin expressi
Slide 11: RGX-202's proactive, comprehensive therapeutic approach
RGX-202’s proactive, comprehensive therapeutic approach Multiple factors may contribute to better outcomes for patients Novel Construct Immune Suppression Purity • NAV® AAV8 vector • Comprehensive, proactive • Leading purity levels (>80% full • Muscle-specific promoter immune suppression regimen cap
Slide 12: Topline pivotal data support potential best-in-class profile
Topline pivotal data support potential best-in-class profile AFFINITY DUCHENNE® pivotal study of RGX-202 in ambulatory patients aged 1+: results summary Primary endpoint1 met with high Participants exceeded expected Favorable, differentiated safety statistical significance (p<0.0001) disease traject
Slide 13: Biomarkers support consistent robust expression, transduction, and sarcolemmal localization
Topline Data Biomarkers support consistent robust expression, transduction, and sarcolemmal localization of RGX-202 microdystrophin WEEK 12 BIOPSY RGX-202 Microdystrophin1 VCN Positive Fibers2 mean by western blot copies/nucleus by immunofluorescence (±SD) (% of normal control) (qPCR) (%) Aged 1 to
Slide 14: Proactive immune suppression regimen designed to mitigate AEs
Proactive immune suppression regimen designed to mitigate AEs observed in other Duchenne gene therapy programs PREVIOUSLY OBSERVED IMMUNE RESPONSE WITH EXISTING GENE THERAPIES REGENXBIO Eculizumab (Inhibits Complement) TOTAL OF 5 DOSES NOVEL IMMUNE Sirolimus (Inhibits B and T cells via mTOR) W8 W9-1
Slide 15: RGX-202 continues to demonstrate favorable safety profile
Topline Topline Interim Data RGX-202 continues to demonstrate favorable safety profile • RGX-202 was well-tolerated • 2 TR-SAEs, both easily managed and resolved without sequelae • All other TR-TEAEs mild to moderate and resolved without sequelae Aged <4 Years Aged ≥4 Years Overall TR-SAEs N = 11, n
Slide 16: AFFINITY DUCHENNE external control methodology
AFFINITY DUCHENNE® external control methodology 35 External Data STEP 1 STEP 2 30 Sources Filter EC Participants by Key Entry Further Balance Baseline Covariates Limited Peak Performance Criteria1 of RGX and EC Group at Individual • FOR-DMD Patient Level 25 • Stable dose of corticosteroid for 12 • B
Slide 17: Participants exceeded external controls on all functional measures at 1 year
Topline Interim Data Participants exceeded external controls on all functional measures at 1 year Functional improvements vs. external controls using propensity score weighting LSMean Difference Endpoint RGX, n EC, n Favoring RGX-202 (95% CI) vTTStand 9 177 0.075 (0.055, 0.095) vTTRW 8* 176 0.351 (0
Slide 18: Participants aged 8+ exceeded external controls on all functional measures at 1 year
Topline Interim Data Participants aged 8+ (N=5) exceeded external controls on all functional measures at 1 year Functional improvements vs. external controls using propensity score weighting LSMean Difference Endpoint RGX, n EC, n Favoring RGX-202 (95% CI) vTTStand 5 88 0.092 (0.068, 0.116) vTTRW 5
Slide 19: RGX-202 microdystrophin expression correlated with functional improvement
Topline Interim Data (n=9) RGX-202 microdystrophin expression correlated with functional improvement Statistically significant correlation with NSAA change from baseline and from cTAP predicted value at 1 year Difference in change from baseline of RGX-202 20 20 N=9 N=9 Change from baseline total NSA
Slide 20: RGX-202 momentum and commercial readiness
RGX-202 momentum and commercial readiness Submit BLA in Q1 2027 under accelerated approval pathway to support potential Preparing to meet demand for improved approval and launch in 2H 2027 options May ‘26: Positive topline pivotal data; primary • Planning for broad ambulatory access endpoint met wit
Slide 21: Surabgene lomparvovec (sura-vec, ABBV-RGX-314)
Surabgene lomparvovec (sura-vec, ABBV-RGX-314): Potential to be the first gene therapy for Wet AMD and Diabetic Retinopathy
Slide 22: Sura-vec: Potential first gene therapy for wet AMD and diabetic retinopathy
Sura-vec: Potential first gene therapy for wet AMD and diabetic retinopathy • High treatment burden: Chronic, VEGF-driven diseases see high treatment burden (frequent intraocular or intravitreal injections) that leads to undertreatment and ultimately vision loss over time − Today’s predominant appro
Slide 23: Sura-vec is a one-time investigational gene therapy designed to deliver sustained treatment effect
Sura-vec is a one-time investigational gene therapy designed to deliver sustained treatment effect Uses the NAV® AAV8 vector to encode an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF) and fluid accumulation in the retina MECHANISM OF ACTION GENE NAV® VECTOR Reducing
Slide 24: Majority of anti-VEGF-treated patients face high burden + poor compliance
Majority of anti-VEGF-treated patients face high burden + poor compliance, leading to vision loss over time 341,000,000 US Population ~900,000 US Population with wAMD ~775,000 Diagnosed wAMD Patients ~642,000 Anti-VEGF Treated wAMD Patients ~360,000 Anti-VEGF Treated wAMD Patients with High Treatmen
Slide 25: Sura-vec designed to disrupt cycle of undertreatment
Sura-vec designed to disrupt cycle of undertreatment Burdensome dosing regimens are Subretinal sura-vec anticipated to integrate into existing main driver of current anti-VEGF retina practice infrastructure: Retina specialists routinely treatment discontinuation perform vitrectomy procedures as part
Slide 26: Wet AMD: Subretinal sura-vec is in late-stage clinical trials
Wet AMD: Subretinal sura-vec is in late-stage clinical trials and is on track to be the first gene therapy Two pivotal trials evaluating subretinal delivery for wet Phase III evaluating subretinal AMD, with Phase I/IIa data demonstrating long-term sura-vec in ~540 wet AMD patients at 2 dose levels*
Slide 27: Wet AMD: Data seen to date in Phase I/II subretinal studies
Wet AMD: Data seen to date in Phase I/II subretinal studies of sura-vec supportive of potential pivotal outcomes Phase I/IIa LTFU (BCVA) Overall Safety • Sura-vec has been well tolerated across Phase I/II (up to 5 years)* and Phase II Bioreactor Bridging^ studies (at 1 year) at doses similar to pivo
Slide 28: DR: Positive safety and efficacy in Phase II ALTITUDE trial
DR: Positive safety and efficacy in Phase II ALTITUDE® trial support initiation of pivotal Phase IIb/III NAAVIGATE study Phase IIb/III NAAVIGATE ALTITUDE evaluating suprachoroidal delivery of sura-vec in ~100 DR patients across 3 dose levels and 30 DME participants at DL4* • Multicenter, randomized,
Slide 29: ≥2-Step DRSS improvement without additional DR treatment at 2.5 years
≥2-Step DRSS improvement without additional DR treatment at 2.5 years; DL3 sura-vec treated subjects outperformed all other groups and controls Data cut: May 25, 2026. *Includes 1 subject who crossed over from control; **Includes 2 subjects who crossed over from control. Control subjects were eligib
Slide 30: 55% Achieved ≥2-Step Improvement without Additional DR Treatment at 2.5 Years
55% Achieved ≥2-Step Improvement without Additional DR Treatment at 2.5 Years > 70% of Pivotal Dose Subjects Did Not Experience a VTE ALTITUDE LTFU Day 1 Dose Level 3 Year 5 Sura-vec Year 1 Year 2.5 Total IVT Baseline DRSS through 47 1 Step Better 2 Steps Better Year 2.5 47 1 Step Better 2 Steps Bet
Slide 31: Global eye-care alliance with AbbVie validates retina franchise
Global eye-care alliance with AbbVie validates retina franchise and enables world-class commercialization capabilities REGENXBIO brings leadership and Sura-vec global pivotal trials on track AbbVie brings 75+ years of expertise in AAV and retinal gene to deliver highly differentiated commitment in e
Slide 32: RGX-121 (clemidsogene lanparvovec): Potential first gene therapy for Hunter Syndrome
RGX-121 (clemidsogene lanparvovec): Potential first gene therapy for Hunter Syndrome (MPS II)
Slide 33: RGX-121: Potential to move MPS II treatment paradigm beyond ERT
RGX-121: Potential to move MPS II treatment paradigm beyond ERT • No cure: Ultra-rare, rapidly progressive, life-threatening genetic disease; most do not live past the age of 20 • High treatment burden: Current SOC is weekly IV enzyme replacement therapy (ERT) • Urgent need: RGX-121 has the potentia
Slide 34: RGX-121 is designed to address genetic cause of MPS II
RGX-121 is designed to address genetic cause of MPS II What is RGX-121? One-time gene therapy using NAV® AAV9 vector to deliver a working copy of the gene missing or malfunctioning in boys with MPS II; administered directly to the central nervous system RGX-121 has shown strong signs of efficacy and
Slide 35: >80% reduction in CSF HS D2S6, key biomarker likely to predict clinical benefit
>80% reduction in CSF HS D2S6, key biomarker likely to predict clinical benefit in MPS II brain disease, sustained through 1 year Pivotal: Neurodevelopmental Skill Acquisition or Stability on all BSID-III Subscales in Participants at 1 Year Pivotal: Primary Endpoint Achieved with Sustained Reduction
Slide 36: RGX-121 data demonstrate correlation between CSF HS D2S6 level and cognitive outcomes
RGX-121 data demonstrate correlation between measured CSF HS D2S6 level at Week 16 and cognitive outcomes at 1 year Cognitive Subdomain Baseline BSID-III Cognitive Baseline BSID-III Cognitive Baseline Cognitive Function ≥ -2 SD Function > -2SD Function < -2SD Dose-finding & Interim Pivotal Dose-find
Slide 37: Strategic partnership with Nippon Shinyaku bolsters commercialization capabilities
Strategic partnership with Nippon Shinyaku bolsters commercialization capabilities Nippon Shinyaku leads REGENXBIO leads NS Pharma prepared to commercialization of RGX-121 manufacturing commercialize RGX-121 upon and RGX-111 in U.S. and Asia potential approval in US, focused on qualified treatment c
Slide 38: Discovering the next wave of gene therapies
Discovering the next wave of gene therapies Preclinical pipeline driven by new, efficient capsids
Slide 39: Expanding the therapeutic potential of AAV gene delivery
Expanding the therapeutic potential of AAV gene delivery AI-powered engineering platform generates new capsids that can improve efficacy at lower doses Developing new capsids Approaching IND readiness that can: for capsid that has • Improve tissue tropism demonstrated higher and cell specificity tra
Slide 40: Seeking to improve lives through the curative potential of gene therapy
Seeking to improve lives through the curative potential of gene therapy © 2026 REGENXBIO INC. • ALL RIGHTS RESERVED.