Ocugen, Inc. · NASDAQ:OCGN
Ocugen — Investor Presentation (January 2026)
37 slides from Ocugen's investor presentation, Jan 15, 2026
Slide 1: Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market
Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market and Working Even Harder to Provide Access to Patients Globally
Slide 2: Forward-looking statements
2 This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development
Slide 3: Leader in Ophthalmology Gene Therapy
Pioneering biotechnology company leading the way to address major blindness diseases with novel modifier gene therapy Leader in Ophthalmology Gene Therapy
Slide 4: Introduction: Making an Impact Through Courageous Innovation
4 Introduction We're Here to Make an Impact Through Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies to Market and Working Even Harderto Provide Access to Patients Globally Respect Integrity Teamwork Accountability GMP Facility Headquarters, Malvern, Pennsylvania Values Ocuge
Slide 5: Targeting Three BLAs in Three Years
5 Targeting Three Biologics License Applications (BLAs) in Three Years Pipeline Phase I Phase II Phase III Target BLA/ MAA* Submission Program 300,000 (U.S. + EU) Gene-agnostic targeting >100 genes, broad indication ABCA4-associated retinopathies caused by 1,200+ mutations Advanced dry age-related m
Slide 6: Modifier Gene Therapy Platform
Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions Modifier Gene Therapy Platform
Slide 7: Problem: Millions living with retinal disease
7 Problem 3million+ people living with retinal disease globally Most retinal therapies treat symptoms ortarget single genes, leaving millions with progressive vision loss and no effective, lasting treatment options. No therapy available for many retinal diseases, specially inherited retinal diseases
Slide 8: Traditional therapy has limited therapeutic potential
Traditional therapy has limited therapeutic potential Traditional therapy can only target one single gene at a time, limiting therapeutic potential. Problem 8 of all human proteins are expressed in the retina genes are highly specialized to it, interacting through complex pathways mutations affectin
Slide 9: The Solution is a Gene-Agnostic Approach
9 The Solution is a Gene-Agnostic Approach Solution Our breakthrough technology is designed to address rare diseases and complex diseases Targeting master regulators Master regulators control entire gene networks. By targeting them, Ocugen’s gene therapy platform addresses the root cause of IRDs and
Slide 10: The Platform: Modifier Gene Therapy
10 The Platform Modifier Gene Therapy Platform AAV5 Carries Modifier Gene (M)* 1. Modifier Gene (M) Regulation of target gene Expression 2. Molecular Reset of the Gene Network Restoration of Molecular and cellular homeostasis 3. Retina Increased survival of retinal cells 4. * Modifier Genes: NR2E3 o
Slide 11: Modifier Gene Therapy: A Platform Technology Based on AAV5 Vector
Key Attributes of AAV5 vector: • Non-integrating Vector • Enhanced Tissue Specificity • High Transduction Efficiency • Long Term Gene Expression • Low Immunogenicity The Platform Product Highlights: • Novel Gene Agnostic Therapy • Broad Patient Eligibility • Durable Benefit • Favorable Safety & Tole
Slide 12: OCU400 Retinitis Pigmentosa (RP)
OCU400 Retinitis Pigmentosa (RP) Broad indication, gene-agnostic, targets 100+ genes
Slide 13: OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa
13 OCU400 First-in-Class Gene Therapy for Retinitis Pigmentosa Retinitis Pigmentosa Retinitis Pigmentosa (RP) is a group of rare, inherited retinal diseases caused by mutations in over 100 genes, leading to progressive vision loss and, in many cases, blindness. 1.6 million 2,000 1 Market Potential U
Slide 14: A Novel Modifier Gene Therapy for RP Patients
14 A Novel Modifier Gene Therapy for RP Patients OCU400 A gene-agnostic, broad-spectrum approach targeting retinal health at the root Potential curative therapy with a single subretinal injection using NR2E3 rd1 Rho -/- Rho P23H Preclinical data demonstrates activity across multiple retinal degenera
Slide 15: Preclinical studies demonstrating MOA
Preclinical studies demonstrating MOA Immunofluorescence (IF) Electroretinography Biomarkers: Molecular Reset Fundus Imaging Histology Ocugen - January 2026 15
Slide 16: Improved Patient Visual Function and Field in Phase 1/2 Trials
16 Improved Patient Visual Function and Field in Phase 1/2 Clinical Trials OCU400 *RHO +AR-NR2E3 Subjects (- Adverse Events, Sentinel); and Ceiling Effect (RHO) Subjects; ceiling effect (AR-NR2E3) # AR-NR2E3 Subjects: Baseline MLMT at 5 Lux level; 1Lux level improvement resulted in ceiling effect on
Slide 17: Long-Term Safety & Durability Data
17 Long-Term Safety & Durability Data OCU400 Improvement in visual function in treated eyes when compared to untreated eyes, demonstrates gene-agnostic Mechanism of Action 0 Serious Adverse Events Reported 100% treated evaluable subjects showed improvement or preservation invisualfunction compared t
Slide 18: Phase 3 liMeliGhT Trial Design
18 Phase 3 liMeliGhT Trial—Largest RP Data Set OCU400 Phase 3 Study Design Endpoints MTD Determined in Phase 1/2 Control Group No Treatment Treatment Group 2.5×1010 vg per eye 250 µL 100+ RP patients 2:1 ratio Visual function improvement in treated eye vs. control eye Assessed by Low Luminance Visua
Slide 19: OCU410ST Stargardt Disease
OCU410ST Stargardt Disease ABCA4 -associated retinopathies >1,200 mutations
Slide 20: OCU410ST First-in-Class Gene Therapy for Stargardt Disease
OCU410ST First-in-Class Gene Therapy for Stargardt Disease Stargardt Disease A rare IRD associated with 1,200+ mutations of the ABCA4 gene 1 million 0 Market Potential U.S. + EU globally suffer from ABCA4- approved treatments available associated retinopathies Regulatory Milestones (Completed/antici
Slide 21: Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit
21 Phase 1 GARDian1 Trial Demonstrated Clinically Meaningful Benefit OCU410ST Treated Eyes Untreated Eyes Atrophic Lesion Growth* 54% slower compared to untreated eyes Visual Function* 100% Stabilized or Improved compared to untreated eyes Nearly 1-line gain In visual acuity compared to untreated ey
Slide 22: GARDian3 Phase 2/3 Pivotal Confirmatory Trial
22 GARDian 3- Phase 2/3 Pivotal Confirmatory Trial OCU410ST Trial Design Endpoints Randomized 2:1 (N=51) DSMB 4-week Data Reviews Functional improvement in vision vs. control eye Assessed by LLVA and BCVA Target Population Early- to late-stage disease population Including pediatrics (3+ years) 12-mo
Slide 23: OCU410 Geographic Atrophy
OCU410 Geographic Atrophy Advanced dry age-related macular degeneration (dAMD)
Slide 24: OCU410 First-in-Class Gene Therapy for GA Patients
24 OCU410 First-in-Class Gene Therapy for GA Patients Geographic Atrophy Geographic Atrophy (GA) is an advanced form of dry AMD. GA causes irreversible degeneration of retina cells in the macula, leading to loss of central vision. ~8 million 2 Market Size U.S. + EU approved treatments available that
Slide 25: OCU410 (RORA): A Modifier Therapeutic Approach for dAMD
Anti-oxidative: Improves ARPE19 cells survival Anti-complement: Increased anti-compliment (Cd59) protein Anti-inflammatory: Suppresses inflammation in HMC3 cells Anti-drusen activity and improves retinal function 0 50 100 150 200 Blue Autofluorescence (AU) Months 1 7 1 7 1 7 Control Abca4-/- Uninjec
Slide 26: Phase 1 ArMaDa Trial Design
Phase 1 ArMaDa Trial OCU410 Trial Design Endpoints Dose Escalation (1:1:1) DSMB 4-week post-OCU410 Safety Reviews First Second 3 3 All Subjects 9 • Safety, AEs and SAEs •Indirect ophthalmoscopy •IOP Primary • Humoral/cellular immune response • Vector shedding Secondary 3 Medium Dose Group 5x 1010 vg
Slide 27: OCU410 Phase 1 Baseline Characteristics and Safety
OCU410 Phase 1—Baseline Characteristics and Safety No adverse or severe adverse events related to the study drug were reported including: • No development of exudation • No infectious endophthalmitis • No intraocular Inflammation • No anterior ischemic optic neuropathy (AION) • No vasculitis Mean ±
Slide 28: Phase 1 Treatment Shows Reduction in GA Lesion Growth
Phase 1—OCU410 Treatment shows reduction in GA Lesion Growth 28 Data points at 12M (N=7, 3 Low Dose, 2 Med Dose, 2 High Dose) 1 High dose subject with loss-to-follow up, and 1 Med dose subject with foveal detachment during surgery were not included in the analysis GA= Geographic Atrophy, SQRT= squar
Slide 29: Phase 1 Retinal Structural Preservation
Phase 1—Treatment Provides Retinal Structural Preservation by Reducing EZ Loss & Photoreceptor Degeneration 29 M=Months Data points at 12M (N=7, 3 Low Dose, 2 Med Dose, 2 High Dose) 1 High dose subject with loss-to-follow up, and 1 Med dose subject with foveal detachment during surgery were not incl
Slide 30: Phase 2 ArMaDa Trial Design
Trial Design Endpoints Randomization 1:1:1 DSMB 4-week Safety Reviews Change in LLVA from baseline at 12M Preservation of retinal tissue around areas of atrophy Change in GA lesion size Measured in mm2 by fundus autofluorescence (FAF) First Second 17 17 All Subjects 51 MTD Determined in Phase 1 17 C
Slide 31: Phase 2 Baseline Characteristics and Safety
Phase 2—Baseline Characteristics and Safety Age (Years) 75.96 ± 6.27 Male 20 (39.2) Female 31 (60.8) Mean ± SD Overall Study Mean ± SD Treated Eye Mean ± SD Control Eye BCVA Letters 53.96± 19.27 56.55 ± 18.93 47.15 ± 19.20 LLVA Letters 30.32±17.18 30.41 ± 18.30 30.07 ± 14.50 Lesion Size at BL (mm2)
Slide 32: Phase 2 Reduction in GA Lesion Growth at 12 Months
12 Months 0 1 2 3 4 GA Lesion Area (mm 2 ) Mean (±SEM) change from BL Control Treatment (OCU410) -46% BL M6 M12 0 1 2 3 4 GA Lesion Area (mm 2 ) Mean (±SEM) change from BL Control Treatment (OCU410) 46% Includes both foveal and subfoveal GA: geographic atrophy; Treated Eyes that received OCU410 Medi
Slide 33: Phase 2 Lesion Reduction by Dose at 12 Months
BL M6 M12 0 1 2 3 4 Timeline (Months) Control OCU410 - Medium Dose OCU410 - High Dose GA Lesion Area (mm 2 ) Mean (±SEM) change from BL -36% -54% 12 Months 0 1 2 3 4 GA Lesion Area (mm 2 ) Mean (±SEM) change from BL Control OCU410 - Medium Dose OCU410 - High Dose -54% -36% Greater reduction observed
Slide 34: Phase 2 Demonstrates Superior Clinical Effects in GA Patients
Phase 2—Demonstrates Superior Clinical Effects in GA Patients Significant Lesion Size Reduction Compared to Control Responder Rate to OCU410 Treatment 46% 22% OCU410 Approved Therapies with monthly injections1 Percentage Reduction in GA Lesion Growth (Treatment vs Control) Approved therapies benchma
Slide 35: OCU410 Transformative Gene Therapy for Geographic Atrophy
35 OCU410 Transformative Gene Therapy for Geographic Atrophy (GA) Comprehensive Mechanism of Action • Targets Multiple pathways and Demonstrated anti-oxidative, anti-complement, anti-inflammatory, and anti-drusen activity in preclinical models Clinical Efficacy Data • 46% reduction in treated (high
Slide 36: Executive Summary: Anticipated Near-Term Milestones
36 Executive Summary Anticipated Near-Term Targeted Milestones RP Stargardt Disease GA OCU400 OCU410ST OCU410 100% Enrollment Completion Interim data Initiate Rolling BLA Submission Phase 3 topline data Phase 2 Study Results Initiate Phase 3 2026 2027 1Q 2Q 3Q 4Q 1Q 2Q Phase 3 top line data BLA Subm
Slide 37: We're advancing blindness cures. Seeking partners.
We’re advancing blindness cures. Seeking partners who share our vision. Reach Out BD@ocugen.com