Slide 1: 1Q 2025 Business Update
—_—_ ~ ocugen. _ Courageous Innovation Dedicated to Bringing Game-Changing Gene Therapies, Cell Therapies, and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally 1Q 2025 Business Update May 9, 2025
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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
Slide 3: Potential for Three Biologics License Applications (BLAs) in the Next Three Years
Potential for Three Biologics License Applications (BLAs) in the Next Three Years Product Candidate Status BSE Target Ria Prevalence Submission ocu400 Phase 3 in Progress Retinitis Pigmentosa (RP) (Largest Orphan Gene Therapy 300,000 2026 (Gene-agnostic targeting Clinical Trial) >100 genes, broad in
Slide 4: OCU400 First-in-Class Novel Modifier Gene Therapy for RP
OCU400 First-in-Class Novel Modifier Gene Therapy for RP Unmet Need ~300,000 people in the U.S./EU and 1.6 million globally suffer from RP, which affects >100 genes One approved product (Luxturna®), a gene therapy, which can address only one gene (RPEG65) Potential Solution OCU400 uses a gene-agnost
Slide 5: Safety and Durability of Visual Function (LLVA) - Long-Term Follow-up
Safety and Durability of Visual Function (LLVA) - Long-Term Follow-up Multiple Mutations* (N=10) RHO(N=8) * No Severe Adverse Events (SAE) related to OCU400 p=0.021 p=0.055 have been reported ° o u lo iS 3 a ° Mean ALLVA (+SEM) from Baseline a Mean ALLVA (+SEM) from Baseline ° a 100% (10/10) treated
Slide 6: OCU400 Phase 3 liMeliGhT Trial: Design and Key Criteria
OCU400 Phase 3 liMeliGhT Trial: Design and Key Criteria OCU400 Phase 3 MTD established from Phase 1/2 trial Randomization 2:1 Arms Dose Control Group No treatment Treatment Group 2.5x10"° vg/eye in 250 pL — ocugen. nes Study Endpoints Primary Efficacy Endpoint: * Change in functional vision of respo
Slide 7: OCU410ST First-in-Class Novel Modifier Gene Therapy Product for Stargardt Disease
OCU410ST First-in-Class Novel Modifier Gene Therapy Product for Stargardt Disease Unmet Need ~100,000 people in U.S./EU and 1 million globally suffer from ABCA4-associated retinopathies' (>1,200 mutations). Currently there are no approved products in the market. Potential Solution OCU410ST has poten
Slide 8: Improvement of Visual Function in OCU410ST Treated vs Untreated Eyes
Improvement of Visual Function in OCU410ST Treated vs Untreated Eyes — ocugen. woes 10 ra -e TreatedEye -® Untreated Eye > Od Improvement Mo Sf ee || from Baseline i) = 1 OE | D ce a Stabilizati gr Baseline ~~__ M3 Mey eehetten On a cf sf CESS gO Decrease 3 r from Baseline 6/6 (100%) evaluable treat
Slide 9: • • 6M 0.0 0.2 0.4 0.6 0.8 Timeline Treated Eye Untreated Eye 54%
• • 6M 0.0 0.2 0.4 0.6 0.8 Timeline Treated Eye Untreated Eye 54%
Slide 10: Clinical Study Design for Phase 2/3 Pivotal Confirmatory BLA Trial
Clinical Study Design for Phase 2/3 Pivotal Confirmatory BLA Trial Phase 2/3 Pivotal Confirmatory Trial MTD established from Phase 1 trial — ocugen. — Randomization 2:1 Phase 2/3 Dose Vg/ml Control Group Control Group nei7 No treatment Treatment Group 1.5*10" vg/mL Study Endpoints Primary Efficacy E
Slide 11: OCU410 First-in-Class Novel Modifier Gene Therapy for GA
OCU410 First-in-Class Novel Modifier Gene Therapy for GA Unmet Need ~19 million people in U.S. and EU and 260 million globally suffer from dAMD' ~2-3 million people in U.S. & EU and ~8 million globally suffer from GA—advanced dAMD? Currently, approved products target only one disease pathway associa
Slide 12: Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes
Preservation of Visual Function (LLVA) in OCU410 Treated vs Untreated Eyes Stabilization Treated eyes demonstrate . 4-line (23-letter*) gainin the 2 — visual acuity when | from Bassline compared to untreated 15 fellow eyes -e Treated Eye -=- Untreated Eye Mean change in LLVA (ETDRS) from Baseline 3
Slide 13: Substantial Slowing of GA Lesion Growth After Single Sub-Retinal Injection
Substantial Slowing of GA Lesion Growth After Single Sub-Retinal Injection o 25: & 4 Natural History Studies BS 29] > Untreated Eye a a E ~® Treated Eye ie bgt </ a3 ia . . a2 10 AZ — ° 41% slower GA lesion growth in ° LF Se os ie treated eye versus fellow eyes s . ee . 2 after single injection at 1
Slide 14: OCU410 Treatment Preserves Retinal Tissue in GA Lesions
OCU410 Treatment Preserves Retinal Tissue in GA Lesions o8 07 2 @ ° a Pe a Single sub-retinal injection of OCU410 at 12M is more effective when compared to PM and PEOM treatment 2 rs ° is 2 0.4 Os 0.76 Difference in Mean GA lesion Size between Treated and Untreated Eyes (mm?) ° ° @ PEOM (12M) PM (12
Slide 15: OCU410 Phase 2 Study: Design and Key Criteria
OCU410 Phase 2 Study : Design and Key Criteria MTD established from e 1 trial Primary Endpoint Phase2 * Change in GA lesion size (mm?) using Randomization 1:1:1 Fundus Autofluorescence Phase 2 Study Endpoints Medium-dose High-dose Group cup Control Group n=i7 ni? n=17 Secondary Endpoint * Change in
Slide 16: Update on Additional Technologies
Update on Additional Technologies OCU200 NeoCart® OCUSO0O0 — ocugen. ssn Therapeutic Focus: diabetic macular edema (DME), diabetic retinopathy (DR), and wet AMD: total prevalence = 130 million Differentiator: Targets underlying disease mechanisms through the integrin pathway and holds promise to pro
Slide 17: Financial Update
Financial Update
Slide 18: Financial Update
Financial Update Three months ended March 31, Statement of Operations 2025 2024 Research and development expense $9.5 $6.8 General and administrative expense 65: 6.4 Interest (expense) income, net (0.9) 03 Net loss $015.3) $011.9) Net loss per share of common stock — basic and diluted $(0.05) $(0.05
Slide 19: Questions & Answers
Questions & Answers
Slide 20: Near-Term Targeted Milestones—2025
Near-Term Targeted Milestones—2025 * Complete OCU400 Phase 3 recruitment and continue to monitor patients to ensure BLA/MAA filings are complete in mid-2026 * Provide interim Phase 2 data from OCU410 in fall 2025, with full data in the first quarter of 2026 * — Initiate OCU410ST Phase 2/3 pivotal co
Slide 21: Ocugen Vision
Ocugen” Vision Fully integrated, patient-centric biotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation — ocugen. wees