Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Retina Forum 2026
15 slides from Opus Genetics's conference presentation, Sep 30, 2026
Slide 1: Clinical Momentum and Pipeline Progress at Opus Genetics
Clinical Momentum and Pipeline Progress at Opus Genetics Sally Tucker, MCOptom, PhD Retina Forum 2026 September 30, 2026
Slide 2: Disclosures and Forward-Looking Statements
Disclosures and Forward-Looking Statements Certain statements contained in this presentation are not statements of historical fact and are 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, a
Slide 3: Building a Differentiated Gene Therapy Platform
Building a Differentiated Gene Therapy Platform Building a Differentiated Gene Therapy Platform Preclinical IND-enabling Phase 1/2 Phase 3 Approval OPGx-LCA5 LCA co-funded by FDA OOPD OPGx-BEST1 Bestrophinopathies OPGx-RDH12 LCA co-funded by Global RDH12 Alliance OPGx-MERTK RP co-funded by FFB RD Fu
Slide 4: OPGx-LCA5 Gene Therapy is Designed to Restore a Key Protein of the Visual Cycle
OPGx-LCA5 Gene Therapy is Designed to Restore a Key Protein of the Visual Cycle • Lebercilin is a ciliary protein critical for the function of photoreceptor inner and outer segments1 OPGx-LCA5 Gene Therapy • In LCA5 patients, photoreceptor function is severely impaired due to a lack of functioning V
Slide 5: Ongoing Phase 1/2 Study: Visual Acuity Maintained in Adults Over 24 Months
Ongoing Phase 1/2 Study: Visual Acuity Maintained in Adults Over 24 Months and Improved Over 6 Months in Pediatric Cohort M e a n C h a n g e f r o m B a s e l i n e i n VA M e a n C h a n g e f r o m B a s e l i n e i n VA Adu lt Coh ort (N =3) Pediat ric Cohort (N=3) -0.6 -0.6 Mean CFB in VA (logM
Slide 6: Ongoing Phase 1/2 Study: Full-Field Stimulus Test Demonstrated Durable Vision Improvement
Ongoing Phase 1/2 Study: Full-Field Stimulus Test Demonstrated Durable Vision Improvement Mean CFB in Red and B lue FST Mean CFB in Red and B lue FST Adul t Cohort (N= 3) Pedi at r ic Cohort ( N=3) -2 -2 [log phot-cd.s.m-2] -1.6 [log phot-cd.s.m-2] -1.6 CFB in Red FST CFB in Red FST -1.2 -1.2 -0.8 -
Slide 7: Ongoing Phase 1/2 Study: Microperimetry Data Provides Evidence of Increased Sensitivity
Ongoing Phase 1/2 Study: Microperimetry Data Provides Evidence of Increased Sensitivity and Movement of Fixation Toward the Fovea BL BL M12 M6 M24 M12 Adult Participant 01-04 Pediatric Participant 01-06 7 BL, baseline; M, month.
Slide 8: OPGx-BEST1 Gene Therapy is Designed to Restore Retinal Ion Homeostasis
OPGx-BEST1 Gene Therapy is Designed to Restore Retinal Ion Homeostasis • BEST1 mutations disrupt cellular ion and fluid homeostasis resulting in electrophysiological OPGx-BEST1 Gene Therapy abnormalities, RPE dysfunction, and retinal degeneration Vector AAV2 • Most bestrophinopathies exhibit a slow
Slide 9: Participant Demographics: Low-Dose Cohort 1
Participant Demographics: Low-Dose Cohort 1 101-101 101-104 102-101 102-102 101-106 Ag e 63 59 50 45 31 Se x Female Female Male Male Male BE ST ph enotype ARB ARB BVMD BVMD BVMD Ba seline VA * (study eye † ) 1 50 49 61 47 Ba seline VA * (fellow eye) 43 65 68 72 56 Follow-up duration 6 months 6 mont
Slide 10: Ongoing Phase 1/2 Study Safety and Efficacy Summary: Low Dose Cohort 1 at Month 3
Ongoing Phase 1/2 Study Safety and Efficacy Summary: Highly Encouraging Proof-of-Concept Results from Low Dose Cohort 1 at Month 3 Safety • OPGx-BEST1 was well-tolerated in 5/5 participants, with no SAEs or DLTs • 80% of participants (4/5) had structural improvements: Structural • BVMD: 67% of parti
Slide 11: Participant 101-106 (BVMD): Vitelliform Material Reduction Co-localized with Microperimetry Improvements
Participant 101-106 (BVMD): Vitelliform Material Reduction Co- localized with Microperimetry Improvements Baseline Vitelliform lesions reduced Month 3 Baseline Mont h 3 Lesion reduction consistent with improved function of RPE cells and treatment activity as indicated in microperimetry improvements
Slide 12: Current Cash to Support Multiple Clinical Inflection Points
Current Cash to Support Multiple Clinical Inflection Points 2027 Current cash runway extends into BEST1 Pivotal 2029, funding five clinical programs Q2 2027 Study Start through multiple critical inflection points BEST1 Cohort 2 Q1 2027 results MERTK clinical study initiation Q4 2026 RDH12 clinical Q
Slide 13: A Conversation with Lindsay Rambo, a Patient Living with LCA5
A Conversation with Lindsay Rambo, a Patient Living with LCA5: What do you wish people could feel or experience to truly understand the potential impact of treatment?
Slide 14: Lindsey Rambo
Lindsey Rambo
Slide 15: Thank you
Thank you OPUS iG E NN T Aes