Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Corporate Update (October 2026)
12 slides from Opus Genetics's corporate update, Oct 15, 2026
Slide 1: Gene Therapy For Congenital Leber Amaurosis (LCA5)
Gene Therapy For Congenital Leber Amaurosis (LCA5) Dr. Rodrigo Jorge Ribeirão Preto Medical School University of São Paulo - Brazil
Slide 2: LCA5: Early-onset, Severe IRD with Early Vision Loss
LCA5: Early-onset, Severe IRD with Early Vision Loss Overview & prevalence Very early onset severe disease typically presenting in infancy or early childhood1 LCA5 represents ~2% of all LCA cases1 Global prevalence*: ~3,240 patients2 U.S. prevalence: ~170 patients2 Clinical features1,3 Pigmentary re
Slide 3: 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 OPGx-LCA5 Gene Therapy Vector AAV8 Delivery Single subretinal injection Status Phase 1/2 ongoing Phase 3 run-in ongoing Phase 3 dosing initiation expected Q4 2026 Lebercilin is a ciliary protein critical for function of
Slide 4: Clinical Development Status
Clinical Development Status 4 PART B: Natural history in untreated participants PART C: Eligible Part B participants continue to Part C Treatment initiation expected Q4 2026 Primary efficacy endpoint: Mean retinal sensitivity improvement of >7 dB across central 16 test loci LCA5, Leber congenital
Slide 5: Clinical Development Status
Clinical Development Status 5 PART B: Natural history in untreated participants PART C: Eligible Part B participants continue to Part C Treatment initiation expected Q4 2026 Primary efficacy endpoint: Mean retinal sensitivity improvement of >7 dB across central 16 test loci LCA5, Leber congenital
Slide 6: Phase 1/2 Ongoing Study: Visual Acuity Improved in Adults and Pediatric Cohort
Phase 1/2 Ongoing Study: Visual Acuity Improved in Adults Over 24 Months and in the Pediatric Cohort Over 6 Months 6 Improvement Improvement Mean Change from Baseline in VA Adult Cohort (N=3) Mean Change from Baseline in VA Pediatric Cohort (N=3) BL, baseline; CFB, change from baseline; M, month; VA
Slide 7: Full-field Stimulus Testing (FST)
Full-field Stimulus Testing (FST) Measures dimmest flash seen across the whole visual field, separating rod and cone sensitivity Setup: Dilated, dark-adapted eyes (>30 min); one eye tested at a time in a Ganzfeld dome Method: Forced-choice “seen / not seen” responses to random flashes at varying lum
Slide 8: Phase 1/2 Ongoing Study: Full-Field Stimulus Test Demonstrated Durable Vision Improvement
Phase 1/2 Ongoing Study: Full-Field Stimulus Test Demonstrated Durable Vision Improvement 8 BL, baseline; CFB, change from baseline; FST, Full-field stimulus test; M, month. Mean CFB in Red and Blue FST Adult Cohort (N=3) Mean CFB in Red and Blue FST Pediatric Cohort (N=3)
Slide 9: Phase 1/2 Ongoing Study: Microperimetry Data Provides Evidence of Increased Sensitivity
Phase 1/2 Ongoing Study: Microperimetry Data Provides Evidence of Increased Sensitivity and Movement of Fixation Toward the Fovea Microperimetry is a detailed, eye-tracking-assisted visual field test that creates a retinal sensitivity map of the macula by testing a patient's response to light at spe
Slide 10: Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment
Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment 01-01: Able to identify her children within a larger group of children one month after surgery, which she previously could not do Adult Participants 01-03: Had no formative vision prior to treatment. Able to see his newborn niece for t
Slide 11: 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 this treatment?
Slide 12: Thank you
Thank you Gene Therapy For Congenital Leber Amaurosis (LCA5) Dr. Jorge Rodrigo Ribeirão Preto Medical School University of São Paulo - Brazil