Opus Genetics, Inc. · NASDAQ:IRD
Opus Genetics — Technical Presentation (June 2026)
80 slides from Opus Genetics's technical presentation, Jun 16, 2026
Slide 1: Opus Genetics R&D Science Forum
Opus Genetics R&D Science Forum June 16, 2026 | 10:00 AM – 12:00 PM ET
Slide 2: 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, as amended. Forward-looking statements give
Slide 3: Meet Our Speakers
Meet Our Speakers George Magrath, MD Chief Executive Officer Ben Yerxa, PhD President Sally Tucker, PhD Chief Medical Officer Ash Jayagopal, PhD, MBA Chief Scientific & Development Officer Joe Schachle, MBA Chief Operating Officer Jean Bennett, MD, PhD University of Pennsylvania Kenneth Fan, MD, MBA
Slide 4: Agenda – Part 1
Agenda – Part 1 Topic Speaker(s) Company Introduction George Magrath, MD Jean Bennett, MD, PhD RDH12 Kenneth Fan, MD, MBA Ash Jayagopal, PhD MERTK Professor Robert MacLaren Ash Jayagopal, PhD RHO Lejla Vajzovic, MD Ash Jayagopal, PhD Clinical Development Strategy Sally Tucker, PhD Q&A Ben Yerxa, PhD
Slide 5: Agenda – Part 2
Agenda – Part 2 Topic Speaker(s) LCA5 Bart Leroy, MD, PhD BEST1 Ash Jayagopal, PhD George Magrath, MD IRD Patient Journey and Disease Prevalence Joe Schachle, MBA Patient Recruitment & Retention Panel Ben Yerxa, PhD (Moderator) Jean Bennett, MD, PhDTodd Durham, PhD, MS Bart Leroy, MD, PhD Q&A Ben Ye
Slide 6: Company Introduction
Company Introduction George Magrath, MD Chief Executive Officer Opus Genetics
Slide 7: The Opus Opportunity: Fully-Funded to Advance IRD Clinical Programs
The Opus Opportunity: Fully-Funded to Advance 5 IRD Clinical Programs Portfolio approach produces multiple data readouts and milestones Follow-on treatments from the first approved IRD gene therapy Broad IP protection and eligible for Orphan Drug exclusivity Cost-effective development: Efficient pro
Slide 8: IRD Mutations of Interest
IRD Mutations of Interest Targeting Seven IRD-Causing Gene Mutations Inherited Retinal Disorders Retinitis Pigmentosa Leber Congenital Amaurosis Bestro-phinopathies ADRP RHO LCA5 NMNAT1 BEST1 BVMD ARB LCA LCA Targeted Types of IRDs Targeted IRD Disease Subtypes Targeted IRD-Causing Gene Mutations AR
Slide 9: Building a Differentiated Gene Therapy Pipeline
Preclinical IND-enabling Phase 1/2 Phase 3 Approval Building a Differentiated Gene Therapy Pipeline Opus Genetics owns worldwide rights to all gene therapy programs.adRP, autosomal dominant retinitis pigmentosa; BEST1, bestrophin 1; CNGB1, cyclic nucleotide-gated channel β1; FDA OOPD, Food and Drug
Slide 10: Fully-Funded to Support Multiple Clinical Inflection Points
10 Fully-Funded to Support Multiple Clinical Inflection Points 2H 2027 RHO clinical study initiation Q1 2027 MERTK clinical study initiation Q4 2026RDH12 clinical study initiation Q4 2026LCA5 Phase 3 dosing initiation Oct 2026PDUFA date for Phentolamine sNDA Sept 2026 BEST1 Cohort 1 3-month results
Slide 11: Scientific Overview
Scientific OverviewJean Bennett, MD, PhDF.M. Kirby Emeritus Professor of Ophthalmology Perelman School of Medicine, University of PennsylvaniaPhiladelphia, PA
Slide 12: Inherited Retinal Degeneration Disease Target Selection
12 Inherited Retinal Degeneration Disease Target Selection* *Assumes gene has been cloned, characterized and intervention is gene-specific. Optimal Targets Developmental conditions Large genes Slowly progressive disease Natural history unknown Asymmetric disease Extraordinarily rare Can target affec
Slide 13: Structure-Function Dissociation: The Clinical Imperative
13 Treat the function to reverse pathology and restore or preserve vision Structure-Function Dissociation: The Clinical Imperative Targeting Diseases Where the Structure is Intact Retinal structure is relatively preservedeven though visual function is already impaired This creates a “therapeutic win
Slide 14: Inspiration from Patients and Families
14 Inspiration from Patients and Families LCA-RDH12 Kids LCA-LCA5 and LCA-RDH12: Ciliopathies Rare Imaging: Treatable photoreceptors in childhood, young adults Animal & cell models Compared to RPE65: More severe Earlier onset 1st to be identified with LCA5 LCA5, Leber congenital amaurosis 5; RPE65,
Slide 15: Patient-Academic Partnerships and Opus Genetics: The LCA5 Story
15 Patient-Academic Partnerships and Opus Genetics: The LCA5 Story Identification of LCA5 (Nijmegen, 2007) Proof-of-Concept: LCA5 Gene Therapy (2018) Lca5-/- Mouse (P. Nishina) LCA5 Consortium (2011) GMP AAV LCA5 Clinical Trial Initiated (2023) 1990 1995 2000 2005 2025 2020 2015 2010 AAV, adeno-asso
Slide 16: Abundant Safety Data
Abundant Safety Data >140 retinal gene therapy clinical trials initiated Gene therapy centers around the world Thousands of eyes have been injected Numerous disease targets Variety of strategies Excellent safety data Familiarity with gene therapy surgical procedures, vector handling and storage, gen
Slide 17: Numerous Obstacles to Retinal Gene Therapy Have Been Overcome
> In the 2000’s No path/regulatory issues De-risk safety of subretinal AAV Accurate dosing (prevent AAV adherence) Surgical delivery Lack of genotyped patients Pediatric subjects Immune status FDA requirements: Injection of contralateral eye Control group Lack of natural history data Lack of relevan
Slide 18: RDH12 Overview
RDH12 Overview Kenneth Fan, MD, MBA Retina Consultants of Texas Houston, TX
Slide 19: RDH12: An Early-Onset Inherited Retinal Degeneration
Overview & prevalence Mutations in RDH12 cause a severe form of LCA, which leads to early, rapid vision loss in infancy/childhood, often resulting in legal blindness before the third decade of life1 Most frequent phenotype of RDH12 is autosomal recessive/LCA2 Accounts for ~3.5-10.5% of all LCA cases
Slide 20: RDH12 is a Critical Protector of Photoreceptor Health and Function
RDH12 is an enzyme expressed specifically in photoreceptor inner segments1 Plays a key role in clearing toxic byproducts, such as all-trans-retinal, created during the visual process1 When RDH12 is not functioning properly, toxic substances build up and damage retinal cells, leading to progressive v
Slide 21: RDH12 Structural Changes Define the Therapeutic Window
Longitudinal studies of RDH12 patients suggest relatively preserved visual acuity with steep decline in second decade of life Generally, VA maintained to ~20/200 or better for first two decades Thereafter, VA steeply declines toward finger counting, light perception only Functional signals remain
Slide 22: OPGx-RDH12 Scientific Overview
OPGx-RDH12 Scientific Overview Ash Jayagopal, PhD, MBA Chief Scientific and Development Officer Opus Genetics
Slide 23: OPGx-RDH12 Designed to Restore a Key Component of the Visual Cycle
Success with LUXTURNA® indicates potential restoration of the retinoid cycle can rescue retinal function Established AAV8 vector, codon optimized transgene, and specific promoter targets photoreceptors Designed to restore visual cycle function in the inner segments of photoreceptors OPGx-RDH12 Gene
Slide 24: OPGx-RDH12 Delivered a Functional RDH12 Enzyme In Vivo
OPGx-RDH12 restored RDH12 protein expression and enzymatic activity in an RDH12 deficient mouse model (RDH12KO) in a dose-dependent manner Restoration of RDH12 expression and function was achieved in RDH12KO mice (3E10 vg/eye) OPGx-RDH12 Delivered a Functional RDH12 Enzyme In Vivo Substrate Conversi
Slide 25: OPGx-RDH12 Gene Therapy Restored Structure and Function
RDH12KO mice lack retinal phenotype observed in human RDH12 IRD However, mice are susceptible to bright light induced retinal degeneration vs. WT mice1 AAV8 encoding for hRDH12 in RDH12-/- mouse models of light induced retinal degeneration restored structure and function, supporting potential clinic
Slide 26: MERTK Overview
MERTK Overview Professor Robert MacLarenUniversity of Oxford Oxford, England
Slide 27: MERTK: Early-onset Inherited Retinal Disease with Rapid Vision Loss
MERTK: Early-onset Inherited Retinal Disease with Rapid Vision Loss *Global prevalence estimate includes United States, EU4 (France, Spain, Germany, & Italy), UK, Middle East/North Africa, and China. MERTK, MER proto-oncogene tyrosine kinase; RP, retinitis pigmentosa. 1. Audo I, et al. Hum Mutat. 20
Slide 28: MERTK is the Metabolic Gatekeeper in the Retina
MERTK is a receptor tyrosine kinase highly expressed on RPE cells Essential for phagocytosis of photoreceptor outer segments by the RPE Loss of MERTK function results in impaired phagocytosis, debris accumulation, leading to eventual photoreceptor death and progressive vision loss 28 MERTK is the Me
Slide 29: Functional Loss Precedes Structural Degeneration
Early and reduced rod photoreceptor function with cones affected later1 Significant, often asymmetric, reduction in VA usually occurs by teenage years, followed by progressive VA loss through adulthood1 MERTK-related IRD is considered a prime candidate for gene therapy because the underlying structu
Slide 30: OPGx-MERTK Scientific Overview
OPGx-MERTK Scientific Overview Ash Jayagopal, PhD, MBA Chief Scientific and Development Officer Opus Genetics
Slide 31: OPGx-MERTK Designed to Restore RPE Metabolic Functions
Vector AAV2 Delivery Single subretinal injection Same AAV2 capsid used in LUXTURNA® OPGx-MERTK is designed to restore critical RPE metabolic functions needed for retinal homeostasis RPE-specificity conferred by promoter technology 31 OPGx-MERTK Gene Therapy is Designed to Restore Critical Retinal Pi
Slide 32: Preclinical Studies Show Retinal Preservation of Structure and Function
32 Preclinical Studies Provide Evidence of Retinal Preservation of Structure and Function AAV2, adeno-associated virus serotype 2; hMERTK, human MER proto-oncogene tyrosine kinase; ONL, outer nuclear layer. LaVail MM, et al. Adv Exp Med Biol. 2016;854:487-93. Mertkkd mice exhibited rapid loss of pho
Slide 33: OPGx-MERTK Preserved ONL Thickness in RCS Rat Model
33 OPGx-MERTK Preserved ONL Thickness in RCS Rat Model of Retinal Degeneration RCS naturally-occurring rat model of MERTK models the structural and functional time course of human MERTK degeneration Exhibits MERTK deficiency in RPE cells, which enables testing of gene therapy interventions OPGx-MERT
Slide 34: RHO Overview
RHO Overview Lejla Vajzovic, MD Duke University Durham, NC
Slide 35: RHO: Highly Variable, Often Slowly Progressive Rod-Cone Dystrophy
Overview & prevalence Autosomal dominant RP is the most common autosomal dominant retinal disease1 More than 290 identified RHO mutations1 Variants in RHO are the most frequent cause of adRP (~20-30% of cases) 1 Global prevalence*: ~30,200 patients2 U.S. prevalence: ~8,800 patients2 Clinical feature
Slide 36: Misfolded Rhodopsin Drives Photoreceptor Stress and Degeneration
Misfolded or Dysfunctional Rhodopsin Drives Photoreceptor Stress and Progressive Rod-Cone Degeneration RHO encodes for rhodopsin, a rod photoreceptor 11-cis-retinaldehyde photopigment located in the outer segment, critical for phototransduction Mutations in RHO can cause the rhodopsin protein to mis
Slide 37: Progressive Peripheral Field Loss Followed By Central Vision Decline
Typical pattern of rod degeneration beginning in childhood (accompanied by night blindness) with subsequent degeneration of cones1 Two distinct phenotypes1,2 Class A: Severely abnormal rod function from early life, cystoid changes common Class B: Maintain rod function into adulthood, structural dege
Slide 38: OPGx-RHO Scientific Overview
OPGx-RHO Scientific Overview Ash Jayagopal, PhD, MBA Chief Scientific and Development Officer Opus Genetics
Slide 39: OPGx-RHO is Designed to Silence and Replace
Vector AAV5 Delivery Single subretinal injection 39 OPGx-RHO Gene Therapy is Designed to “Silence and Replace” in Autosomal Dominant RHO Silence (shRNA820) and replace (RHO820) strategy Use of AAV5 and rod-specific promoter Targets expression of codon-optimized human RHO transgene to rod and cone ph
Slide 40: Canine Model of RHO adRP as a Test Bed
Canine Model of RHO adRP as a Test Bed for Therapeutic Studies Canine model of RHO with naturally-occurring heterozygous T4R mutation provides opportunity to test therapeutic interventions Slow recovery of rod function after light exposure Mirrors pattern of retinal degeneration in human adRP Suscep
Slide 41: OPGx-RHO: Knockdown and Replacement in T4R adRP-RHO Canines
41 OPGx-RHO: Knockdown and Replacement in T4R adRP-RHO Canines adRP, autosomal dominant retinitis pigmentosa. Cideciyan AV, et al. Proc Natl Acad Sci USA. 2018;115:E8547-E8556.
Slide 42: OPGx-RHO Maintained Rod Morphology in TgP23H Retinas
42 OPGx-RHO Maintained Rod Morphology in TgP23H Retinas Dose-dependent preservation of rod morphology observed at 20-31 weeks post injection of OPGX-RHO 1.5 x 1010 vg/eye (“mid-low dose”) estimated to be minimal effective dose for rod preservation using immunofluorescence analysis Data on file.
Slide 43: OPGx-RHO Maintained Cone Morphology in TgP23H Retinas
43 OPGx-RHO Maintained Cone Morphology in TgP23H Retinas Dose-dependent preservation of cone morphology observed at 20-31 weeks post injection of OPGX-RHO 1.5 x 1010 vg/eye (“mid-low dose”) estimated to be minimal effective dose for cone preservation using immunofluorescence analysis Data on file.
Slide 44: Clinical Development Strategy
Clinical Development Strategy Sally Tucker, PhD Chief Medical Officer Opus Genetics
Slide 45: Targeting IRDs with Structure-Function Dissociation
45 Targeting IRDs with Structure-Function Dissociation Bestrophinopathies LCA RP Age of onset Childhood (ARB) to adulthood (BVMD) Birth or infancy Adolescence to adulthood Progression Often slow and variable progression Severe, early disease and variable progression Progression over decades Symptomo
Slide 46: Signs and Symptoms Directly Inform Selection of Key Endpoints
46 Signs and Symptoms Directly Inform Selection of Key Endpoints Signs Vessel Narrowing, Pigment Retinopathy, Optic Disc Pallor, RPE Changes ONL Thinning, Ellipsoid Zone Disruption, IRF/SRF Fluid, Macular Atrophy Symptoms Decreased central vision Night blindness Visual field loss / tunnel vision Nys
Slide 47: Clinical Studies Follow a Dose Exploration, Data-Driven Approach
47 Clinical Studies Follow a Dose Exploration, Data-Driven Approach Cohort 1 – Low Dose 5-6 participants; Adults (and adolescents) Early efficacy signal allows conversion to a pivotal study Safety; evaluate efficacy signal at higher dose Cohort 2/3 –Mid/High Dose 5-6 participants; Adults (and adoles
Slide 48: Targeting Initiation of Clinical Testing in All Three Programs
48 Targeting Initiation of Clinical Testing in All Three Programs within the Next Year Program Region Study Initiation RDH12 U.S. Q4 2026 MERTK Abu Dhabi Q1 2027 RHO Global locations 2H 2027 Clinical development timelines are based on current estimates and are subject to change. MERTK, MER proto-onc
Slide 49: Our Strategic Partnerships Provide Path to Accelerated Recruitment
49 Our Strategic Partnerships Provide Path to Accelerated Recruitment Collaborative platform uniting key patient advocacy groups dedicated to RDH12-related IRDs RDH12 Fund for Sight in the U.S. Eyes on the Future in the UK Partnership with Abu Dhabi Department of Health; MERTK clinical development a
Slide 50: Patient Recruitment and Retention Strategies
Patient Recruitment and Retention Strategies Patient education videos Increase awareness of IRDs and study activities Clinical trial participation video series Observational study initiation (prior to treatment) to proactively identify IRD patients Patient engagement Involvement in family days and e
Slide 51: Patient Commitment to Expanding Awareness of Disease & Treatment
51 Patient Commitment to Expanding Awareness of Disease & Treatment
Slide 52: Q&A
Q&A Moderated by Ben Yerxa President Opus Genetics
Slide 53: OPGx-LCA5 Update
OPGx-LCA5 Update Bart Leroy, MD, PhD Ghent University Hospital Ghent, Belgium
Slide 54: 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 55: OPGx-LCA5 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 55 Lebercilin is a ciliary protein critical for the function of photoreceptor inner and outer segments1 In LCA5 patients, photoreceptor function is
Slide 56: Visual Acuity Maintained in Adults and Improved in Pediatric Cohort
Visual Acuity Maintained in Adults Over 24 Months and Improved Over 6 Months in Pediatric Cohort 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, visual acuity. 56
Slide 57: Full-Field Stimulus Test Demonstrated Durable Vision Improvement
Full-Field Stimulus Test Demonstrated Durable Vision Improvement 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) 57
Slide 58: Microperimetry Data Shows Increased Sensitivity
Microperimetry Data Provides Evidence of Increased Sensitivity and Movement of Fixation Toward the Fovea Adult Participant 01-04 Pediatric Participant 01-06 Microperimetry is a detailed, eye-tracking-assisted visual field test that creates a retinal sensitivity map of the macula by testing a patient
Slide 59: Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment
Powerful Participant-Reported Outcomes After OPGx-LCA5 Treatment 59 Adult Participants 01-01: Reported being able to identify her children within a larger group of children 1 month after surgery which she previously could not have done. 01-03: Had no formative vision prior to treatment. Reported bei
Slide 60: BEST1 Update
BEST1 Update Ash Jayagopal, PhD, MBA Chief Scientific and Development Officer Opus Genetics
Slide 61: BEST1: Group of IRDs with a Range of Onset and Slow Progression
61 BEST1: Group of IRDs with a Range of Onset and Slow Rate of Progression Overview & prevalence Mutations in BEST1 have been associated with at least five clinically distinct retinal degenerative diseases, with onset from childhood to adulthood1 Accounts for ~3.5% of all IRDs1 Global prevalence*: ~
Slide 62: BEST1 Disease Biology
BEST1 gene encodes for bestrophin-1, a homopentameric (i.e. 5 identical monomers) Ca2+-activated chloride channel required for RPE maintenance and retinal physiology BEST1 mutations disrupt cellular ion and fluid homeostasis resulting in electrophysiological abnormalities, RPE dysfunction, and retin
Slide 63: Pathogenic Mechanisms of BEST1 Variants
BEST1, bestrophin 1 gene; ARB, autosomal recessive bestrophinopathy; BVMD, Best vitelliform macular dystrophy; ADVIRC, autosomal dominant vitreoretinochoroidopathy. Haldrup SB, et al. Int J Mol Sci. 2025;26(19). 63 Pathogenic Mechanisms of BEST1 Variants ↓Reduced protein expression, trafficking, or
Slide 64: Most BEST1 Variants May be Amenable to Gene Augmentation
Cl- Cl- Cl- Cl- Cl- Cl- Cl- Cl- Abnormal ion flux Cl- Cl- Cl- Cl- Cl- Cl- Cl- BEST1, bestrophin 1 gene; ARB, autosomal recessive bestrophinopathy; BVMD, Best vitelliform macular dystrophy; ADVIRC, autosomal dominant vitreoretinochoroidopathy. Haldrup SB, et al. Int J Mol Sci. 2025;26(19). 64 Most BE
Slide 65: Paradigm for Guiding Treatment Decisions
*Treatment-response assessment conducted on applicable mutations. BEST1, bestrophin 1 gene; pA/pF, picoamperes per picofarad; YFP, yellow fluorescent protein; NaI, sodium iodide. Sinha D, et al. Am J Hum Genet. 2020;107(2):278-292. 65 Paradigm for Guiding Treatment Decisions In vitro engineering of
Slide 66: OPGx-BEST1 Designed to Target RPE and Restore Bestrophin Function
Designed to restore retinal ion homeostasis in bestrophinopathies, ameliorating retinal structural and functional deficits BEST1 is targeted using the AAV2 capsid employed in LUXTURNA® and an RPE-specific promoter Most bestrophinopathies exhibit a slow rate of decline and central photoreceptors usua
Slide 67: OPGx-BEST1 Clinical Update
OPGx-BEST1 Clinical Update George Magrath, MD Chief Executive Officer Opus Genetics
Slide 68: Baseline Participant Demographics
*Worse eye deemed study (treated) eye.ARB, autosomal recessive bestrinopathy; BVMD, best vitelliform macular dystrophy; OD, right eye; OS, left eye; VA, visual acuity. Baseline Participant Demographics Participant # 101-101 101-104 102-101 102-102 101-106 Age 63 59 50 45 31 Sex Female Female Male Ma
Slide 69: Baseline OCT Example: 101-106 (BVMD)
69 Baseline OCT Example: 101-106 (BVMD) Study Eye(Treated) Fellow Eye (Untreated) BVMD, best vitelliform macular dystrophy; OCT, optical coherence tomography.
Slide 70: Baseline Microperimetry Example: 101-106 (BVMD)
70 Baseline Microperimetry Example: 101-106 (BVMD) Fellow Eye (Untreated) Study Eye (Treated) Sensitivity Map Fixation Plot Average Threshold BVMD, best vitelliform macular dystrophy.
Slide 71: IRD Patient Journey & Disease Prevalence
IRD Patient Journey & Disease Prevalence Joe Schachle, MBA Chief Operating Officer Opus Genetics
Slide 72: Patient Perspective: IRD Patient Flow Through Diagnosis & Genetic Testing
72 Patient Perspective: IRD Patient Flow Through Diagnosis & Genetic Testing IRD, inherited retinal disease. Sources: Lam, B. L., et al., Genetic Testing and Diagnosis of Inherited Retinal Diseases, Orphanet Journal of Rare Diseases. December 2021; American Academy of Ophthalmology, Guidelines on Cl
Slide 73: Evolution of IRD Prevalence Estimates
Stone, et al1 1,000 IRD families from 40 states in U.S. Covers large portion of IRDs (104) Hanany, et al2 Global study of 277K sequence variants across 6 world populations ONLY recessive for 187 IRDs Triangle Insights Analysis3 Conducted in 2023 73 Evolution of IRD Prevalence Estimates Triangle Insi
Slide 74: IRD Patient Prevalence Across Select Global Markets
IRD Patient Prevalence Across Select Global Markets Provides Significant Opportunity China 39,600 Middle East/North Africa 45,200 EU4 + UK 12,880 United States 25,770 EU4 = France, Spain, Germany, & Italy. BEST1, bestrophin 1; CNGB1, cyclic nucleotide-gated channel β1; IRD, inherited retinal disease
Slide 75: BEST1 Disease is Likely Underreported
IRD Prevalence May Be Higher Than Current Estimates BEST1 Prevalence Analysis based on published studies with diagnostic testing to confirm diagnosis* Estimated 8,400 Patients in the U.S. with BEST1 Patients may not undergo genetic testing when clinicians diagnose BEST1 disease based on the presence
Slide 76: Patient Recruitment & Retention Panel
Patient Recruitment &Retention Panel Moderator: Ben Yerxa Panelists: Jean Bennett, MD, PhD Todd Durham, PhD Bart Leroy, MD, PhD
Slide 77: Q&A
OPUS GENET |: S° Q&A
Slide 78: Summary Takeaways
Summary Takeaways George Magrath, MD, MBA, MS Chief Executive Officer
Slide 79: Fully-Funded to Support Multiple Clinical Inflection Points
79 Fully-Funded to Support Multiple Clinical Inflection Points 2H 2027 RHO clinical study initiation Q1 2027 MERTK clinical study initiation Q4 2026RDH12 clinical study initiation Q4 2026LCA5 Phase 3 dosing initiation Oct 2026PDUFA date for Phentolamine sNDA Sept 2026 BEST1 Cohort 1 3-month results
Slide 80: OPUS
OPUS iG E NWS T Alec