Opus Genetics在Retina in Rio 2026公布两款在研基因疗法阳性数据，但核心读数为9月已披露的旧数据，会议仅补充探索性分析

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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM
8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
October 14, 2026
Opus Genetics, Inc.
(Exact name of registrant as specified in its charter)
Delaware
001-34079
11-3516358
(State or other jurisdiction of incorporation)
(Commission File Number)
(IRS Employer Identification No.)
8 Davis Drive
Durham
,
NC
27713
(Address of principal executive offices)
(Zip Code)
(
984
)
884-6030
(Registrant’s telephone number, including area code)
N/A
(Former name or former address, if changed since last report)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the
following provisions:
☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR
240.14d-2(b))
☐
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR
240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title of each class
Trading Symbol(s)
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Common Stock, $0.0001 par value per share
IRD
The Nasdaq Stock Market LLC
Indicate by check mark whether the registrant is an emerging growth company as defined in
Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter). Emerging growth company
☐
If an emerging growth company, indicate by check mark if the registrant has elected not to
use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
☐
Item 7.01
Regulation FD Disclosure.
On October 15, 2026, Opus Genetics, Inc. (the “Company”) issued a press release regarding the Company’s October 14, 2026
presentations at the Retina in Rio 2026 conference held in Rio de Janeiro, Brazil.  The presentations covered clinical data from Cohort 1 of the Company’s ongoing Phase 1/2 clinical trial of OPGx-BEST1 (the “OPGx-BEST1 Presentation”) and clinical
data from the Company’s ongoing Phase 1/2/3 clinical trial of OPGx-LCA5 (the “OPGx-LCA5 Presentation”).
A copy of the press release is furnished as Exhibit 99.1 to
this Current Report on Form 8-K. A copy of the OPGx-BEST1 Presentation is furnished as Exhibit 99.2 to this Current Report on Form 8-K.
A
copy of the OPGx-LCA5 Presentation is furnished as Exhibit 99.3 to this Current Report on Form 8-K.
The information in this report is furnished pursuant to Item 7.01, including Exhibit 99.1 attached hereto, shall not be
deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the
Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01
Financial Statements and Exhibits.
(d)
Exhibits
Exhibit No.
Description
99.1
Press release issued by Opus Genetics, Inc. on October 15, 2026, furnished herewith
99.2
Retina in Rio 2026 – OPGx-BEST1 Presentation, furnished herewith
99.3
Retina in Rio 2026 – OPGx-LCA5 Presentation, furnished herewith
104
Cover page from this Current Report on Form 8-K, formatted in Inline XBRL
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned
hereunto duly authorized.
OPUS GENETICS, INC.
Date:
October 15, 2026
By:
/s/ Dr. George Magrath
Dr. George Magrath
Chief Executive Officer

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EX-99.1
2
ef20083857_ex99-1.htm
EXHIBIT 99.1
Exhibit 99.1
Opus Genetics Presents Positive Clinical Data from OPGx-BEST1 and OPGx-LCA5 Gene Therapy Programs at Retina in Rio 2026
OPGx-BEST1 showed improved retinal sensitivity in 75% of evaluable participants in the initial low-dose Cohort 1 of the Company’s Phase 1/2 clinical
trial, supporting further clinical development
New BEST1 data analyses link retinal structure to visual function, enabling focus on regions most likely to respond to treatment
OPGx-LCA5 demonstrated sustained improvements in visual acuity and light sensitivity through 24 months in adults, with encouraging results in
pediatric participants through six months in the Company’s Phase 1/2/3 clinical trial
Phase 3 clinical trial dosing for OPGx-LCA5 expected in the fourth quarter of 2026
RESEARCH TRIANGLE PARK, N.C. – October 15, 2026 -
Opus Genetics, Inc.
(Nasdaq: IRD) (“Opus Genetics” or the “Company”), a clinical-stage biopharmaceutical company
developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), presented clinical findings from its OPGx-BEST1 and OPGx-LCA5 gene therapy programs at Retina in Rio 2026 in Rio de Janeiro,
Brazil.
The Company presented findings from the low-dose Cohort 1 of its Phase 1/2 BIRD-1 trial of OPGx-BEST1, which showed improved retinal sensitivity in three of four
evaluable participants (75%). Additional analyses identified retinal structural and imaging characteristics associated with visual function and treatment response, providing insights that may help guide future clinical development. Additional
findings from the ongoing Phase 1/2 trial of OPGx-BEST1 demonstrated encouraging improvements in retinal sensitivity in participants with BEST1-associated retinal diseases, along with new insights into imaging markers that may help predict
treatment response.
The Company also presented findings from the Phase 1/2 clinical trial of OPGx-LCA5, which demonstrated sustained improvements in visual function among adults and children
treated with OPGx-LCA5 for Leber congenital amaurosis type 5 (LCA5), a rare inherited retinal disease that causes severe vision loss beginning in childhood. In the OPGx-LCA5 Phase 1/2 study, improvements in visual acuity and light sensitivity were
observed in three adult participants through 24 months and three pediatric participants through six months. Participants also reported improvements in everyday activities, including navigating independently, recognizing family members, and
identifying objects and seeing colors.
“The data presented at Retina in Rio highlight meaningful progress across our gene therapy pipeline, with sustained improvements in visual function observed through 24
months in adults treated with OPGx-LCA5 and encouraging early evidence of improved retinal sensitivity in participants receiving OPGx-BEST1,” said George Magrath, M.D., Chief Executive Officer, Opus Genetics. “Importantly, these findings strengthen
our understanding of how to measure treatment response in IRDs and support the continued advancement of both programs. Our OPGx-BEST1 Phase 1/2 trial has completed enrollment in the high-dose Cohort 2 with 11 participants, and with our OPGx-LCA5
Phase 3 trial progressing toward dosing initiation later this year, we remain focused on bringing potentially transformative gene therapies to patients living with rare inherited retinal diseases.”
OPGx-BEST1
Opus presented findings from the ongoing Phase 1/2 BIRD-1 trial of OPGx-BEST1, an investigational gene therapy designed to restore bestrophin-1 function in patients with
IRDs caused by mutations in the BEST1 gene, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).
The presentation included results from five participants treated in the initial low-dose cohort, with follow-up ranging from three to six months. Among four participants
who could complete retinal sensitivity testing, three (75%) demonstrated improvements in the treated transitional zone, an area surrounding damaged retinal tissue where light-sensitive cells remain and may be responsive to treatment. Improvements
were measured using microperimetry, a test that evaluates the retina's ability to detect light at specific locations. In responding participants, increases in retinal sensitivity were also associated with reductions in intraretinal fluid
characteristic of BEST1-associated disease.
Additional analyses examined the relationship between retinal structure and function, suggesting that measurements obtained through optical coherence tomography (OCT) and
other imaging techniques may help identify retinal regions most likely to respond to treatment. The presentation also highlighted natural history findings demonstrating that retinal sensitivity declines most noticeably in the transitional zone
surrounding damaged tissue, even when measurements across the entire retina appear relatively stable.
Together, these findings are informing future clinical development of OPGx-BEST1, including more targeted retinal sensitivity assessments and imaging approaches designed
to improve the measurement of treatment response.
OPGx-LCA5
Rodrigo Jorge, M.D., of Ribeirão Preto Medical School at the University of São Paulo, presented findings from the ongoing Phase 1/2/3 trial of OPGx-LCA5, an
investigational gene therapy designed to restore production of lebercilin, a protein essential for normal retinal function. The presentation included data from six participants treated at the low dose of 1E10 vector genomes per eye, including three
adults and three adolescents. Adults demonstrated sustained improvements in visual acuity through 24 months, while pediatric participants demonstrated
restoration of cone-mediated vision
with sensitivity improvements reaching normal ranges through six months. Both groups also demonstrated improved light sensitivity, as measured by full-field stimulus testing (FST), compared with
their untreated eyes.
Additional retinal imaging using microperimetry showed increased retinal sensitivity and movement of visual fixation toward the central retina, which is responsible for
sharp, detailed vision. Participants also reported meaningful improvements in everyday activities, including recognizing family members, navigating independently without a cane, identifying objects and participating in activities that had
previously been difficult because of severe vision loss.
Based on the encouraging Phase 1/2 findings, Opus advanced OPGx-LCA5 into Phase 3 development using the low dose without requiring further dose escalation. The Phase 3
clinical trial’s natural history and treatment cohorts are fully enrolled, with dosing initiation expected in the fourth quarter of 2026. The clinical trial’s primary efficacy endpoint will assess improvements in retinal sensitivity across 16
central retinal test locations.
OPGx-LCA5 is an investigational adeno-associated virus serotype 8 (AAV8)-based gene therapy designed to deliver a functional copy of the LCA5 gene to retinal
photoreceptors. Mutations in LCA5 disrupt production of lebercilin, a protein critical for normal retinal function, resulting in severe visual impairment beginning early in life.
The full presentations and a video recording of the BEST1 slides will be available on the Opus Genetics website in the
Publications and Presentations
section.
Forward-Looking Statements
This press release contains certain statements that are not statements of historical fact and are forward-looking statements. In some cases, you can identify
forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “strive,” “will,” “would” or the negative
of these terms or other comparable terminology, although not all forward-looking statements contain these words. Such statements include, but are not limited to, statements related to the Company’s continued clinical development, clinical results,
preclinical data, and future plans for OPGx-BEST1 and OPGx-LCA5, including the anticipated timing of dosing initiation for the Company’s Phase 3 clinical trial of OPGx-LCA5; the Company’s ability to use insights from Cohort 1 of the Phase 1/2
OPGx-BEST1 clinical trial to help guide future clinical development and predict treatment response; the Company’s expectations regarding the clinical and therapeutic potential of OPGx-BEST1 and OPGx-LCA5, including with respect to the ability of
OPGx-BEST1 to improve retinal sensitivity and the ability of OPGx-LCA5 to enable sustained improvements in visual function; the Company’s participant-selection, treatment response assessment and development strategy for subsequent clinical trials
of OPGx-BEST1 and OPGx-LCA5; the Company’s expectations regarding the design of, and potential endpoints for, current and future clinical trials of OPGx-BEST1 and OPGx-LCA5; and the Company’s expectations regarding its business prospects and
results of operations. The OPGx-BEST1 and OPGx-LCA5 trials referenced in this press release are ongoing, and the data described are interim, subject to change, and based on data available as of a specified date. As participant monitoring and
enrollment in these trials continue and additional follow-up data is obtained, the reported data and other clinical outcomes may change materially. There can be no assurance that the initial results will be predictive of final clinical trial
results or that additional data will confirm or support these observations. The forward-looking statements contained herein are subject to certain risks and uncertainties posed by many factors and events that could cause the Company’s actual
business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the
heading “Risk Factors” included in the Company’s most recent Annual Report on Form 10-K for the fiscal year ended December 31, 2025, its Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, and in the Company’s other filings with the
U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. These forward-looking statements are based upon the Company’s
current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various
risks and uncertainties. The Company undertakes no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.
About Opus Genetics
Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases
(IRDs). The Company is developing durable, one-time treatments designed to address the underlying genetic causes of severe retinal disorders. The Company’s pipeline includes seven AAV-based programs, led by OPGx-LCA5 for LCA5-related mutations and
OPGx-BEST1 for BEST1-related retinal degeneration, with additional candidates targeting RDH12, MERTK, RHO, CNGB1 and NMNAT1. The Company is based in Research Triangle Park, NC. For more information, visit
www.opusgtx.com
.
Contacts:
Investors
Jenny Kobin
Remy Bernarda
IR Advisory Solutions
ir@opusgtx.com
Media
Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com
Source: Opus Genetics, Inc.

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EX-99.2
3
ef20083857_ex99-2.htm
EXHIBIT 99.2
Exhibit 99.2
Prediction of Retinal Sensitivity Changes Following Treatment in BEST Disease  George Magrath,
MD  Retina in Rio 2026
2  BEST1: Group of Inherited Retinal Diseases with a Range of Onset and Slow Rate of Progression
and no Approved Treatment Options  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*: ~45,400 patients2  U.S. prevalence: 23,600 patients (~23,200 BVMD and ~400 ARB)2   Clinical Features1,3  Serous retinal detachment  BVMD (most common) is characterized by vitelliform (“egg-yolk”) lesion beneath
the macula1  Macular atrophy  CNV  Symptoms3,4  Loss of central vision  Metamorphopsia (distorted vision)  Scotoma (blind spot)  Photophobia4  *Global prevalence estimate includes United States, EU4 (France, Spain, Germany, & Italy),
UK, Middle East/North Africa, and China.  ARB, autosomal recessive bestrophinopathy; BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy; CNV, choroidal neovascularization; IRD, inherited retinal disease.  1. Amato A, et al. Saudi
J Ophthalmol. 2023;37(4):287-295. 2. BEST1 Market Landscape Quantitative Market Research, Triangle Insights Group, Q3 2026. 3. Johnson AA, et al. Prog Retin Eye Res. 2017;58:45-69. 4. Tripathy K, et al. StatPearls [Internet]. Treasure
Island (FL): StatPearls Publishing; 2024.  BVMD  ARB  Most Common BEST1 Phenotypes  Prevalence  BVMD 23,200 (98%)2  ARB 400 (2%)2
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 in BVMD disrupt cellular ion and fluid homeostasis resulting in electrophysiological abnormalities, RPE dysfunction, and retinal
degeneration via:  Defective clearance of toxic waste products (e.g., lipid deposits)  Impaired RPE-photoreceptor interactions (e.g., defective phagocytosis)  Build-up of vitelliform material (toxic waste products) and fluid under the
retina  BEST1 Disease Biology  BEST1, bestrophin 1; BVMD, best vitelliform macular dystrophy; RPE, retinal pigment epithelium.Guziewicz KE, et al. Prog Retin Eye Res. 2017;58:70-88.   ATROPHY  FRAGMENTED VITELLIFORM MATERIAL  SUBRETINAL
FLUID  PSEUDOHYPOPYON  Photoreceptors  Retinal Pigment Epithelium  Bruch’s Membrane  Choriocapillaris  VITELLIFORM LESION  VITELLIFORM DEPOSITS  STAGE 1  Pre-vitelliform  STAGE 2  Vitelliform  STAGE 3  Pseudohypopyon  STAGE
4  Vitelliruptive  STAGE 5  Atrophy/Fibrosis  Stages of BVMD  3  Cohort 1 Participants  Vitelliform material occurs early and is the hallmark of BVMD  Subretinal fluid occurs late in disease and pools in atrophic areas
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 usually remain viable for
decades, providing a wide therapeutic window  BIRD-1: Phase 1/2 Study of OPGx-BEST1 Subretinal Gene Therapy in adult (≥18 years old) participants with BVMD or ARB  Cohort 1: low dose - 1.5E9 vg/eye  Cohort 2: high dose - 4.5E9
vg/eye  OPGx-BEST1 Gene Therapy is Designed to Target RPE and Restore Bestrophin Function  OPGx-BEST1 Gene Therapy  Vector  AAV2  Delivery  Single subretinal injection  4  AAV2, adeno-associated virus serotype 2; RPE, retinal pigment
epithelium.
*ETDRS letters equivalent calculated from logMAR.†Worse eye deemed study eye. ARB, autosomal
recessive bestrophinopathy; BEST, bestrophin; BVMD, best vitelliform macular dystrophy; ETDRS, Early Treatment Diabetic Retinopathy Study; RPE, retinal pigment epithelium; VA, visual acuity.   Participant Demographics: Low-Dose Cohort
1  101-101  101-104  102-101  102-102  101-106  Age  63  59  50  45  31  Sex  Female  Female  Male  Male  Male​  BEST phenotype  ARB  ARB  BVMD  BVMD  BVMD  Baseline VA*(study eye†)  1  50  49  61  47  Baseline VA* (fellow
eye)  43  65  68  72  56  Follow-up duration  6 months  6 months  3 months  3 months  3 months  Severity of disease  End-stage with significant atrophy  End-stage with significant atrophy  End-stage with sub-foveal, sub-RPE scar  Advanced
stage vitello-eruptive with some scarring  Earlier stage without significant atrophy or scarring  5
Defining the Transitional Zone  Border area surrounding the atrophic lesion with RPE and
photoreceptors that are structurally intact but functionally compromised or at risk of imminent atrophic progression   Highest potential to be rescued with OPGx-BEST1  Treated RPE Transitional Zone is the area of rescuable photoreceptors
around the atrophic lesion where OPGx-BEST1 treatment is administered  New natural history data1: Untreated BVMD sensitivity only declines with highest rate detected in the transition zone – No untreated eye met the 3 dB improvement
threshold, consistent with fellow eyes in Cohort 1  Microperimetry Showed BVMD Improvement in the Treated RPE Transitional Zone  *All eyes includes all evaluable patients (n=4); 101-101 could not complete microperimetry due to low
vision.  †Improvement is defined as ≥5 loci area within the treated transitional zone improving by ≥3 dB from baseline.BVMD, best vitelliform macular dystrophy; dB, decibel; FDA, Food and Drug Administration; RPE, retinal pigment
epithelium.  1. Bianco L, et al. Invest Ophthalmol Vis Sci. 2026;67(11):1.   3/4 evaluable participants* demonstrated retinal sensitivity improvement† in the Transitional Zone  75%  FDA aligned on ≥3 dB change from baseline in ≥5 loci in
treated RPE Transitional Zone anchored to a patient reported outcome   Treated Area  Central atrophy  Treated RPE Transitional Zone showing all loci that improved by ≥3 dB (circled) correlate with reduced vitelliform
material  Microperimetry  Autofluorescence  Reduction in vitelliform material  6
Transition Zone as a Microperimetry Endpoint
BVMD lesions have a core that is already scotomatous, and normal retina farther out  Between
them sits the transitional border zone: Points that still test normal today, immediately next to degenerating retina  This zone is concentrated within the central 5° around the fovea  Study objective:   Natural history cohort study of 23
eyes from 13 patients with confirmed BVMD over 4.4 years  Tested sensitivity at 68 points (central 10°) at each visit, then tracked how much that sensitivity declined per year  8  Natural history of macular sensitivity in BVMD:
Microperimetry-derived outcome measures in preparation for clinical trials  Fovea  Deep scotoma (core)  Relative scotoma  Transition zone   (normal points within central 5°)  Normal retina (periphery)  BVMD, Best vitelliform macular
dystrophy.  Bianco L, et al. Invest Ophthalmol Vis Sci. 2026;67:1.
No meaningful changes in the whole macula, but transition zone had a significant decline in mean
sensitivity over time  Annual mean sensitivity rate of change (dB/year), by subset of test points - per-eye regression, N = 23 eyes  Mean sensitivity across all 68 points did not decline significantly over 4 years (BVMD looks “stable”)
*Statistically significant.  ns=not significant.  BVMD, Best vitelliform macular dystrophy; dB, decibel; MS, mean sensitivity.   Bianco et al., Invest Ophthalmol Vis Sci. 2026;67:1.  9  Restricting the grid to normal points at baseline
and closest to the lesion border (transition zone), turned a flat global average into a small, steady, statistically significant decline  -1.2  -0.8  -0.4  0  0.2  All 68 points (whole macula)  -0.164  p=0.21; ns  Normal points (whole
grid)  -0.355  p=0.006*  Normal points within central 5°  -0.556  p=0.028*  Rate of change (dB/year)  Greater decrease in MSover time
Why this matters for trial design  1  Whole-macula averages hide real progression  Global mean
sensitivity across all 68 points was statistically unchanged over 4 years; an averaging artifact, not evidence of a stable retina  Averages are misleading; mixes healthy-looking spots with already-damaged spots, which can’t decline much
further  2  Transition zone reveals a real and significant decrease in sensitivity over time  Restricting analysis to points normal at baseline, especially those within the central 5°, closest to the lesion border, uncovers a small but
statistically significant, steady annual sensitivity decline (−0.36 to −0.56 dB/year)  3  Transition zone as a candidate microperimetry endpoint  BVMD is characterized by a slow and progressive functional decline, and whole-macula averages
aren't sensitive enough to measure progression within a realistic trial timeframe   But if you specifically track the transition points near the edge of the lesion (between preserved and degenerated outer retina), you can detect a
measurable decline and determine whether a treatment is slowing disease progression  BVMD, Best vitelliform macular dystrophy; dB, decibel.  Bianco et al., Invest Ophthalmol Vis Sci. 2026;67:1.  10
Learnings from the Ongoing Phase 1/2 Study of OPGx-BEST1
12  Transition Zone Could be Optimized for Greater Specificity  Current Study  TTZ loci
selected  Averages of 3.125 and 3.5 dB  What We Learned  Averages could have been higher with more selective loci  Hyperautofluorescent spots alone would have averaged 4-5 dB   Future Study  Increase specificity for loci likely to improve
using:  Multimodal imaging (FAF, OCT)  Baseline demographics  Treatment areas  FAF, fundus autofluorescence; OCT, optical coherence tomography; TTZ, treated transition zone.
BVMD, best vitelliform macular dystrophy.  Baseline  Month 1  Month 3  Microperimetry Improved
within the Transitional Zone and Translated to Reduction in Vitelliform Lesions  13
Baseline  Month 1  Month 3  Microperimetry Improved in the Transitional Zone and Correlated with
Decreased Vitelliform Material  Baseline  Month 3  14
OCT-derived EZ-to-RPE Thickness as a Structural Endpoint
Fovea-highest sensitivity (sharpest vision)  Blind spot (optic disc) ~15° temporal to
fixation  Periphery (far edge of vision)  Sensitivity  Also known as the Island of Vision in a Sea of Blindness   Classic 3-D model of the visual field introduced by Harry Traquair in the 1920s  Sensitivity peaks at the fovea and fades
toward the periphery  Used to interpret kinetic and static/automated perimetry (each tested point is a height on the hill)  In retinal diseases, volume under the hill can serve as an endpoint that is less affected by floor effects than
simple mean sensitivity   The Hill of Vision: Volumetric Visual Fields Predict Treatment Effect  Sea of blindness
Learnings from the Ongoing Phase 1/2 Study of OPGx-BEST1
18  Analysis 1: Characterization of spatial relationship between EZ-to-RPE thickness and retinal
sensitivity   Comparison  Pearson  Spearman  Measured microperimetry  0.760  0.784  HOV sensitivity  0.749  0.788  Measured, within-eye (subject-eye centered)  0.760  0.784  HOV, within-eye (subject-eye centered)  0.761  0.772  EZ-to-RPE
thickness was strongly associated with both microperimetry and HOV sensitivities; Associations were unchanged after subject-eye centering, demonstrating strong within-eye spatial relationship  Non-linear models significantly outperformed
linear regression, with 68% (microperimetry) and 69% (HOV) variances explained  Data supports EZ-to-RPE thickness as a spatial structural correlate of retinal function   OCT-derived EZ-to-RPE thickness was compared, sector by sector,
against retinal sensitivity measured with MAIA microperimetry - testing whether retinal structure predicts retinal function in 3 participants (N=6 eyes; 161 sector comparisons)  EZ, ellipsoid zone; HOV, hill of vision; MAIA, Macular
Integrity Assessment; OCT, optical coherence tomography; RPE, retinal pigment epithelium.
19  Analysis 2: Evaluation of treatment on EZ-to-RPE thickness   Treated eyes demonstrated a
consistent improvement in EZ-to-RPE thickness, with no change in fellow eyes   By Day 90, the treated-minus-fellow difference was positive in all 3 participants despite heterogeneous early (Day 30) responses  Largest responder (101-106)
showed a confirmed structural gain  Mean EZ-to-RPE thickness increased by ~11.1 µm in treated eye vs 1.5 µm in fellow eye by Day 90  Treated-minus-fellow change in mean EZ-to-RPE thickness  Participant  Day 30 (OS − OD)  Day 90 (OS −
OD)  101-106  +8.69 µm  +9.59 µm  102-101  +2.94 µm  +1.21 µm  102-102  −7.62 µm  +1.40 µm  Cohort mean  +1.34 µm  +4.07 µm  Evaluate whether treatment is associated with changes in EZ-to-RPE thickness (using the fellow untreated eye as
control) in 3 participants (N=6 eyes)  EZ-to-RPE thickness may provide a more sensitive structural endpoint than microperimetric sensitivity;  Thickness improvement from baseline occurred between Month 1 and Month 3, suggesting a treatment
effect  EZ, ellipsoid zone; OD, right eye; OS, left eye; RPE, retinal pigment epithelium.
HOV revealed a modest, but more consistent positive structure-function relationship compared to
microperimetry  Longitudinal change was coupled with baseline intermediate EZ-to-RPE thickness  Strongest coupling was observed in EZ-to-RPE thickness of 5-20 µm  Little coupling seen in low (≤5 µm) or high (>20 µm) EZ-to-RPE
thickness  20  Analysis 3: Longitudinal structure-function association over time  Comparison  Pearson  Spearman  Measured microperimetry change  0.050  0.044  Registered HOV change  0.200  0.167  HOV change, within-eye  0.133  0.145  HOV
vs. matched-location sensitivity  0.918  0.916  Compared longitudinal change in EZ-to-RPE thickness with change in retinal sensitivity using microperimetry and HOV mapping in 3 participants (N=6 eyes; 106 sector comparisons)  EZ, ellipsoid
zone; HOV, hill of vision; RPE, retinal pigment epithelium.
OCT Biomarkers and Retinal Sensitivity in BVMD  BVMD, best vitelliform macular dystrophy; OCT,
optical coherence tomography.
22  Clinical Correlation Between OCT Biomarkers and Retinal Sensitivity in BVMD  Variable  Beta
coefficient (dB)  Hyperautofluorescent (NIR-FAF pattern)*  +7.4  Vitelliform lesion (vs atrophy)  +7.6  Mixed lesion (vs atrophy)  +6.2  Subretinal fluid lesion (vs atrophy)  +4.7  ONL thickness, per µm  +0.2  P<0.001 across groups  Mean
MAIA Retinal Sensitivity by OCT Lesion Type  Sensitivity declined as lesions progressed toward atrophy  *Univariable analysis only; All other variables were from the multivariable analysis.dB, decibel; NIR-FAF, near infrared fundus
autofluorescence; OCT, optical coherence tomography; ONL, outer nuclear layer.  Battaglia Parodi M, et al. Transl Vis Sci Technol. 2022;11:24  Cross-sectional study of 57 eyes of 30 patients with BVMD (84% / n=48 eyes were in a clinical
stage) to determine whether lesion composition and other OCT/OCTA biomarkers predict microperimetric sensitivity   Analysis of Clinical Imaging Factors on Retinal Sensitivity  Hyperautofluorescence, OCT lesion type, and ONL thickness best
correlated with mean retinal sensitivity   R2=0.641 | OCT lesion type and ONL thickness  All P<0.05
Hyperautofluorescence Correlates with Microperimetry Changes in Participant 102-102
Hyperautofluorescence in treated transitional zone  Loci improving  Hyperautofluorescence in the treated transitional zone was strongly associated with improvement in mean retinal sensitivity  23
24  Hyperautofluorescence Correlates with Microperim Changes in Participant 101-106  Loci
improving   Hyperautofluorescence in the treated transitional zone was strongly associated with improvement in mean retinal sensitivity  Hyperautofluorescence in treated transitional zone
PH 1/2 - COHORT 2 DESIGN  25  Application to the Opus Clinical Trials  Microperimetry loci will
be prespecified  Target: Treated transitional zone, enriched by:   Baseline EZ-RPE thickness  Hyperautofluorescence   Vitelliform material on OCT  Clinical rationale: Earlier stage patients have higher magnitude of improvement  Cohort 2
will further inform pivotal trial  Longitudinal data from Cohort 1 will also further inform pivotal trial  ULTIMATE GOAL: Create a linear regression model to describe loci likely to improve  PATH TO PIVOTAL TRIAL  EZ, ellipsoid zone; OCT,
optical coherence tomography; retinal pigment epithelium.

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EXHIBIT 99.3
Exhibit 99.3
Gene Therapy For Congenital Leber Amaurosis (LCA5)  Dr. Rodrigo Jorge  Ribeirão Preto Medical
School  University of São Paulo - Brazil
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 retinopathy  Macular
atrophy  Photoreceptor structure often preserved   Symptoms1,3  Vision loss (typically starting in infancy); often limited to hand motions or light perception  Nystagmus   Hyperopia  Severe and early photoreceptor loss results in severely
abnormal or non-detectable visual fields  2  *Global prevalence estimate includes United States, EU4 (France, Spain, Germany, & Italy), UK, Middle East/North Africa, and China.  LCA, Leber congenital amaurosis; VA, visual acuity. 1. Boldt
K, et al. J Clin Invest. 2011;121(6):2169-2180. 2. Triangle Insights Group Analysis, February 2026. 3. Uyhazi KE, et al. Invest Ophthalmol Vis Sci. 2020;61:30.  M/31yrs, 20/300 VA  F/21yrs, light perception  LCA5 patients exhibit preserved
photoreceptors in the central retina in adulthood despite disease severity and early onset
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 photoreceptors1   In LCA5
patients, photoreceptor function is severely impaired due to a lack of functioning lebercilin1  OPGx-LCA5 is designed to address mutations in the LCA5 gene, which encodes for the lebercilin protein  Clinically derisked AAV8 vector delivers a
functional LCA5 gene directly to photoreceptor cells, using same promoter technology as LUXTURNA® (voretigene neparvovec-rzyl)  Clinical development timelines are based on current estimates and are subject to change.  LUXTURNA® is a
registered trademark of Spark Therapeutics, Inc.  AAV8, adeno-associated virus serotype 8; LCA5, Leber congenital amaurosis 5.  1. Uyhazi KE, et al. Invest Ophthalmol Vis Sci. 2020;61:30. 2. Song JY, et al. Mol Ther. 2018;26:1581-1593.  3
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 amaurosis 5.  PH 1/2 –
PART A  X  X  PH 3 - PART B  Ongoing follow-up  No dose-escalation needed  Advanced to Ph 3 using low dose  PART C  Fully enrolled  Fully enrolled
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 amaurosis 5.  PH 1/2 –
PART A  X  X  PH 3 - PART B  Ongoing follow-up  No dose-escalation needed  Advanced to Ph 3 using low dose  PART C  Fully enrolled  Fully enrolled  Ph 1/2 Part A Results Presented Today
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, visual acuity.
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 luminance  D-FST, Diagnosys LLC,
Littleton, MA  7
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)
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 specific points.BL, baseline; M,
month.  9  Adult Participant 01-04  Pediatric Participant 01-06  BL  M12  M24  BL  M6
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 the first time and watch his
sister get married.  Able to pour a glass of wine and drink unassisted for the first time in his life.  Able to travel independently and, as a result, acquired a new job, which requires a greater level of independence than he had
previously.  01-04*: Since treatment, no longer requires a cane and can navigate urban environments independently.  Pediatric Participants  01-05: Able to walk and cook without assistance of others; treatment has helped in her writing
capabilities  01-06*: Noticeable difference in visual brightness between his treated and untreated eyes  Able to watch basketball and can see the players and follow the ball instead of just watching the score ticker and listening.  01-07:
Mostly nonvisual prior to treatment. Reported visiting a local zoo, where she was able to visualize an owl for the first time, and also reported having to learn colors and what a window was in a hotel  Participant-reported outcomes provided
in follow up visits in 2025.  *01-04 and 01-06 were able to complete microperimetry.
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?
Thank you  Gene Therapy For Congenital Leber Amaurosis (LCA5)  Dr. Jorge Rodrigo  Ribeirão Preto
Medical School  University of São Paulo - Brazil