Opus Genetics R&D Science Forum

Opus Genetics R&D Science Forum June 16, 2026 | 10:00 AM - 12:00 PM ET

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 current

expectations or forecasts of future events or our future financial or operating performance. Such statements include, but are not limited to, statements concerning our data from and future enrollment for our clinical trials, our pipeline of

additional indications and anticipated regulatory milestones. In some cases, you can identify forward-looking statements by the following words: "aim," "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may,"

"ongoing," "plan," "potential," "predict," "project," "should," "will," "would" or the negative of those terms, and similar expressions that convey uncertainty of future events or outcomes to identify these forward-looking statements. These

forward-looking statements reflect our management's beliefs and views with respect to future events, are based on estimates and assumptions as of the date of this presentation and are subject to risks and uncertainties, many of which are

beyond our control, that could cause our actual results to differ materially from those in these forward-looking statements, including, without limitation: our gene therapy product candidates are based on a novel technology and manufactured

by a third party, which may result in delays and difficulties in obtaining regulatory approval; our planned clinical trials may face substantial delays, result in failure, or provide inconclusive or adverse results that may not satisfy U.S.

Food and Drug Administration ("FDA") requirements to further develop our therapeutic products; delays or difficulties associated with patient enrollment in clinical trials may affect our ability to conduct and complete those clinical trials

and obtain necessary regulatory approvals; changes in regulatory requirements could result in increased costs or delays in development timelines; we depend heavily on the success of our product pipeline; if we fail to find strategic partners

or fail to adequately develop or commercialize our pipeline products, our business will be materially harmed; others may discover, develop, or commercialize products similar to those in our pipeline before or more successfully than we do or

develop generic variants of our products even while our product patents remain active, thereby reducing our market share and potential revenue from product sales; we do not currently have any sales or marketing infrastructure in place and we

have limited drug research and discovery capabilities; the future commercial success of our products could significantly depend upon several uncertain factors, including third-party reimbursement practices and the existence of competitors

with similar products; product liability lawsuits against us or our suppliers or manufacturers could cause us to incur substantial liabilities and could limit commercialization of any product candidate that we may develop; failure to comply

with health and safety laws and regulations could lead to material fines; we have not generated significant revenue from sales of any products and expect to incur losses for the foreseeable future; our future viability is difficult to assess

due to our short operating history and our future need for substantial additional capital, access to which could be limited by any adverse developments that affect the financial markets; raising additional capital may cause our stockholders

to be diluted, among other adverse effects; instability and operational disruptions at government agencies, such as the FDA, may adversely impact our development and commercialization plans by causing delays and requiring the use of

additional, unforeseen resources to obtain regulatory approval for trials or products in our pipeline; we operate in a highly regulated industry and face many challenges adapting to sudden changes in legislative reform or the regulatory

environment, including due to government shutdowns and disruptions at government agencies, which cause delays, requires the use of additional, unforeseen resources, affects our pipeline stability, and could impair our ability to compete in

international markets; we may not receive regulatory approval to market our developed product candidates within or outside of the U.S.; with respect to any of our product candidates that receive marketing approval, we may be subject to

substantial penalties if we fail to comply with applicable regulatory requirements; our potential relationships with healthcare providers and third-party payors will be subject to certain healthcare laws and regulations, which could expose us

to extensive potential liabilities; we rely on third parties for material aspects of our business, such as conducting our nonclinical and clinical trials and supplying and manufacturing bulk drug substances, which exposes us to certain risks;

we may be unsuccessful in entering into or maintaining licensing arrangements or establishing strategic alliances on favorable terms, which could harm our business; inadequate patent protection for our product candidates may result in our

competitors developing similar or identical products or technology, which would adversely affect our ability to successfully commercialize; we may be unable to obtain full protection for our intellectual property rights under U.S. or foreign

laws; we may become involved in lawsuits for a variety of reasons associated with our intellectual property rights, including alleged infringement suits initiated by third parties; we are dependent on our key personnel, and if we are not

successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy; as we grow, we may not be able to operate internationally or adequately develop and expand our sales,

marketing, distribution, and other corporate functions, which could disrupt our operations; the market price of our common stock is expected to be volatile and if we fail to comply with the continued listing standards of Nasdaq, our common

stock may be delisted; and factors out of our control related to our securities, such as securities litigation or actions of activist stockholders, could adversely affect our business and stock price and cause us to incur significant

expenses. We discuss many of these risks in greater detail under Part I, Item 1A "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2025 and in subsequent reports filed with or furnished to the Securities and

Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment. New risks emerge from time to time. It is not possible for our management to predict all risks, nor can we assess the impact of all factors on

our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements we may make. Given these uncertainties, you should not place undue

reliance on these forward-looking statements. Any forward-looking statement in this presentation speaks only as of the date hereof or as of the date specified herein. We undertake no obligation to publicly update any forward-looking

statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable laws or regulations. Disclosures and Forward-Looking Statements 2

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 Retina Consultants of Texas Professor Robert MacLaren University of Oxford Lejla Vajzovic, MD Duke University Todd Durham, PhD, MS Foundation Fighting Blindness Opus Genetics Leadership Key Opinion Leaders Bart Leroy, MD,

PhD Ghent University Hospital 3

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 (Moderator) 4

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 Yerxa, PhD (Moderator) Summary Takeaways George Magrath, MD 5

Company Introduction George Magrath, MD Chief Executive Officer Opus

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 programs with

compelling economics Flexibility & potentially streamlined paths to approval Non-dilutive & voucher funding plus partnered strategic financial asset Validated science & delivery approach 7 Targeted IRD AAV gene therapy

assets Mover advantage in multiple indications 1st Streamlined timelines & capital efficiency Rare disease regulatory advantages Revenue & partnership streams drive value 7 AAV, adeno-associated virus; BEST1, bestrophin 1;

CNGB1, cyclic nucleotide-gated channel 1; IP, intellectual property; IRD, inherited retinal disease; LCA5, Leber congenital amaurosis 5; MERTK, MER proto-oncogene tyrosine kinase; NMNAT1, nicotinamide mononucleotide adenylyltransferase;

RDH12, retinol dehydrogenase 12; RHO, rhodopsin. LCA5 BEST1 RDH12 MERTK RHO CNGB1 NMNAT1

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 ARRP MERTK CNGB1 LCA RDH12 ADRP, autosomal dominant retinitis pigmentosa; ARRP, autosomal recessive retinitis pigmentosa; BEST1, bestrophin 1; CNGB1, cyclic nucleotide-gated channel 1; IRD, inherited retinal disease; LCA,

Leber congenital amaurosis; MERTK, MER proto-oncogene tyrosine kinase; NMNAT1, nicotinamide mononucleotide adenylyltransferase; RHO, rhodopsin; RDH12, retinol dehydrogenase 12. 8

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 1; FDA OOPD, Food and Drug

Administration Office of Orphan Products Development; FFB, Foundation Fighting Blindness; LCA5, Leber congenital amaurosis 5; NIH, National Institutes of Health; RD, retinal degeneration; RDH12, retinol dehydrogenase 12; RHO, rhodopsin; RP,

retinitis pigmentosa; MERTK, MER proto-oncogene tyrosine kinase; NMNAT1, nicotinamide mononucleotide adenylyltransferase. OPGx-LCA5 LCA co-funded by FDA OOPD OPGx-BEST1 Bestrophinopathies OPGx-RHO adRP co-funded by FFB &

NIH OPGx-RDH12 LCA co-funded by Global RDH12 Alliance OPGx-MERTK RP co-funded by FFB RD Fund & Abu Dhabi's Healthcare Research and Innovation Fund OPGx-NMNAT1 LCA OPGx-CNGB1 RP NIH-funded consortium Undisclosed IRD GTx All gene

therapy programs have the potential to qualify for a Priority Review Voucher 9

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 Current

Cash Runway into 2029 Funds Five Clinical Programs Through to Potential Product Approvals and PRV Opportunities 4 Clinical Data Readouts Expected in 2027 Clinical development timelines are based on current estimates and are subject to

change; data readouts are targeted for ~9-12 months after study initiation. Phentolamine ophthalmic solution 0.75% is a commercial partnered program; it is FDA-approved for the treatment of pharmacologically-induced mydriasis; an sNDA has

been submitted for the treatment of presbyopia. BEST1, bestrophin 1; LCA5, Leber congenital amaurosis 5; MERTK, MER proto-oncogene tyrosine kinase; PDUFA, Prescription Drug User Fee Act; PRV, Priority Review Voucher; RDH12, retinol

dehydrogenase 12; RHO, rhodopsin; sNDA, supplemental New Drug Application.

Scientific OverviewJean Bennett, MD, PhDF.M. Kirby Emeritus Professor of

Ophthalmology Perelman School of Medicine, University of PennsylvaniaPhiladelphia, PA

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 affected cells

before they have degenerated Lack of function disease Small gene/promoter AAV transduces target cells efficiently Disease severe enough to detect improvement Relevant animal/cell model(s) Quick readout Relatively prevalent More

Challenging Spark Therapeutics 377 348 RPE65 CHM

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 window"

where there are still enough viable cells for AAV gene replacement to restore function Pick the right patients, and choose meaningful endpoints for our clinical trials Clinical evidence for curative potential in IRDs AAV, adeno-associated

virus; IRD, inherited retinal disease.

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, retinal pigment epithelium-specific 65 kDa protein; RDH12, retinol dehydrogenase 12.

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-associated virus; GMP, Good Manufacturing Practices; LCA5, Leber congenital amaurosis 5.

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,

genotype-phenotype correlations, outcome measures Half a dozen retinal gene therapy clinical trials will read out within the next year Efficacy/Durability Data - LUXTURNA (voretigene neparvovec-rzyl) Long-term durability data (>9 years

and counting) for Phase 3 Real world efficacy similar to that reported in clinical trials 16 Status of Retinal Gene Therapy LUXTURNA is a registered trademark of Spark Therapeutics, Inc.

> 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 relevant

outcome measures Potency assays Numerous Obstacles to Retinal Gene Therapy Have Been Overcome > 2026: More Potential Outcome Measures Multi-Luminance Mobility Test Navigation Orientation, Object

Detection Perimetry Anatomy Change in Disease Progression Reading the Eye Chart 17 AAV, adeno-associated virus; FDA, Food and Drug Administration; GMP, Good Manufacturing Practices; LCA5, Leber congenital amaurosis 5.

RDH12 Overview Kenneth Fan, MD, MBA Retina Consultants of Texas Houston, TX

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 cases1 Global

prevalence*: ~30,900 patients3 Middle East / North Africa prevalence: ~17,500 patients3 U.S. prevalence: ~2,500 patients3 Clinical features Dense intraretinal pigmentation and macular atrophy with yellowish discoloration and

pigmentation1,4 Gold foil-like reflectance and watercolor appearance1 Early peripheral RPE atrophy with pigmented deposits, including bone spicules1 Peripapillary sparing1 Symptoms1,4 Loss of peripheral and central

vision Photophobia Nystagmus Night blindness 19 RDH12: An Early-Onset Inherited Retinal Degeneration *Global prevalence estimate includes United States, EU4 (France, Spain, Germany, & Italy), UK, Middle East/North Africa, and

China. LCA, Leber congenital amaurosis; RDH12, retinol dehydrogenase 12; RPE, retinal pigment epithelium. 1. Varela MD, et al. Ophthalmic Genet. 2022;43:301-306. 2. Aleman TS, et al. Invest Ophthalmol Vis Sci. 2018;59:5225-5236. 3. Triangle

Insights Analysis 2026. Opus Genetics, Inc. 4. Kumaran N. Br J Ophthalmol. 2017;101:1147-1154. Images courtesy of Kenneth Fan, MD

RDH12 is an enzyme expressed specifically in photoreceptor inner