Form 8-K

8-K — Opus Genetics, Inc.

Accession: 0001140361-26-035979

Filed: 2026-09-09

Period: 2026-09-09

CIK: 0001228627

SIC: 2834 (PHARMACEUTICAL PREPARATIONS)

Item: Regulation FD Disclosure

Item: Other Events

Item: Financial Statements and Exhibits

Documents

8-K — ef20081753_8k.htm (Primary)

EX-99.1 — EXHIBIT 99.1 (ef20081753_ex99-1.htm)

EX-99.2 — EXHIBIT 99.2 (ef20081753_ex99-2.htm)

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8-K

8-K (Primary)

Filename: ef20081753_8k.htm · Sequence: 1

false000122862700012286272026-09-092026-09-09

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): September 9, 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)

Name of each exchange on which registered

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 September 9, 2026, Opus Genetics, Inc. (the “Company”) issued a

press release and held an investor conference announcing clinical data from Cohort 1 of the Company’s ongoing Phase 1/2 clinical trial of OPGx-BEST1 in patients with BEST1-related retinal diseases, including

best vitelliform macular dystrophy (“BVMD”) and autosomal recessive bestrophinopathy (“ARB”). A copy of the press release is furnished as Exhibit 99.1 to this Current Report on

Form 8-K. A copy of the data presentation used in connection with the investor conference is furnished as Exhibit 99.2 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 8.01

Other Events.

On September 9, 2026, the Company shared 3- and 6-month results from the

low-dose Cohort 1 of BIRD-1, the Company’s ongoing Phase 1/2 clinical trial of OPGx-BEST1 in patients with BEST1-related retinal diseases, including BVMD and ARB. Cohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three

participants with BVMD who have reached three months of follow-up and two participants with ARB who have reached six months of follow-up. Following treatment with OPGx-BEST1, all five participants demonstrated clinically meaningful improvement in

visual function, measured as one or more of the following: best-corrected visual acuity (“BCVA”), low-luminance visual acuity (“LLVA”), contrast sensitivity (“CS”) or microperimetry, an advanced eye test that maps how well the central part of the

retina sees light. Structural improvements were also observed across four participants.

OPGx-BEST1 demonstrated a favorable safety and tolerability profile, with no serious adverse events or dose-limiting toxicities, no

intraocular inflammation and no vital-sign or safety-laboratory findings of note. All treatment-related adverse events were mild or moderate in severity. BCVA improved in 60% of participants (3/5), LLVA improved in 40% of participants (2/5), and

contrast sensitivity improved in 40% of participants (2/5). Among evaluable participants, 75% (3/4) demonstrated clinically meaningful improvement in retinal sensitivity by microperimetry. Importantly, these gains were concentrated in the treated

retinal pigment epithelial Transitional Zone, where viable photoreceptors remain, with the greatest functional improvements observed in participants with less advanced disease. Structural improvements were observed in four of five participants,

with reductions in vitelliform material, the hallmark of BVMD, in 67% of participants with BVMD (2/3) and reductions in intraretinal fluid in 100% of participants with ARB (2/2). The third BVMD participant had possible, but not definitive,

reduction in vitelliform material.

Based on the safety profile and positive proof-of-concept findings from Cohort 1, the Company has advanced to the higher-dose Cohort 2, evaluating

OPGx-BEST1 at 4.5 x 10⁹ vg/eye, with dosing expected to be complete in the fourth quarter of 2026 and topline three-month data expected to be available in the second quarter of 2027. Originally designed to enroll five participants, Cohort 2 has

been over-enrolled with eight participants, most of whom have BVMD. Data from Cohort 2 are expected to further characterize the safety, functional and structural responses to OPGx-BEST1 at the higher dose and inform the design of a potential

pivotal clinical trial.

In addition, the Company announced that in August 2026, the Company met with the U.S. Food and Drug Administration (“FDA”) to discuss OPGx-BEST1

development and potential endpoints for a pivotal clinical trial. The Company aligned with the FDA on a potential pivotal endpoint based on ≥3 dB microperimetry improvement in ≥5 prespecified loci, in conjunction with a patient-reported outcome in

a randomized, controlled trial. BCVA, LLVA and CS may also be acceptable endpoints. The Company also aligned with the FDA on Phase 3 and commercial manufacturing requirements, which it expects to complete in early 2027. The Company currently

expects to begin planning for participant dosing in the Phase 3 clinical trial in 2027.

Finally, new epidemiology research estimates approximately 23,600 symptomatic BEST1 patients in the U.S., including 13,000 diagnosed

and 10,600 undiagnosed patients, and approximately 45,400 symptomatic BEST1 patients globally.

Forward-Looking Statements

This Current Report on Form 8-K contains forward-looking statements. All statements contained in this Current Report on Form 8-K that do not relate to

matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the Company’s continued clinical development, clinical results, preclinical data, and future plans for OPGx-BEST1,

including the anticipated timing of dosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase 1/2 clinical trial; the outcome of the Company’s ongoing regulatory interactions with the FDA and its expectations regarding the design of,

and potential endpoints for, of any pivotal clinical trial of OPGx-BEST1; the Company’s expectations regarding the clinical and therapeutic potential of OPGx-BEST1; the Company’s estimates of the BEST1 symptomatic patient population in the U.S. and

globally; and the Company’s expectations regarding its business prospects and results of operations. The clinical trial referenced in this Current Report on Form 8-K is ongoing, and the data described are interim, subject to change, and based on data

available as of a specified date. As patient enrollment continues and additional follow-up data is obtained, the reported safety profile and other clinical outcomes may change materially. There can be no assurance that the interim results will be

predictive of final clinical trial results or that additional data will confirm or support these observations. In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,”

“could,” “designed”, “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,”, “strive”, “target,” “will,” “would,” or the negative thereof and similar words and

expressions. Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to the Company. Such statements are neither promises nor guarantees, and involve a number of known

and unknown risks, uncertainties and assumptions that may cause the Company’s actual results, performance or achievements to be materially different from any expressed or implied by the forward-looking statements. Such risks and uncertainties

include, but are not limited to, the risk that the results of preclinical studies or clinical trials will not be predictive of future results in connection with future studies or clinical trials, uncertainty regarding the timing and results of

regulatory submissions, the risk that any Investigational New Drug Applications, New Drug Applications or other global regulatory submissions the Company may file with the FDA or other global regulatory agencies are not cleared on the Company’s

expected timelines, or at all, risks related to the Company’s ability to protect and maintain the Company’s intellectual property position, and risks related to manufacturing, supply, and distribution of the Company’s product candidates, along with

the risks detailed under the heading “Risk Factors” included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025 and in the Company’s other filings with the U.S. Securities and Exchange Commission. The

forward-looking statements in this Current Report on Form 8-K speak only as of the date of this Current Report on Form 8-K, and the Company undertakes no obligation to update or revise any of the statements. The Company’s business is subject to

substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.

Item 9.01

Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.

Description

99.1

Press release issued by Opus Genetics, Inc. on September 9, 2026, furnished herewith

99.2

Data presentation issued by Opus Genetics, Inc. on September 9, 2026, 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:

September 9, 2026

By:

/s/ Dr. George Magrath

Dr. George Magrath

Chief Executive Officer

EX-99.1 — EXHIBIT 99.1

EX-99.1

Filename: ef20081753_ex99-1.htm · Sequence: 2

Exhibit 99.1

Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting

with Potential Phase 3 Dosing in 2027

OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no serious adverse events or dose-limiting toxicities observed

All five participants demonstrated clinically meaningful improvement in visual function, with structural improvements observed in four

participants

FDA aligned on ≥3 decibels microperimetry improvement in conjunction with patient reported outcomes as a potential pivotal endpoint

Clinically meaningful best-corrected visual acuity improvements in 3 of 5 participants and retinal sensitivity improvements on

microperimetry observed in 3 of 4 evaluable participants

Cohort 2 over-enrolled, with dosing expected to be completed in Q4 2026 and topline 3-month data expected in Q2 2027

Cash runway into 2029 expected to support multiple clinical inflection points and opportunities for priority review vouchers

Webcast and conference call today at 8:00 a.m. ET with management and Key Opinion Leader and retinal specialist, Mark Pennesi, M.D., PhD.

RESEARCH TRIANGLE PARK, N.C. - Opus Genetics, Inc. (Nasdaq: IRD) (the “Company” or “Opus Genetics”), a clinical-stage biopharmaceutical company developing gene

therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced positive 3- and 6-month results from the low-dose Cohort 1 of BIRD-1, its ongoing Phase 1/2 clinical trial evaluating OPGx-BEST1 in

patients with BEST1-related retinal diseases, including Best vitelliform macular dystrophy (BVMD) and autosomal recessive bestrophinopathy (ARB).

Cohort 1 enrolled five participants treated at 1.5 x 10⁹ vg/eye: three participants with BVMD who have reached three months of follow-up and two with ARB who have

reached six months of follow-up. All five participants demonstrated clinically meaningful improvement in visual function, measured as one or more of the following: best-corrected visual acuity (BCVA), low-luminance visual acuity (LLVA), contrast

sensitivity (CS) or microperimetry, an advanced eye test that maps how well the central part of the retina sees light. Structural improvements were also observed across four participants. The greatest functional gains were observed in participants

with less advanced disease, supporting the potential benefit of treating patients while viable retinal tissue remains. The Company expects to announce 6-month data for the three participants with BVMD in Q2 2027.

“These positive results provide important evidence of OPGx-BEST1’s potential to improve both visual function and retinal structure in patients with BEST1-related

retinal disease, which we believe has a significantly larger underserved patient population than we previously thought,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “The functional and structural improvements across Cohort 1,

particularly the greater functional gains observed in patients with viable retinal tissue, reinforce our confidence in OPGx-BEST1’s potential to have a positive impact on the lives of patients with BEST disease. Together with our recent FDA

interaction and rapid enrollment of Cohort 2, we believe these data provide a clear path toward pivotal development, which we plan to begin next year. We want to recognize the contributions of our investigators, clinical teams, and most importantly,

the patients helping advance a potential treatment for this blinding disease.”

“These Cohort 1 data provide encouraging evidence that OPGx-BEST1 can be delivered safely and may improve both retinal structure and visual function in patients

with advanced BEST1-related retinal disease,” said Christine Nichols Kay, M.D., clinical trial investigator and Director of Clinical Research and Retinal Genetics at Vitreo Retinal Associates.

“We are encouraged by the localization of functional gains to areas of viable, but compromised retina and by the opportunity to apply these insights prospectively

as OPGx-BEST1 is advanced into Cohort 2 and potential pivotal development,” said Mark Pennesi, M.D., Ph.D., clinical trial investigator and Chief Medical Officer at the Retina Foundation and Adjunct Professor of Ophthalmology, Casey Eye Institute,

Oregon Health & Science University. “From a regulatory perspective, it is particularly exciting to align with the FDA on a >3 decibels change from baseline in microperimetry anchored to patient reported outcomes as a potential pivotal

endpoint.”

OPGx-BEST1 demonstrated a favorable safety and tolerability profile, with no serious adverse events or dose-limiting toxicities, no intraocular inflammation and

no vital-sign or safety-laboratory findings of note. All treatment-related adverse events were mild or moderate in severity.

All five participants demonstrated clinically meaningful improvement in visual function following treatment with OPGx-BEST1. BCVA improved in 60% of participants

(3/5), LLVA improved in 40% of participants (2/5), and contrast sensitivity improved in 40% of participants (2/5). Among evaluable participants, 75% (3/4) demonstrated clinically meaningful improvement in retinal sensitivity by microperimetry.

Importantly, these gains were concentrated in the treated retinal pigment epithelial (RPE) Transitional Zone, where viable photoreceptors remain, with the greatest functional improvements observed in participants with less advanced disease.

Structural improvements were observed in four of five participants, with reductions in vitelliform material, the hallmark of BVMD, in 67% of participants with

BVMD (2/3) and reductions in intraretinal fluid in 100% of participants with ARB (2/2). The third BVMD participant had possible, but not definitive, reduction in vitelliform material. These findings provide proof-of-concept that OPGx-BEST1 may

improve visual function and retinal structure in areas where viable retinal tissue remains and are informing patient selection and future clinical development.

In BVMD, vitelliform material accumulates early and is the defining structural feature of the disease, while subretinal fluid appears late and pools in areas of

established atrophy. Reduction of vitelliform material is therefore the more direct measure of restored RPE function in this population, and it is the structural change that corresponded with functional improvement in those participants. In ARB,

where intraretinal fluid is the dominant structural manifestation, fluid reduction was substantial and consistent in both participants.

In August 2026, the Company met with the U.S. Food and Drug Administration (FDA) to discuss OPGx-BEST1 development and potential endpoints for a pivotal clinical

trial. The Company aligned with the FDA on a potential pivotal endpoint based on ≥3 dB microperimetry improvement in ≥5 prespecified loci, in conjunction with a patient-reported outcome in a randomized, controlled trial. BCVA, LLVA, and CS may also

be acceptable endpoints. The Company also aligned with the FDA on Phase 3 and commercial manufacturing requirements, which it expects to complete in early 2027. The Company expects to begin planning for the Phase 3 trial immediately, with

participant dosing expected to begin in 2027.

Based on the safety profile and positive proof-of-concept findings from Cohort 1, the Company has advanced to the higher-dose Cohort 2, evaluating OPGx-BEST1 at

4.5 x 10⁹ vg/eye, with dosing expected to be completed in Q4 2026 and topline three-month data expected in Q2 2027. Originally designed to enroll five participants, Cohort 2 has been over-enrolled with eight participants, most of whom have BVMD. Data

from Cohort 2 are expected to further characterize the safety, functional and structural responses to OPGx-BEST1 at the higher dose and inform the design of a potential pivotal clinical trial.

New epidemiology research conducted by Triangle Insights Group, based on a survey of more than 150 eye care professionals, estimates approximately 23,600

symptomatic BEST1 patients in the U.S., including 13,000 diagnosed and 10,600 undiagnosed patients, and approximately 45,400 symptomatic BEST1 patients globally. These findings suggest a substantially larger addressable patient population and unmet

need than previously estimated.

Conference Call & Webcast Details

Opus Genetics will host a webcast and conference call with accompanying slides today at 8:00 A.M. ET, including comments by management and key opinion leader,

Mark Pennesi, M.D., PhD., FARVO, a board-certified retinal surgeon at the Retina Foundation of the Southwest. The live and archived webcast may be accessed on the Opus Genetics website under the Investors section: Events. Opus Genetics suggests

participants join 15 minutes in advance of the event.

About BEST1 and OPGx-BEST1

BEST1-related inherited retinal diseases, or bestrophinopathies, are rare forms of inherited macular degeneration caused by mutations in the BEST1 gene. These

mutations disrupt the normal function of RPE cells, leading to retinal lesions, progressive degeneration and vision loss. BEST1-related diseases include BVMD and ARB, and there are currently no approved therapies that address the underlying genetic

cause of these diseases.

OPGx-BEST1 is an investigational gene therapy designed to address the underlying genetic cause of BEST1-related inherited retinal diseases, including BVMD and

ARB. OPGx-BEST1 uses an AAV vector to deliver a functional copy of the BEST1 gene to retinal pigment epithelial cells. The ongoing BIRD-1 clinical trial is an adaptive, open-label Phase 1/2 clinical trial evaluating the safety and efficacy of

single-eye subretinal administration of OPGx-BEST1 in adults with BVMD or ARB.

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.

Forward-Looking Statements

This press release contains certain statements that 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, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. 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,

including the anticipated timing of dosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase 1/2 clinical trial; the Company’s patient-selection and development strategy for subsequent clinical trials of OPGx-BEST1; the outcome of the

Company’s ongoing regulatory interactions with the FDA and its expectations regarding the design of, and potential endpoints for, of any pivotal clinical trial of OPGx-BEST1; the Company’s expectations regarding the clinical and therapeutic potential

of OPGx-BEST1, including with respect to its ability to improve visual function and retinal structure in patients with BEST1-related retinal disease; the potential benefit of treating patients with viable retinal tissue; the Company’s estimates of

the BEST1 symptomatic patient population in the U.S. and globally; and the Company’s expectations regarding its business prospects and results of operations. The clinical trial referenced in this press release is ongoing, and the data described are

interim, subject to change, and based on data available as of a specified date. As patient enrollment continues and additional follow-up data is obtained, the reported data and other clinical outcomes may change materially. There can be no assurance

that the interim 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.

Contacts:

Investors

Jenny Kobin

Remy Bernarda

IR Advisory Solutions

ir@opusgtx.com

Media

Kimberly Ha

KKH Advisors

917-291-5744

kimberly.ha@kkhadvisors.com

EX-99.2 — EXHIBIT 99.2

EX-99.2

Filename: ef20081753_ex99-2.htm · Sequence: 3

Exhibit 99.2

OPGx-BEST1 Gene Therapy Phase 1/2 Study Low Dose Cohort 1 3-month

Results September 9, 2026

This presentation contains forward-looking statements within the meaning of the

Private Securities Litigation Reform Act of 1995. 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,” “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 our continued clinical development, clinical results, preclinical data, and future plans for OPGx-BEST1, including the anticipated timing of dosing completion and topline data from Cohort 2 of the OPGx-BEST1 Phase

1/2 clinical trial; the outcome of our ongoing regulatory interactions with the U.S. Food and Drug Administration (the “FDA”) and our expectations regarding the design of, and potential endpoints for, of any pivotal clinical trial of

OPGx-BEST1; our expectations regarding the clinical and therapeutic potential of OPGx-BEST1, including with respect to its ability to improve visual function and retinal structure in patients with BEST1-related retinal disease; our estimates

of the BEST1 symptomatic patient population in the U.S. and globally; and our expectations regarding our company, its business prospects, and our results of operations. These forward-looking statements are subject to certain risks and

uncertainties posed by many factors and events that could cause our 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: our clinical data related to gene therapies for the treatment of inherited retinal diseases is preliminary and related to a relatively small group of patients, and, as a result, data that

initially appears promising may be revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients; our gene therapy product candidates are based on a novel technology that

is difficult to develop and manufacture, 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 the 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; 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 services markets; 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; and those risks and uncertainties described

under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025 and our subsequent filings with the U.S. Securities and Exchange Commission (the “SEC”). Readers are cautioned not to

place undue reliance on these forward-looking statements, which speak only as of the date of this presentation. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might

subsequently arise. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Disclosures and Forward-Looking Statements 2

3 Management Team and KOL Participants Ash Jayagopal, PhD, MBA Chief Scientific

& Development Officer Sally Tucker, MCOptom, PhD Chief Medical Officer Rob Gagnon, CPA, MBA Chief Financial Officer Mark E. Pennesi, MD, PhD, FARVO Retina Foundation of the Southwest Dallas, TX George Magrath, MD Chief Executive

Officer Ben Yerxa, PhD President

Well-tolerated with no SAEs, no DLTs, and no intraocular inflammation Structural

and functional improvements at 3 months FDA meeting aligned on potential pivotal endpoint >3 dB microperimetry improvement in >5 prespecified loci In conjunction with a RCT using the PGI-S Phase 3 and commercial manufacturing on

schedule for delivery in early 2027 Cohort 2 overenrolled at 8 participants, weighted towards earlier stage BVMD 6-month Low-Dose Cohort 1 and 3-month High Dose Cohort 2 data expected in Q2 2027 Pivotal trial to commence dosing in

2027 New epidemiology report surveying 150 eye care professionals estimates 23,600 BEST1 patients in the U.S. 4 BEST1, bestrophin 1; DLT, dose-limiting toxicity; FDA, Food and Drug Administration; SAE, serious adverse event.; PGI-S, patient

global impression – severity; RCT, randomized controlled trial OPGx-BEST1 Program and Low-Dose Cohort 1 Clinical Data Highlights OPGx - B E S T 1 C o h o r t 1 & P r o g r a m S u m m a r y T O D AY ’ S T O P I C S 01 O p u s &

B E S T 1 O v e r v i e w 0 2 T r i a l D e s i g n & To p l i n e R e s u l t s 03 P a r t i c i p a n t C a s e S t u d i e s 04 P r o g r a m S u m m a r y & N e x t S t e p s 7 5 % o f e v a l u a b l e p a r t i c i p a

n t s m e t t h e F D A - a l i g n e d m i c r o p e r i m e t r y of > 3 d B i m p r o v e m e n t i n > 5 p r e s p e c i f i e d l o c i

Building a Differentiated Gene Therapy Platform 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 Administration Office of Orphan Products Development; FFB, Foundation

Fighting Blindness; GTx, gene therapy; 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. 5 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 Fund

& Abu Dhabi’s Healthcare Research and Innovation Fund OPGx-RHO adRP co-funded by FFB & NIH OPGx-NMNAT1 LCA OPGx-CNGB1 RP NIH-funded consortium Undisclosed IRD GTx Preclinical IND-enabling Phase 1/2 Phase 3 Approval All gene

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

6 BEST1: Group of Inherited Retinal Diseases 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*: ~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%) ARB 400 (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. A T R O P H Y F R A G M E N T E D V I T E L L I F O R M

MA T E R I A L S U B R E T I N A L F L U I D P S E U D O H Y P O P Y O N Photoreceptors Retinal Pigment Epithelium Bruch’s Membrane Choriocapillaris V I T E L L I F O R M L E S I O N V I T E L L I F O R M D E P O S I T S STAGE

1 Pre-vitelliform STAGE 2 Vitelliform STAGE 3 Pseudohypopyon STAGE 4 Vitelliruptive STAGE 5 Atrophy/Fibrosis S t a g e s o f B V M D 7 Cohort 1 Participants V i t e l l i f o r m m a t e r i a l o c c u r s e a r l y a n d i s t

h e h a l l m a r k o f B e s t V i t e l l i f o r m M a c u l a r D y s t r o p h y. S u b r e t i n a l f l u i d o c c u r s l a t e i n d i s e a s e a n d p o o l s i n a t r o p h i c a r e a s .

8 Trial Design & Topline Cohort 1 Results Sally Tucker, MCOptom, PhD Chief

Medical Officer

9 ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular

dystrophy. OPGx-BEST1: Phase 1/2 Study Overview (BIRD-1) D e s i g n Adaptive, open-label, dose-exploration, safety and tolerability study of subretinal injection of OPGx-BEST1 in adult participants with BVMD or ARB D o s i n g C o h o r

t s Cohort 1: 1.5x109 vg/eye Cohort 2: 4.5x109 vg/eye O b j e c t i v e s Primary: Safety and tolerability; identify appropriate dose for Phase 3 Secondary: Efficacy S t u d y P o p u l a t i o n Minimum of 5 participants at each dose

level S t a t u s o f C o h o r t 1 ( l o w d o s e ) 5 participants dosed: ARB (N=2): Data at 6 months BVMD (N=3): Data at 3 months S t a t u s o f C o h o r t 2 ( h i g h d o s e ) Enrollment complete with 8 participants (6

surgeries scheduled) Dosing expected to be completed in Q4 2026

*Worse eye deemed study eye. †ETDRS letters equivalent calculated from

logMAR ARB, autosomal recessive bestrophinopathy; BEST, bestrophin; BVMD, best vitelliform macular dystrophy; ETDRS, Early Treatment Diabetic Retinopathy Study; OCT, optical coherence tomography; OD, right eye; OS, left eye; 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 10

11 Study Endpoints Microperimetry Measures pointwise sensitivity of the retina

over the lesion Recent natural history data shows a steady decline over 5 years in BVMD, with most of the decline in the area on the edge of the lesion* BCVA & LLVA Methods to measure central fine visual function Contrast

Sensitivity Measures visual function and is sensitive in patients with central atrophy (used extensively in geographic atrophy studies) Autofluorescence A 2-dimensional picture of the retina highlighting vitelliform lesions OCT A

cross-sectional view of the retina * Bianco L, et al. Natural history of macular sensitivity in Best vitelliform macular dystrophy: microperimetry-derived outcome measures in preparation for clinical trials. Invest Ophthalmol Vis Sci.

2026;67(11):1.

Safety and Efficacy Summary: Highly Encouraging Proof-of-Concept Results from

Low-Dose Cohort 1 S a f e t y OPGx-BEST1 was well-tolerated in 5/5 participants, with no SAEs or DLTs S t r u c t u r a l E n d p o i n t s 80% of participants (4/5) had structural improvements: BVMD: 67% of participants (2/3) had a

decrease in vitelliform material ARB: 100% of participants (2/2) had a decrease in intraretinal fluid F u n c t i o n a l E n d p o i n t s 100% of participants (5/5) had improvements* in at least one functional measure: Microperimetry –

75% of participants (3/4) improved Best Corrected Visual Acuity – 60% of participants (3/5) improved Low Luminance Visual Acuity – 40% of participants (2/5) improved Contrast Sensitivity – 40% of participants (2/5) improved Earlier-stage

participant (101-106)† showed the biggest functional gains, suggesting a potential benefit from earlier treatment *Improvements defined as: >5 letters of improvement from baseline and >5 letter improvement from fellow eye in BCVA or

LLVA, 0.2 logMAR improvement in contrast sensitivity, and ≥5 loci improving by 3 or more decibels in RPE transitional zone on microperimetry. †101-106 was the youngest participant with most recent onset of disease and less progressive

disease than other participants. ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy; DLT, dose-limiting toxicity; logMAR, logarithm of the minimum angle of resolution; RPE, retinal pigment epithelium; SAE,

serious adverse event. 12

Well-tolerated in 100% of participants No intraocular inflammation No serious

adverse events No dose-limiting toxicities No treatment-related systemic AEs All ocular treatment-related AEs were mild/moderate in severity No vital sign issues or safety lab findings of note 13 OPGx-BEST1 Demonstrated a Favorable

Safety Profile Independent Data Monitoring Committee Recommended the Phase 1/2 Trial Advance to Cohort 2 at Higher Dose No intraocular inflammation was observed in any patient at any study visit *Based on the SUN Working Group Grading

Scheme. AE, adverse event. 101-101 101-104 (ARB) (ARB) 102-101 (BVMD) 101-106 (BVMD) 0 102-102 (BVMD) 1+ 2+ (cells in field)* 3+ 4+

Participant-Level Improvement by

Endpoint* Endpoint 101-101 ARB 101-104 ARB 102-101 BVMD 102-102 BVMD 101-106 BVMD BCVA >5 letter improvement compared to baseline and fellow eye ✓ ✓ ✓ LLVA >5 letter improvement compared to baseline and fellow

eye ✓ ✓ Contrast Sensitivity >0.2 logMAR improvement from baseline ✓ ✓ Microperimetry >5 loci cluster improvement ≥3 dB NE ✓ ✓ ✓ OCT IRF / vitelliform material reduced ✓ ✓ ✓ ✓ Improvement NE: Not

evaluable *Improvement defined as: >5 letters of improvement from baseline in BCVA or LLVA and >5 letter improvement compared to fellow eye, >0.2 logMAR improvement from baseline in contrast sensitivity, and >5 loci cluster

improvement by >3 decibels in Transitional Zone on microperimetry. ARB, autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BVMD, best vitelliform macular dystrophy; dB, decibel; IRF, intraretinal fluid; LLVA, low

luminance visual acuity; logMAR, logarithm of the minimum angle of resolution; OCT, optical coherence tomography. 14

2 0 -2 -4 -6 14 12 10 8 6 4 Baseline M1 M3 Change from Baseline in

BCVA (Letters Equivalent) All Treated Eyes (N=5) All Fellow Eyes (N=5) Treated Eyes with Structural Improvement (N=4)* Corresponding Fellow Eyes (N=4)* Mean BCVA Improved from Baseline Through 3 Months in Cohort 1 *Excludes Participant

102-101 due to significant foveal atrophy (with a subfoveal, sub-RPE scar) at baseline, likely exclusionary from the pivotal trial. Participant 102-101 had a 0.2 logMAR decrease in vision from baseline, all other participants had an

improvement in vision; Participant 102-101 had a meaningful improvement in contrast sensitivity. Error bars represent the standard error of the mean. BCVA, best corrected visual acuity; M, month. 15 +9 Letters -3 Let ters

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 data**: untreated BVMD sensitivity only declines with highest rate detected in the transition zone – No untreated eye met the 3db

improvement threshold, consistent with fellow eyes in cohort 1 Microperimetry Showed BVMD Improvement in the Treated RPE Transitional Zone 16 *All eyes includes all evaluable patients (n=4); 101-101 could not complete microperimetry due to

low vision. ** Bianco L, et al. Natural history of macular sensitivity in Best vitelliform macular dystrophy: microperimetry-derived outcome measures in preparation for clinical trials. Invest Ophthalmol Vis Sci. 2026;67(11):1. †Improvement

is defined as ≥5 loci area within the treated transitional zone improving by ≥3 dB from baseline. dB, decibel; FDA, Food and Drug Administration; RPE, retinal pigment epithelium. 3/4 evaluable participants* demonstrated † retinal

sensitivity improvement in the Transitional Zone 75% Treated Area Treated RPE Transitional Zone showing all loci that improved by ≥3 dB (circled) correlate with reduced vitelliform material Central atrophy Autofluorescence

Microperimetry FDA aligned on ≥3 dB change from baseline in ≥5 loci in treated RPE Transitional Zone anchored to a patient reported outcome Reduction in vitelliform material

BVMD: 67% of participants (2/3) had reduction in vitelliform material on

multimodal imaging All three BVMD participants had improvements in visual function, with highest gains in areas of vitelliform material reduction ARB: 100% of participants (2/2) had reduction in intraretinal fluid on OCT Both ARB

participants had improvements in visual function in areas where intraretinal fluid decreased Functional gains were co-localized to structural improvements Retinal sensitivity improved in the treated transitional zone at the edge of the

lesion where fluid was minimal Fixation moved from outside the lesion to within the lesion in all four evaluable participants Areas of the transitional zone treated within the subretinal bleb had the highest functional gains Structural

Improvements Seen in BVMD and ARB Participants Results I nform Future E nrollment Earlier-stage participants showed the greatest structural and functional improvements, suggesting earlier intervention may yield improved outcomes ARB,

autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BVMD, best vitelliform macular dystrophy; CS, contrast sensitivity; LLVA, low luminance visual acuity; logMAR, logarithm of the minimum angle of resolution; OCT,

optical coherence tomography. A T R O P H Y F R A G M E N T E D V I T E L L I F O R M M A T E R I A L S U B R E T I N A L F L U I D P S E U D O H Y P O P Y O N Photoreceptors Retinal Pigment Epithelium Bruch’s

Membrane Choriocapillaris V I T E L L I F O R M L E S I O N V I T E L L I F O R M D E P O S I T S STAGE 1 Pre-vitelliform STAGE 2 Vitelliform STAGE 3 Pseudohypopyon STAGE 4 Vitelliruptive STAGE 5 Atrophy/Fibrosis S t a g e s o f

B V M D 17

Post-Treatment Participant Feedback 101-101 ARB Able to see on the eye chart

for the first time in 30 years Wants the second eye treated 102-102 BVMD Wants the second eye treated Reports less eye strain after prolonged computer work; colors on TV appear brighter and clearer Happy with the study, mostly stable

vision 101-104 ARB 102-101 BVMD 101-106 BVMD 18 ARB, autosomal recessive bestrophinopathy; BVMD, best vitelliform macular dystrophy.

Participant Case Studies & Next Steps Ash Jayagopal, PhD Chief Scientific

and Development Officer 19

Baseline Month 3 Participant 101-106 (BVMD): Microperimetry

Improvements 20 BVMD, best vitelliform macular dystrophy; dB, decibel; RPE, retinal pigment epithelium, TTZ; Treated transitional zone. Treated transitional zone follows the vitelliform arc as seen on fundus autofluorescence and

OCT Microperimetry loci were not pre-specified Threshold for success > 3dB Average improvement in Treated Transitional Zone (TTZ) = 3.125 dB Fellow eye TZ = 0.5 dB 7 loci had ≥3 dB improvement (circled) This participant met the

microperimetry threshold for success of > 3dB.

Baseline Month 3 Participant 101-106 (BVMD): Vitelliform Material Reduction

Co-localized with Microperimetry Improvements 21 BVMD, best vitelliform macular dystrophy; dB, decibel; RPE, retinal pigment epithelium. L e s i o n r e d u c t i o n c o n s i s t e n t w i t h i m p r o v e d f u n c t i o n o f R P E c

e l l s a n d t r e a t m e n t a c t i v i t y a s i n d i c a t e d i n m i c r o p e r i m e t r y i m p r o v e m e n t s Vitelliform lesions reduced B a s e l i n e M o n t h 3

Baseline Month 3 Participant 102-102 (BVMD): Microperimetry

Improvements 22 BVMD, best vitelliform macular dystrophy; dB, decibel; RPE, retinal pigment epithelium, TTZ: Treated transitional zone. Yellow box represents the treated transitional zone Microperimetry loci were not

prespecified Threshold for success > 3 dB Average improvement in TTZ = 3.5 dB Fellow eye TZ = 0.5 dB 6 loci had ≥ 3 dB improvement (circled) – clustered within the treated transitional zone This participant met the microperimetry

threshold for success of > 3dB.

Baseline Month 3 Participant 102-102 (BVMD): Microperimetry Improved in the

Treated RPE Transitional Zone Baseline Month 3 B a s e l i n e M o n t h 3 23 Vitelliform material decreased BVMD, best vitelliform macular dystrophy; dB, decibel; RPE, retinal pigment epithelium. Yellow box represents treated

transitional zone Fixation moves to a more natural location in the fovea

Participant 102-101 (BVMD): Photoreceptor and RPE Atrophy Limit

Improvement 24 Images are consecutive registered OCT rasters at baseline, 1 month, and 3 months. BVMD, best vitelliform macular dystrophy; OCT, optical coherence tomography; RPE, retinal pigment epithelium; CS, Contrast Sensitivity. CS

improvements defined as >2 dB change from baseline M o s t A d v a n c e d B V M D P a r t i c i p a n t S u b R P E A t r o p h y a n d S c a r r i n g , E x c l u s i o n a r y f r o m a p o t e n t i a l p i v o t a l t r i a l V i t

e l l i f o r m m a t e r i a l C h a n g e s O v e r T i m e C S i m p r o v e m e n t s a k i n t o o b s e r v a t i o n s i n G e o g r a p h i c A t r o p h y 3 Months Baseline 1 Month

P a r a f o v e a l A r e a I n f e r i o r A r e a Baseline Month 3 Month

6 Participant 101-101 (ARB): Functional and Structural Outcomes Through 6 Months Functional improvement maintained to 6 months on BCVA, CS, and LLVA Unable to perform MP due to poor baseline vision IRF decreased at 3 months in the

parafoveal area Fluctuations observed in both areas between 3 and 6 months 25 ARB, autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BL, baseline; IRF, intraretinal fluid; CFB, change from baseline. BL M1 M2 M3 M4

M5 M6 12 9 9 0 5 10 15 BCVA CFB (Letters equivalent) Study Eye - OS (Treated) Fellow Eye - OD (Untreated)

Participant 101-104 (ARB): Microperimetry

Improvement Baseline Month 6 26 Total macular IRF volume = 19.06 ul at baseline, 7.55 ul at 6 months. Microperimetry loci were not prespecified ARB, autosomal recessive bestrophinopathy; dB, decibel; IRF, intraretinal fluid; RPE, retinal

pigment epithelium. Threshold for success: > 3dB change Average improvement in area of IRF = 3.3 dB Fellow eye corresponding area = 2.3 dB 13 loci had ≥ 3 dB improvement (circled) – clustered within the treated transitional zone This

participant met the microperimetry threshold for success of > 3dB.

Participant 101-104 (ARB): IRF Improvement Co-localized with Microperimetry

Improvement Baseline Month 6 27 Total macular intraretinal fluid volume: Baseline = 19.06 µl, Month 6 = 7.55 µl ARB, autosomal recessive bestrophinopathy; dB, decibel; IRF, intraretinal fluid; RPE, retinal pigment epithelium. Yellow

circles denote pockets of intraretinal fluid Fixation moves to a more natural location in the fovea Two areas of baseline intraretinal fluid

4 milestones cleared on the path to pivotal OPGx-BEST1 Advancing Based on

Positive Cohort 1 Results 1 2 3 4 Well-tolerated with no SAEs, no DLTs, and no intraocular inflammation Efficacy observed on both functional and structural endpoints Positive FDA Type C meeting aligned on potential pivotal endpoint with

microperimetry CMC, chemistry, manufacturing, and controls; DLT, dose-limiting toxicity; FDA, Food and Drug Administration; IDMC, independent data monitoring committee; SAE, serious adverse event. 28 75% of Cohort 1 evaluable participants

met the microperimetry threshold for success of > 3dB

Program Summary and Next Steps George Magrath, MD Chief Executive Officer 29

Accomplished Significant OPGx-BEST1 Program Milestones P H 1 / 2 C O H O R T 2 S

TAT U S D o s e 4.5x109 vg/eye E n r o l l m e n t Already over-enrolled Originally planned for 5 participants Over-enrollment of 8 participants C l i n i c a l D e v e l o p m e n t T i m e l i n e * Dosing expected to be completed

Q4 2026 Topline 3-month data expected Q2 2027 Pivotal trial planning initiated, dosing expected in 2027 R E G U L AT O R Y S TAT U S A u g u s t 2 0 2 6 F D A T y p e C m e e t i n g Aligned on Phase 3 CMC Constructive dialogue on

potential endpoint options for Phase 3 P o t e n t i a l P i v o t a l E n d p o i n t s Use of ≥3 dB microperimetry improvement in ≥5 prespecified loci area and; Randomized controlled trial with a patient-reported outcome BCVA, LLVA,

contrast sensitivity may also be acceptable endpoints N e x t R e g u l a t o r y I n t e r a c t i o n Following early data from Cohort 2 *Clinical development timelines are based on current estimates and are subject to change; data

readouts are targeted for ~9-12 months after study initiation. ARB, autosomal recessive bestrophinopathy; BCVA, best corrected visual acuity; BVMD, best vitelliform macular dystrophy; CMC, chemistry, manufacturing, and controls; dB,

decibels; FDA, Food and Drug Administration; LLVA, low luminance visual acuity; RPE, retinal pigment epithelium. 30

Current Cash to Support Multiple Clinical Inflection Points 2027 BEST1 Pivotal

Study Start Q1 2027 MERTK clinical study initiation Q4 2026 RDH12 clinical study initiation Q4 2026 LCA5 Phase 3 dosing initiation Oct 2026 PDUFA date for Phentolamine sNDA Sept 2026 BEST1 Phase 1/2 Cohort 1

3-month results Current cash runway extends into 2029, funding five clinical programs through multiple critical inflection points 4 Clinical Data Readouts Expected in 2027: BEST1, LCA5, RDH12, MERTK 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. Q2 2027 BEST1 Cohort 2 results 31

N a s d a q : I R D

33 Mark E. Pennesi, MD, PhD, FARVO Leading ophthalmologist, researcher

specializing in inherited retinal diseases, and pioneer in gene therapy Current Appointments Chief Medical Officer, Steve and Debbie Gray Inherited Retinal Degeneration Endowed Chair; Director, Inherited Retinal Degeneration Center – Retina

Foundation, Dallas, Texas Adjunct Professor of Ophthalmology – Paul H. Casey Ophthalmic Genetics Division, Casey Eye Institute, Oregon Health & Science University, Portland, Oregon Education B.S. in Biomedical Engineering, University

of Pennsylvania (summa cum laude); Combined MD/PhD, Baylor College of Medicine; Ophthalmology residency, UCSF; Ophthalmic genetics fellowship, Casey Eye Institute/OHSU Research and Awards Research focuses on developing novel treatments for

inherited retinal diseases Author of 170+ peer-reviewed publications; principal or co-principal investigator on numerous first-in-human gene therapy trials Research to Prevent Blindness and the Foundation Fighting Blindness have recognized

Dr. Pennesi with career development awards; Additionally, he was the recipient of the 2011 ARVO/Alcon Early Clinician Scientist, the Alcon Young investigator Award in 2014, and the Casey Eye Institute Resident teach award

34 Q&A with Opus Management and Dr. Pennesi Ash Jayagopal, PhD, MBA Chief

Scientific & Development Officer Sally Tucker, MCOptom, PhD Chief Medical Officer Rob Gagnon, CPA, MBA Chief Financial Officer Mark E. Pennesi, MD, PhD, FARVO Retina Foundation of the Southwest Dallas, TX George Magrath, MD Chief

Executive Officer Ben Yerxa, PhD President

Appendix 35

36 Triangle Insights Group Research, Q3 2026. BEST1 Market Landscape Quantitative

Market Research Patient Group Definition Est. Value Genetic BEST1 genotype (with or without symptoms) ~46,000 – 87,000 patients Symptomatic Diagnosed + undiagnosed (with ocular symptoms) ~23,600 patients Diagnosed

(Post-onset) Diagnosed (with ocular symptoms) ~13,000 patients Diagnosed (Genetically Confirmed) Diagnosed with genetic confirmation ~8,400 patients Undiagnosed (Post-onset) Undiagnosed (with ocular symptoms) ~10,600 patients BEST1

Patients: Estimated Inputs Symptomatic 23,600 pts Undiagnosed: 10,600 pts Diagnosed (Post-Onset) 13,000 pts Diagnosed (Genetic) 8,400 pts BEST1 Mutation: Estimated Subpopulations Genetic 46,000 – 87,000 pts

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