Opus Genetics starts MERTK RP gene therapy trial
Opus Genetics (Nasdaq: IRD) launched a clinical trial for OPGx-MERTK, an AAV-based gene therapy for MERTK-related retinitis pigmentosa, with funding from Abu Dhabi’s Healthcare Research and Innovation Fund.
Cleveland Clinic Abu Dhabi will serve as the clinical site and clinical development activities are expected to commence in 2026. The program targets a disease affecting an estimated 60,000 patients worldwide and addresses an area with no approved treatments.
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Positive
- Clinical trial launch funded by Abu Dhabi Healthcare Research and Innovation Fund
- Cleveland Clinic Abu Dhabi designated as clinical site with advanced capabilities
- Clinical development activities expected to commence in 2026
Negative
- OPGx-MERTK remains investigational; safety and efficacy unproven in humans
- Initial clinical activities concentrated in Abu Dhabi, limiting geographic enrollment diversity
- No approved treatments exist, indicating high development and regulatory risk
Clinical start year
2026
Clinical development activities at Cleveland Clinic Abu Dhabi
Global MERTK-RP patients
60,000 patients
Estimated worldwide MERTK-related retinitis pigmentosa population
IRD prevalence
5%
Inherited retinal diseases affect estimated 5% of population in region
$2.83
Last Close
Volume
Volume 1,026,565 vs 20-day average 506,341 (relative volume 2.03) ahead of the clinical trial launch news.
high
Technical
Price 2.81 is above 200-day MA at 1.48 and near the 52-week high of 2.85 (vs low 0.65).
IRD gained 9.77% while peers were mixed: OVID up 3.57%, ATRA up 1.34%, IMUX up 0.19%, OKYO down 2.7%. Only ABOS appeared on momentum scans, up 4.33% without news, suggesting today’s move is stock-specific.
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Dec 09 |
BEST1 safety review |
Positive |
+4.4% |
IDMC gave positive recommendation to continue OPGx-BEST1 Phase 1/2 trial. |
| Nov 13 |
BEST1 first dosing |
Positive |
-3.6% |
First participant dosed in OPGx-BEST1 Phase 1/2 gene therapy trial. |
| Sep 30 |
LCA5 pediatric data |
Positive |
-7.8% |
Positive three-month pediatric data from OPGx-LCA5 Phase 1/2 trial. |
| Sep 04 |
LYNX-3 Phase 3 start |
Positive |
+0.0% |
Initiation of LYNX-3 Phase 3 trial for phentolamine ophthalmic solution. |
| Jun 26 |
VEGA-3 Phase 3 win |
Positive |
+5.1% |
VEGA-3 Phase 3 trial met primary endpoint in presbyopia treatment. |
Clinical trial news has produced mixed reactions, with both strong gains and notable selloffs around positive updates. Some favorable data and safety milestones were followed by negative or flat price moves, while other trial successes coincided with upside.
Over the past six months, Opus Genetics has repeatedly advanced its gene therapy pipeline through key clinical milestones. Positive pediatric data in LCA5, Phase 3 success in presbyopia, and dosing and monitoring milestones in Best disease all reinforced its focus on inherited retinal and ophthalmic conditions. However, price reactions have varied, with several positive trial updates followed by negative or flat moves. Today’s MERTK-related retinitis pigmentosa trial launch extends this clinical strategy into another rare inherited blindness indication.
Historical Comparison
+4.2
%
Average Historical Move
In the past year, Opus released multiple clinical trial updates with an average move of 4.19%. Today’s 9.77% reaction to the MERTK trial launch stands at the higher end of prior clinical-news responses.
Clinical updates show progression from early-phase dosing and safety in Best disease to positive pediatric LCA5 data and successful Phase 3 presbyopia trials, now expanding into a new MERTK-related retinitis pigmentosa program.
This announcement details the funded launch of a gene therapy clinical trial for MERTK-related retinitis pigmentosa, a rare inherited disease affecting about 60,000 patients worldwide. Clinical activities are planned at Cleveland Clinic Abu Dhabi beginning in 2026, extending Opus Genetics’ inherited retinal disease pipeline. Historical clinical updates have yielded varied stock reactions, so investors may focus on future safety and efficacy readouts, regional recruitment progress, and how this program complements existing LCA5, Best disease and presbyopia efforts.
retinitis pigmentosa
medical
“MERTK-related retinitis pigmentosa (RP), a rare inherited eye disease”
Retinitis pigmentosa is a group of inherited disorders that slowly damage the light-sensing cells in the retina, causing progressive loss of peripheral and night vision and often leading to legal blindness. It matters to investors because the condition defines a clear patient population and long-term need for treatments, making clinical trial design, potential market size, and regulatory approval pathways central considerations when valuing companies developing diagnostics or therapies.
gene therapy
medical
“developing gene therapies to restore vision and prevent blindness”
Gene therapy is a medical technique that involves altering or replacing faulty genes in a person’s cells to treat or prevent disease. It is considered a promising area of innovation because it has the potential to provide long-term or even permanent solutions to genetic conditions. For investors, advancements in gene therapy can signal opportunities in biotech companies and emerging treatments with significant growth potential.
adeno-associated virus (AAV)
medical
“an investigational adeno-associated virus (AAV)-based gene therapy”
Adeno-associated virus (AAV) is a small, naturally occurring virus repurposed as a delivery vehicle to carry corrective genes into human cells for gene therapy. Think of it as a microscopic delivery truck that transports instructions to fix broken cellular machinery. Investors care because AAVs drive the safety, effectiveness, manufacturing difficulty and regulatory review of gene therapies, and those factors strongly influence development costs, approval timelines and commercial value.
photoreceptor
medical
“impair the retina’s ability to recycle photoreceptor components”
A photoreceptor is a light-sensing cell in the eye’s retina that converts light into electrical signals the brain uses to form vision, much like a camera sensor turning light into an image file. For investors, photoreceptors matter because their damage or loss underlies common sight‑threatening conditions; treatments, diagnostics, or devices that protect, repair, or replace these cells can create significant commercial and regulatory opportunities.
precision medicine
medical
“milestone for rare disease research and precision medicine in Abu Dhabi”
Precision medicine uses a person’s unique genetic makeup, lifestyle and environment to choose treatments and preventive steps that are more likely to work for them than one-size-fits-all approaches. For investors, it matters because it can make therapies more effective and efficient—think tailoring a suit rather than buying off the rack—affecting drug development costs, market size, pricing power and the speed at which therapies win regulatory approval.
inherited retinal diseases (IRDs)
medical
“patients with inherited retinal diseases (IRDs)”
Inherited retinal diseases (IRDs) are a group of genetic eye disorders in which faulty genes cause the light-sensing cells at the back of the eye to stop working, often leading to progressive vision loss or blindness. Think of it as a family blueprint that contains a flaw passed down through generations, gradually degrading how the eye processes light. Investors care because IRDs create a defined patient population and clear clinical needs, which can drive demand for new therapies, influence the size of potential markets, affect the pace and cost of clinical testing and regulatory review, and shape long-term revenue and risk profiles for companies developing treatments.
AI-generated analysis. Not financial advice.
- The trial is funded through Abu Dhabi’s Healthcare Research and Innovation Fund
- Clinical development activities will commence at Cleveland Clinic Abu Dhabi in 2026
- MERTK-related retinitis pigmentosa affects an estimated 60,000 patients worldwide
RESEARCH TRIANGLE PARK, N.C., Jan. 27, 2026 (GLOBE NEWSWIRE) — Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage biopharmaceutical company developing gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases (IRDs), today announced the launch and funding of its clinical trial evaluating a gene therapy for MERTK-related retinitis pigmentosa (RP), a rare inherited eye disease that causes progressive vision loss and eventual blindness.
The study is being launched in Abu Dhabi, the capital of the United Arab Emirates in collaboration with the Department of Health – Abu Dhabi (DoH), Cleveland Clinic Abu Dhabi, the Innovative Research Oversight and Support (IROS) division of the M42 group, and the Authority of Social Contribution – Ma’an. Cleveland Clinic Abu Dhabi will serve as the clinical site, equipped with advanced diagnostic imaging, surgical expertise and specialized retinal disease clinics.
The clinical trial will evaluate the safety and efficacy of OPGx-MERTK, an investigational adeno-associated virus (AAV)-based gene therapy developed by Opus Genetics. The therapy is designed to deliver a functional copy of the MERTK gene to retinal cells. Mutations in MERTK impair the retina’s ability to recycle photoreceptor components, leading to progressive degeneration and vision loss. There are currently no approved treatments for MERTK-related retinitis pigmentosa.
The Opus MERTK program is expected to commence clinical development activities in 2026, marking a major milestone for rare disease research and precision medicine in Abu Dhabi and the wider region, where inherited retinal diseases affect an estimated
George Magrath, M.D., Chief Executive Officer of Opus Genetics said: “Launching our clinical trial for a MERTK gene therapy is a defining moment for patients and for the field of inherited retinal disease. This collaboration with the Department of Health – Abu Dhabi and our partners brings together scientific innovation, clinical excellence, and a shared commitment to addressing serious unmet needs. For patients living with MERTK-related retinitis pigmentosa, this trial represents the first real opportunity to potentially change the course of a disease that has historically led to inevitable vision loss.”
H.E. Dr. Noura Al Ghaithi, Undersecretary of the Department of Health – Abu Dhabi, said: “By hosting the first MERTK gene therapy clinical trial in the United Arab Emirates (UAE), Abu Dhabi is demonstrating its leadership in precision medicine and rare disease innovation while turning scientific possibility into tangible impact for patients. In collaboration with our international and local partners, we are proud to support the development of a novel therapy that could transform the lives of individuals living with inherited blindness. This milestone brings renewed hope to families and reinforces Abu Dhabi’s position as a trusted destination for advanced healthcare and life sciences research.”
Dr. Fahed Al Marzooqi, Chief Executive Officer of M42’s Integrated Health Solutions, added: “This trial represents a pivotal step in strengthening Abu Dhabi’s position as a global hub for biotech innovation. Through strategic partnerships and clinical excellence, we are translating scientific breakthroughs into real-world treatments for patients with urgent, unmet needs. This collaboration reflects our mission to accelerate access to transformative therapies and to shape the future of rare disease research from the heart of the region.”
Through this trial, Opus Genetics and its partners reaffirm their commitment to advancing precision medicine, accelerating rare disease research, and bringing transformative gene therapies to patients with urgent unmet needs.
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 RHO, CNGB1, RDH12, NMNAT1, and MERTK. Opus Genetics is also advancing Phentolamine Ophthalmic Solution
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements related to the clinical development, clinical results, preclinical data, and future plans for OPGx-MERTK, and expectations regarding us, our business prospects, and our results of operations and 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, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024, our subsequent Quarterly Report on Form 10-Q, and our 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 our 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. 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. We undertake 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
[email protected]
Media
Kimberly Ha
KKH Advisors
917-291-5744
[email protected]
Source: Opus Genetics, Inc.
FAQ
What is Opus Genetics announcing about OPGx-MERTK and when does the trial start?
Opus Genetics launched a clinical trial for OPGx-MERTK and clinical development activities are expected to commence in 2026.
Where will the Opus Genetics MERTK clinical trial be conducted (IRD)?
The trial will be launched in Abu Dhabi with Cleveland Clinic Abu Dhabi serving as the clinical site.
Who funded the Opus Genetics MERTK gene therapy trial (IRD)?
The trial is funded through Abu Dhabi’s Healthcare Research and Innovation Fund.
How many patients worldwide are affected by MERTK-related retinitis pigmentosa?
MERTK-related retinitis pigmentosa affects an estimated 60,000 patients worldwide.
Does OPGx-MERTK have regulatory approval for MERTK-related RP (IRD)?
No; OPGx-MERTK is investigational and the trial will evaluate its safety and efficacy.
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