What Opus Genetics does

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases (IRDs) and a small-molecule therapy for refractive disorders. Founded in September 2021 by the Retinal Degeneration Fund with $19 million in seed funding and headquartered in Durham, North Carolina, the company became a public entity in October 2024 through acquisition by Ocuphire Pharma and now trades on NASDAQ under ticker IRD. Its core technology is subretinal AAV gene delivery targeting the genetic causes of IRDs, with a seven-program pipeline: OPGx-LCA5 in Phase 3 (FDA designations including RMAT, Orphan Drug, Rare Pediatric Disease, and RDEP); OPGx-BEST1 in Phase 1/2; OPGx-MERTK in Phase 1/2 (launched in Abu Dhabi); and OPGx-RHO, OPGx-RDH12, OPGx-NMNAT1, and OPGx-CNGB1 in preclinical or IND-enabling stages. The OPGx-RHO program uses a differentiated silence-and-replace approach for autosomal dominant retinitis pigmentosa.

The company serves patients with ultra-rare and rare inherited retinal conditions, including approximately 170 U.S. patients with LCA5, ~8,400 with BEST1-related diseases, ~8,800 with RHO-adRP, ~2,500 with RDH12, ~2,600 with MERTK, ~1,200 with NMNAT1, and ~2,100 with CNGB1, totaling roughly 83,000 patients worldwide across all targeted indications. Its commercial-stage asset is phentolamine ophthalmic solution 0.75% (RYZUMVI), FDA-approved for drug-induced mydriasis with a pending sNDA for presbyopia (PDUFA date October 17, 2026), partnered exclusively with Viatris for U.S. commercialization.

Opus generates revenue primarily through licensing royalties and milestone payments on the Viatris-partnered phentolamine asset, with FY2025 consensus revenue of approximately $15.8M. The business model relies on a mix of equity capital raises (over $68M across 2025-2026 preferred and direct offerings), non-dilutive structured financing ($155M Oberland facility extending runway into 2029), and non-dilutive grants from the Foundation Fighting Blindness and Retinal Degeneration Fund to advance clinical development. The company has not yet commercialized a gene therapy product and remains pre-revenue on its core AAV pipeline.

Name

Opus Genetics

Legal name

Opus Genetics, Inc.

Website

https://opusgtx.com

Company type

Public

Founded year

2021

Operating status

Operating

Headcount range

11–50 employees

Short description

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases. Its seven-program pipeline targets ultra-rare genetic blindness conditions, with OPGx-LCA5 in Phase 3 and OPGx-BEST1 in Phase 1/2 development.

Ownership category

akta.pro rank

Product category

Ophthalmic Gene Therapy Pharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

AAV Vector Gene Therapy Developers (HLAAACAA)

akta.pro secondary industries

Ophthalmology Gene Therapies (HLAAACAG), Gene Therapy for Rare/Monogenic Diseases (HLAAACAE), Rare Ophthalmology Disorder Therapies (HLAIAIAK), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Gene therapy
• Ophthalmic biopharmaceuticals
• Inherited retinal diseases
• AAV-based therapeutics
• Rare disease treatment

Headquarters

HQ city

Raleigh

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Supply Chain

Revenue model

1. Gene Therapy Product Sales: Revenue from FDA-approved gene therapy products for inherited retinal diseases, currently with one FDA-approved product and multiple programs in late-stage clinical development.
2. Licensing Royalties - Viatris Partnership: Royalty revenue from global licensing agreement with Viatris Inc. granting exclusive U.S. commercial rights to phentolamine ophthalmic solution 0.75% for presbyopia and visual disturbances.
3. Strategic Financing: Non-dilutive funding arrangements including senior secured notes facility with Oberland Capital providing up to $155 million to support clinical development.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels6 records

Core offering

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases (IRDs). Its pipeline includes seven gene therapy programs targeting genetic mutations including LCA5, BEST1, RHO, RDH12, MERTK, NMNAT1, and CNGB1, delivered via subretinal injection to restore or preserve vision. The company also has a partnered small-molecule ophthalmic product, phentolamine ophthalmic solution 0.75%, licensed to Viatris for U.S. commercialization for presbyopia and visual disturbances.

Product overview

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing a diversified portfolio of AAV-based gene therapies for inherited retinal diseases (IRDs), alongside a small-molecule therapy for ophthalmic disorders. The company's gene therapy pipeline includes seven programs: two in clinical development (OPGx-LCA5 in Phase 3 for LCA5, OPGx-BEST1 in Phase 1/2 for Best disease), three in IND-enabling stages (OPGx-RHO for autosomal dominant retinitis pigmentosa, OPGx-RDH12 and OPGx-MERTK for respective retinal dystrophies), and two in preclinical development (OPGx-NMNAT1 and OPGx-CNGB1). The company also has a partnered small-molecule product, phentolamine ophthalmic solution 0.75%, licensed to Viatris for U.S. commercialization. Opus executes a strategy centered on regulatory rigor, capital efficiency, and disciplined clinical development for rare inherited retinal conditions affecting over 2 million people worldwide.

Differentiator

Problem solved

Functional benefit

Products and services

• OPGx-LCA5 One-time subretinal AAV gene therapy delivering functional LCA5 gene to photoreceptors for Leber Congenital Amaurosis Type 5, an ultra-rare inherited retinal disease affecting approximately 170 people in the U.S. The therapy restores lebercilin protein essential for photoreceptor outer segment development.
• OPGx-BEST1 AAV vector-based gene therapy delivering functional BEST1 gene to restore bestrophin-1 protein production in retinal pigment epithelial cells for Best vitelliform macular dystrophy and autosomal recessive bestrophinopathy, affecting approximately 8,400 people in the U.S.
• OPGx-RHO Gene therapy targeting autosomal dominant retinitis pigmentosa caused by RHO mutations using a knockdown-and-replacement approach to preserve rod photoreceptors, affecting approximately 8,800 people in the U.S.
• OPGx-RDH12 Gene therapy targeting retinal dystrophy caused by RDH12 gene mutations, a severe early-onset IRD affecting approximately 2,500 people in the U.S.
• OPGx-MERTK Gene therapy for retinal degeneration caused by MERTK gene mutations affecting approximately 2,600 people in the U.S., using modern AAV vector design with improved durability and efficacy targeting RPE cell phagocytosis of photoreceptor outer segments.
• OPGx-NMNAT1 Gene augmentation therapy designed to halt disease progression in pediatric patients with retinal degeneration caused by NMNAT1 gene mutations, affecting approximately 1,200 people in the U.S.; NMNAT1 is essential for regenerating NAD+.
• OPGx-CNGB1 AAV gene therapy for late-onset retinitis pigmentosa caused by CNGB1 gene mutations, affecting approximately 2,100 people in the U.S., being advanced in collaboration with NIH-funded academic consortium.
• Phentolamine Ophthalmic Solution 0.75% Ophthalmic small-molecule solution for treatment of presbyopia and low-light visual disturbances. FDA-approved as RYZUMVI for drug-induced mydriasis with sNDA under FDA review for presbyopia. Licensed to Viatris for exclusive U.S. commercial rights.

Quantifiable outcome

• Over 30-fold improvements in cone sensitivity in pediatric LCA5 patients at 6 months with robust restoration of cone-mediated function following single subretinal injection
• +3 more outcomes

Named customers4 records

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Twelve partnerships are on record, tiered major, core and minor.

• Department of Health - Abu DhabimajorStrategic or Co-development Partner · 27 January 2026Department of Health - Abu Dhabi announced launch and funding of UAE's first clinical trial for gene therapy targeting MERTK-related retinitis pigmentosa through Abu Dhabi's Healthcare Research and Innovation Fund, positioning Abu Dhabi as a hub for precision medicine and biotech innovation.
• Cleveland Clinic Abu DhabimajorImplementation/ SI/ Consulting Partner · 27 January 2026Clinical trial evaluating OPGx-MERTK gene therapy for MERTK-related retinitis pigmentosa conducted at Cleveland Clinic Abu Dhabi, representing first clinical opportunity for patients with this hereditary retinal disease.
• M42's IROS DivisionmajorImplementation/ SI/ Consulting Partner · 27 January 2026M42's IROS (International Research Office Services) division provides clinical operations oversight for the OPGx-MERTK gene therapy trial in Abu Dhabi.
• Viatris Inc.coreChannel Partner/ Reseller/ DistributorGlobal licensing agreement granting Viatris exclusive U.S. commercial rights to phentolamine ophthalmic solution 0.75% for presbyopia treatment. Viatris presented Phase 3 data at ASCRS 2026 and holds PDUFA goal date of October 17, 2026 for sNDA approval.
• University of PennsylvaniacoreStrategic or Co-development PartnerWorld-class science from gene therapy pioneers including Dr. Jean Bennett's lab at Penn, which co-founded Spark Therapeutics (acquired by Roche) and other gene therapy companies.
• Harvard Medical SchoolcoreStrategic or Co-development PartnerPartnered with gene therapy pioneers at Harvard Medical School contributing to Opus' IRD program development based on world-class science.
• University of FloridacoreStrategic or Co-development PartnerPartnered with gene therapy pioneers at University of Florida contributing to Opus' IRD program development.
• NIH-funded Academic ConsortiummajorStrategic or Co-development PartnerCollaboration with NIH-funded academic consortium to advance OPGx-CNGB1 into and through a Phase 1 clinical trial for late-onset retinitis pigmentosa caused by CNGB1 gene mutations.
• Foundation for the NIH's Bespoke Gene Therapy ConsortiummajorStrategic or Co-development PartnerPartnership with Foundation for the NIH's Bespoke Gene Therapy Consortium for OPGx-CNGB1 program to advance gene therapy for CNGB1-related retinitis pigmentosa.
• Hope in FocusminorOthersPatient advocacy group partner supporting patients and families affected by inherited retinal diseases.
• Eyes on the FutureminorOthersPatient advocacy group partner founded by Silvia Cerolini to fund and develop gene therapies for rare genetic disorders affecting vision, raising over $6 million across more than 20 countries.
• RDH12 Fund for SightminorOthersPatient advocacy group focused on supporting RDH12 patients and families affected by RDH12-associated retinal dystrophy.

Scale indicators16 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Spark Therapeutics (Roche): Developer of Luxturna, the first FDA-approved AAV gene therapy for an inherited retinal disease (RPE65 mutation). Most direct comparable: same modality (subretinal AAV), same indication class (IRDs), and same patient/physician ecosystem.
• Adverum Biotechnologies: Clinical-stage gene therapy company developing AAV-based therapies for ocular diseases including wet AMD and IRDs. Competes in the same AAV-ocular delivery space and targets overlapping ophthalmology centers and patient identification infrastructure.
• MeiraGTx: Clinical-stage gene therapy company with multiple IRD programs (including AAV-RPGR for X-linked retinitis pigmentosa) and integrated AAV manufacturing. Direct competitor for AAV ocular delivery and IRD patient populations.
• REGENXBIO: Clinical and commercial-stage AAV gene therapy company with ocular programs (ABBV-RGX-314 for wet AMD via subretinal/suprachoroidal delivery) and a proprietary AAV platform. Competes on AAV technology, manufacturing scale, and ophthalmic indication strategy.

Emerging players

• 4D Molecular Therapeutics: Clinical-stage gene therapy company with a directed-evolution AAV platform and ophthalmology programs including 4D-150 for wet AMD. Emerging peer with next-generation AAV capsids that may offer delivery advantages over Opus's subretinal approach.
• Ray Therapeutics: Preclinical/early-clinical gene therapy company developing optogenetic therapies for retinal degeneration. Comparable as an IRD-focused AAV gene therapy developer with similar patient-identification and ophthalmic-center commercialization model.
• Coave Therapeutics: Clinical-stage gene therapy company developing AAV-based therapies for ocular and CNS diseases, including IRDs. Comparable platform technology and target patient population, with focus on ultra-rare ophthalmic indications.
• SparingVision: Clinical-stage gene therapy company developing therapies for IRDs including retinitis pigmentosa, with both gene-augmentation and mutation-independent approaches. Directly comparable as an IRD-focused AAV gene therapy developer targeting similar patient populations.
• GenSight Biologics: Clinical-stage gene therapy company developing AAV-based therapies for IRDs, including Lumevoq for Leber Hereditary Optic Neuropathy. Comparable in modality, target indications, and co-founded by scientific advisors in Opus's network.

Others

• Viatris (Ophthalmology): Opus's U.S. commercial partner for phentolamine ophthalmic solution 0.75% and a broader ophthalmic and specialty pharmaceutical company. Relevant as Opus's primary commercialization vehicle for its non-gene-therapy assets and as a strategic counterparty.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/opusgenetics
• Facebookfacebook.com/OpusGenetics
• LinkedInlinkedin.com/company/opus-genetics
• Instagraminstagram.com/opusgenetics

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds3 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Opus Genetics