Opus Genetics(formerly Ocuphire Pharma)

What Opus Genetics(formerly Ocuphire Pharma) does

Opus Genetics, Inc. (NASDAQ: IRD; formerly Ocuphire Pharma) is a clinical-stage biopharmaceutical company headquartered in Research Triangle Park, North Carolina, that develops AAV-based gene therapies for inherited retinal diseases (IRDs) and small-molecule ophthalmic therapeutics for refractive disorders and diabetic retinopathy. The company's core technology platform delivers functional genes via adeno-associated virus vectors to retinal photoreceptors or retinal pigment epithelial cells, with a pipeline of seven programs targeting LCA5, BEST1, RDH12, MERTK, RHO, CNGB1, and NMNAT1 mutations. Its lead gene therapy asset OPGx-LCA5 is in Phase 1/2 development for Leber congenital amaurosis, and OPGx-BEST1 is advancing toward the clinic for bestrophin-1-related retinal degeneration.

The company operates a hybrid business model. Nyxol (phentolamine ophthalmic solution 0.75%, branded RYZUMVI for mydriasis reversal) was FDA-approved in September 2023 and is commercialized globally by Viatris under a licensing agreement with FamyGen Life Sciences; Opus receives milestone payments and tiered double-digit royalties on worldwide net sales through 2040. APX3330, an oral Ref-1 inhibitor for diabetic retinopathy, was developed internally. The gene therapy programs are funded through a combination of dilutive equity (e.g., the $25M February 2026 Series B preferred led by Adage Capital Management) and non-dilutive strategic capital (up to $155M from Oberland Capital in April 2026, extending runway into 2029).

Opus is backed by the Foundation Fighting Blindness's RD Fund and has access to its 40-site Clinical Research Consortium and the Uni-Rare natural history study covering ~1,500 patients across over 300 IRD-associated genes. The company was formed through the November 2020 reverse merger of Ocuphire Pharma with Rexahn Pharmaceuticals, rebranded as Opus Genetics in 2023 following its strategic pivot toward AAV gene therapy, and is led by CEO George Magrath (appointed November 2023).

Name

Opus Genetics(formerly Ocuphire Pharma)

Legal name

Opus Genetics, Inc.

Website

https://opusgtx.com

Company type

Public

Founded year

2020

Operating status

Operating

Headcount range

11–50 employees

Short description

Opus Genetics is a clinical-stage biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases and small-molecule ophthalmic drugs, with seven gene therapy programs led by OPGx-LCA5 and partnered commercialization of approved RYZUMVI via Viatris.

Ownership category

akta.pro rank

Product category

Ophthalmic Biopharmaceuticals

NAICS

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

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Ophthalmology Gene Therapies (HLAAACAG)

akta.pro secondary industries

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

Keywords

• Ophthalmic gene therapy
• Inherited retinal diseases
• AAV gene therapy
• Retinal biopharmaceuticals
• Ophthalmic pharmaceuticals

Headquarters

HQ city

Farmington Hills

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Licensing Royalties and Milestone Payments: Under the global license agreement with FamyGen/Viatris for Nyxol, Ocuphire receives upfront payments, development funding, regulatory milestone payments upon FDA approvals, tiered double-digit royalties on worldwide net sales through 2040, and sales milestone payments upon achievement of annual sales thresholds. Received $35 million upfront and eligible for $10 million upon RM approval.
2. Non-Dilutive Financing: Strategic financing agreement with Oberland Capital providing up to $155 million in non-dilutive funding including $35 million upfront payment and $5 million equity investment, with potential additional funding tied to development milestones. This extends cash runway into 2029.
3. Equity Financing: Private placement financing through sale of Series B Non-Voting Convertible Preferred Stock to institutional investors including Adage Capital Management, Trails Edge Capital Partners, and Marshall Wace, raising $25 million in gross proceeds.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company developing AAV-based gene therapies for inherited retinal diseases (IRDs), with seven pipeline programs targeting LCA5, BEST1, RDH12, MERTK, RHO, CNGB1, and NMNAT1 mutations. The company also advances Nyxol/RYZUMVI (phentolamine ophthalmic solution 0.75%) for mydriasis reversal, presbyopia, and night vision disturbances, plus APX3330, an oral Ref-1 inhibitor for diabetic retinopathy. Its lead programs OPGx-LCA5 and OPGx-BEST1 are advancing through clinical evaluation with FDA Rare Pediatric Disease Designation granted to OPGx-LCA5.

Product overview

Opus Genetics is a clinical-stage biopharmaceutical company developing gene therapies for inherited retinal diseases (IRDs). The company's pipeline includes seven AAV-based gene therapy programs led by OPGx-LCA5 and OPGx-BEST1, with additional candidates targeting RDH12, MERTK, RHO, CNGB1, and NMNAT1. The company also continues to advance Nyxol/Phentolamine Ophthalmic Solution (marketed as RYZUMVI for reversal of mydriasis) and APX3330 (oral Ref-1 inhibitor for diabetic retinopathy) through its partnership with Viatris and FamyGen Life Sciences. Opus Genetics was formerly known as Ocuphire Pharma and operates from Research Triangle Park, NC.

Differentiator

Problem solved

Functional benefit

Brands

• OPGx-LCA5: AAV-based gene therapy program for LCA5-related mutations (Leber congenital amaurosis)
• OPGx-BEST1
• OPGx-RDH12
• OPGx-MERTK
• OPGx-RHO
• Nyxol
• RYZUMVI

Products and services

• OPGx-LCA5 AAV8-based gene therapy candidate designed to deliver a functional LCA5 gene to retinal photoreceptors for treatment of Leber congenital amaurosis (LCA) caused by LCA5 gene mutations. One of the lead programs in Opus Genetics' seven AAV-based gene therapy pipeline, with FDA Rare Pediatric Disease Designation granted.
• OPGx-BEST1 Gene therapy candidate for BEST1-related inherited retinal degeneration, designed to deliver a functional BEST1 gene to retinal pigment epithelial cells to produce bestrophin-1 protein and normalize photoreceptor homeostasis. Acquired from Iveric Bio in December 2022.
• OPGx-RDH12 Preclinical gene therapy program targeting RDH12 mutations for inherited retinal diseases. Part of the company's seven AAV-based gene therapy pipeline, with clinical entry expected in 2026.
• OPGx-MERTK Preclinical gene therapy program targeting MERTK mutations for inherited retinal diseases. Part of the company's seven AAV-based gene therapy pipeline, with clinical entry expected in 2026.
• OPGx-RHO Gene therapy candidate acquired from Iveric Bio for rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), affecting approximately one in 51,000 people in the United States.
• OPGx-CNGB1 Preclinical AAV-based gene therapy program in the company's pipeline for inherited retinal diseases, targeting CNGB1 mutations.
• OPGx-NMNAT1 Preclinical AAV-based gene therapy program in the company's pipeline for inherited retinal diseases, targeting NMNAT1 mutations.
• RYZUMVI (Phentolamine Ophthalmic Solution 0.75%) FDA-approved eye drop (Phentolamine Ophthalmic Solution 0.75%) for reversal of pharmacologically-induced mydriasis. Commercialized by partner Viatris following September 2023 FDA approval. Demonstrated statistically significant superiority vs placebo in returning dilated eyes to baseline pupil diameter as early as 60 and 90 minutes.
• Nyxol (Phentolamine Ophthalmic Solution 0.75%) Preservative-free eye drop formulation of phentolamine mesylate 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size. Developed for reversal of pharmacologically-induced mydriasis (RM), presbyopia, and dim light/night vision disturbances (NVD). Licensed to Viatris for global commercialization.
• APX3330 Oral small-molecule inhibitor of Ref-1 (reduction-oxidation effector factor-1) for treatment of diabetic retinopathy and diabetic macular edema. First-in-class drug candidate with dual mechanism of action reducing both angiogenesis (VEGF) and inflammation (NF-kB) pathways. ZETA-1 Phase 2 trial demonstrated 0% binocular 3-step worsening vs 16% for placebo (p=0.04).

Quantifiable outcome

• OPGx-LCA5 received FDA Rare Pediatric Disease Designation, which may provide eligibility for a priority review voucher upon approval
• +3 more outcomes

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

Four partnerships are on record, tiered core.

• Iveric BiocoreStrategic or Co-development Partner · 28 December 2022Acquired rights to two preclinical-stage AAV-based gene therapy candidates for BEST1-related inherited retinal diseases and RHO-mediated autosomal dominant retinitis pigmentosa. Iveric received $500,000 upfront payment and high single-digit percentage ownership of Opus, with potential for development and regulatory milestone payments and low single-digit earnout on net sales.
• Foundation Fighting Blindness Clinical ConsortiumcoreStrategic or Co-development Partner · 14 November 2022Global, 40-site network of clinical research centers conducting the Uni-Rare natural history study for inherited retinal diseases. Jaeb Center for Health Research serves as coordinating center.
• Viatris Inc.coreChannel Partner/ Reseller/ Distributor · 7 November 2022Global license agreement for commercialization of Nyxol across three indications (reversal of mydriasis, presbyopia, night vision disturbances) in US, Europe, Japan, India, China and other global markets. Viatris received rights following separate transaction with FamyGen Life Sciences.
• FamyGen Life SciencescoreOEM/ Whitelabel/ Licensing Partner · 7 November 2022Exclusive license agreement for development and commercialization of Nyxol across three indications. Famy funded Nyxol development through FDA approvals and undertook development in non-US markets. Received $35 million upfront payment with potential for additional milestone and royalty payments.

Scale indicators8 records

Recent moves8 records

Expansion highlights6 records

Direct peers

• Spark Therapeutics: Pioneer in ocular gene therapy; developed and commercializes Luxturna, the first FDA-approved gene therapy for an inherited retinal disease (RPE65 mutations). Directly comparable as the established leader in AAV-based IRD therapeutics.
• MeiraGTx Holdings: Clinical-stage gene therapy company with multiple IRD programs and proprietary AAV manufacturing capabilities. Directly comparable in target indications (IRDs) and AAV-based delivery approach.
• Adverum Biotechnologies: Clinical-stage gene therapy company developing AAV-based therapies for ocular diseases including wet AMD and diabetic retinopathy. Comparable as a fellow ocular gene therapy developer targeting retinal indications.
• Regenxbio: AAV gene therapy platform company with ophthalmology programs (including ABBV-RGX-314 for wet AMD in partnership with AbbVie). Comparable through shared AAV vector technology targeting retinal diseases.
• GenSight Biologics: Clinical-stage gene therapy company developing AAV-based treatments for Leber Hereditary Optic Neuropathy (LHON) and IRDs. Comparable as a fellow ophthalmology-focused gene therapy developer.
• Atsena Therapeutics: Clinical-stage gene therapy company developing treatments for LCA1 and other IRDs using novel AAV capsids. Directly comparable in IRD target indications and AAV delivery platform.
• Beacon Therapeutics: Clinical-stage gene therapy company focused on IRDs including X-linked retinitis pigmentosa. Comparable as a fellow emerging AAV gene therapy developer targeting rare retinal disorders.
• ProQR Therapeutics: Clinical-stage RNA therapeutics company developing therapies for IRDs including Leber congenital amaurosis. Comparable in IRD focus and addressing similar genetic targets through a different modality.
• Ocugen: Clinical-stage biopharmaceutical company developing gene therapies for IRDs including RHO-adRP and modifier gene therapies. Comparable as a fellow public ophthalmology gene therapy developer.

Broad incumbents

• Iveric Bio (Astellas): Acquired by Astellas in 2023; developed Izervay for geographic atrophy. Previously sold Opus rights to two IRD gene therapy programs (BEST1 and RHO-adRP), establishing it as a relevant comparable in the ocular therapeutics and IRD gene therapy space.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/ocuphire-pharma

Revenue estimate

Valuation estimate

Number of profiles

Profiles6 records

Funding overview

Funding rounds10 records

Investors22 records

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

M&A2 records

Investments

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

Frequently asked questions about Opus Genetics(formerly Ocuphire Pharma)