Ocuphire Pharma Inc
Opus Genetics is a clinical-stage ophthalmic biopharmaceutical company dedicated to developing transformative gene therapies for inherited retinal diseases (IRDs) and other retinal and refractive disorders. Their mission is to build an efficient and sustainable pipeline of therapies to address mutations causing loss of vision, leveraging world-class science and innovative manufacturing processes. They focus on targeting mutations in genes such as RHO, RDH12, BEST1, NMNAT1, MERTK, and CNGB1, among others, with a goal to bring first-in-class treatments to patients in need.
Industries
Nr. of Employees
small (1-50)
Ocuphire Pharma Inc
Farmington Hills, Michigan, United States, North America
Products
AAV gene therapy candidate for LCA5-associated retinal degeneration
AAV-mediated gene augmentation therapy designed to deliver a functional LCA5 gene to photoreceptors to preserve or restore vision.
AAV gene therapy candidate for BEST1-related bestrophinopathies
AAV-mediated BEST1 gene delivery to retinal pigment epithelium (RPE) cells to restore bestrophin-1 function and RPE support for photoreceptors.
AAV gene therapy candidate for RHO-associated autosomal dominant retinitis pigmentosa
Gene replacement strategy designed to preserve rod photoreceptors by replacing or supplementing functional RHO gene expression.
AAV gene therapy candidates for RDH12, MERTK, NMNAT1, CNGB1-associated retinal dystrophies
A portfolio of AAV-mediated gene augmentation therapies targeting multiple IRD genes with preclinical evidence of functional improvement and program-specific development plans.
Phentolamine ophthalmic solution 0.75% (topical pupil-modulating formulation)
Non-selective alpha-adrenergic antagonist eye drop formulation developed to moderately reduce pupil size to improve near vision (presbyopia) and low-light visual disturbances in post-keratorefractive patients.
Small-molecule Ref-1 inhibitor for diabetic retinopathy (APX3330)
Orally or systemically delivered small-molecule inhibitor targeting Ref-1 to modulate inflammation, angiogenesis, and oxidative stress in diabetic retinopathy; Phase 2 data reported and SPA obtained for Phase 3 planning.
AAV gene therapy candidate for LCA5-associated retinal degeneration
AAV-mediated gene augmentation therapy designed to deliver a functional LCA5 gene to photoreceptors to preserve or restore vision.
AAV gene therapy candidate for BEST1-related bestrophinopathies
AAV-mediated BEST1 gene delivery to retinal pigment epithelium (RPE) cells to restore bestrophin-1 function and RPE support for photoreceptors.
AAV gene therapy candidate for RHO-associated autosomal dominant retinitis pigmentosa
Gene replacement strategy designed to preserve rod photoreceptors by replacing or supplementing functional RHO gene expression.
AAV gene therapy candidates for RDH12, MERTK, NMNAT1, CNGB1-associated retinal dystrophies
A portfolio of AAV-mediated gene augmentation therapies targeting multiple IRD genes with preclinical evidence of functional improvement and program-specific development plans.
Phentolamine ophthalmic solution 0.75% (topical pupil-modulating formulation)
Non-selective alpha-adrenergic antagonist eye drop formulation developed to moderately reduce pupil size to improve near vision (presbyopia) and low-light visual disturbances in post-keratorefractive patients.
Small-molecule Ref-1 inhibitor for diabetic retinopathy (APX3330)
Orally or systemically delivered small-molecule inhibitor targeting Ref-1 to modulate inflammation, angiogenesis, and oxidative stress in diabetic retinopathy; Phase 2 data reported and SPA obtained for Phase 3 planning.
Services
Preclinical development and translational studies
In vivo and in vitro efficacy testing in animal models and human iPSC models to evaluate candidate retinal therapeutics.
Clinical development and trial execution for ocular therapies
Protocol design, endpoint selection, and operational execution of Phase 1/2/3 ophthalmic clinical trials.
Pharmaceutical manufacturing for ophthalmic products
Manufacturing of pharmaceutical-grade gene therapy vectors and preservative-free ophthalmic formulations tailored for rare disease populations.
Regulatory planning and strategy
Regulatory planning including SPA negotiations, IND-enabling strategy, and alignment of trial design to support potential marketing applications.
Expanded Access (compassionate use) program management
Processes and policy frameworks to evaluate and provide investigational medicines to eligible patients outside clinical trials in compliance with legal and regulatory requirements.
Preclinical development and translational studies
In vivo and in vitro efficacy testing in animal models and human iPSC models to evaluate candidate retinal therapeutics.
Clinical development and trial execution for ocular therapies
Protocol design, endpoint selection, and operational execution of Phase 1/2/3 ophthalmic clinical trials.
Pharmaceutical manufacturing for ophthalmic products
Manufacturing of pharmaceutical-grade gene therapy vectors and preservative-free ophthalmic formulations tailored for rare disease populations.
Regulatory planning and strategy
Regulatory planning including SPA negotiations, IND-enabling strategy, and alignment of trial design to support potential marketing applications.
Expanded Access (compassionate use) program management
Processes and policy frameworks to evaluate and provide investigational medicines to eligible patients outside clinical trials in compliance with legal and regulatory requirements.
Expertise Areas
- Gene therapy development for inherited retinal diseases
- Ophthalmic clinical trial management
- Preclinical translational research (animal and iPSC models)
- Pharmaceutical-grade manufacturing and CMC for rare ophthalmic products
Key Technologies
- AAV-based gene delivery
- Subretinal delivery
- Human iPSC disease modeling
- Rodent (mouse/rat) preclinical models