Ophthalmic Disease Model Development Services

As a leading research service provider, Protheragen has established itself as a trusted partner in the field of ophthalmic disease model development. With decades of expertise and a deep understanding of the complexity of eye-related pathologies, our team of experienced scientists is dedicated to providing specialized ophthalmic disease model development services to researchers and pharmaceutical companies for therapeutic development.

Disease Model Development in Ophthalmic Diseases

Ophthalmic disease model development refers to the systematic creation and characterization of in vitro, ex vivo, and in vivo systems that recapitulate the anatomical, physiological, and pathophysiological features of human ocular disorders. These models serve as indispensable tools in preclinical research, bridging the gap between molecular biology discoveries and translational drug development. The core objective of model development is to replicate key disease phenotypes—such as retinal degeneration, intraocular pressure elevation, or corneal epithelial dysfunction—and the underlying molecular mechanisms, enabling researchers to dissect disease etiology, validate therapeutic targets, and evaluate the efficacy and safety of candidate interventions.

Ideal ophthalmic disease models must exhibit high translational relevance, mirroring human ocular anatomy and pathology with sufficient fidelity. For in vivo models, this includes conservation of retinal architecture (e.g., three nuclear layers separated by two plexiform layers), functional neuronal cell types, and molecular pathways governing disease progression. In vitro models, such as induced pluripotent stem cell (iPSC)-derived retinal organoids or corneal epithelial cultures, offer complementary value by enabling high-throughput screening and mechanistic studies while reducing reliance on animal models. Model development also encompasses rigorous phenotypic validation using advanced ophthalmic imaging and functional assessment tools, such as optical coherence tomography (OCT), fluorescein angiography, electroretinography (ERG), and optokinetic response testing, to ensure consistency and reproducibility across studies.

Fig.1 Schematic illustration of ocular structures and barriers. (Solanki A., et al., 2016)

The scope of ophthalmic disease model development spans a broad spectrum of ocular conditions, from common disorders like age-related macular degeneration (AMD), glaucoma, and dry eye disease (DED) to rare inherited retinal diseases (IRDs) such as retinitis pigmentosa and Leber congenital amaurosis. Each model is tailored to the specific disease's pathogenic drivers—whether genetic mutations, environmental triggers, or systemic disease complications—and optimized for the research goal, whether target validation, drug screening, or preclinical proof-of-concept (PoC) studies. By providing biologically relevant systems, model development accelerates the translation of scientific discoveries into clinical therapies, mitigating the high failure rate of ophthalmic drugs in clinical trials due to inadequate preclinical validation.

Ophthalmic Diseases Model Research

In vivo animal models remain the gold standard for preclinical ophthalmic research due to their ability to replicate complex ocular physiology and disease progression in a whole-organism context. Rodents, rabbits, dogs, pigs, and non-human primates are commonly used, each offering unique advantages based on the disease of interest. For retinal disorders like diabetic retinopathy (DR) and AMD, zebrafish models have emerged as powerful tools due to their high anatomical and functional conservation of the retina, large eye size facilitating in vivo manipulation, and inherent regenerative capacity—traits that enable rapid assessment of compounds targeting retinal repair. Rodent models, meanwhile, are widely utilized for glaucoma research, with established protocols for inducing elevated intraocular pressure (IOP) via steroid application, episcleral vein cauterization, or corneal microbead injection, recapitulating retinal ganglion cell loss and optic nerve damage observed in human glaucoma. Non-human primates, with their near-identical ocular anatomy to humans, are critical for late-stage preclinical evaluation of therapies targeting posterior segment diseases, ensuring translational relevance for clinical trials.

In vitro and organoid models have revolutionized ophthalmic disease research by enabling mechanistic studies at the cellular and molecular level while addressing ethical concerns and reducing animal use. iPSC-derived models, including retinal pigment epithelial (RPE) cells, photoreceptors, and retinal organoids, have become indispensable for studying IRDs, as they can be generated from patient-derived cells to recapitulate disease-specific mutations and phenotypes. These models allow researchers to dissect the pathogenic effects of specific genetic variants, screen for compounds that rescue cellular dysfunction, and evaluate gene therapy approaches. For ocular surface diseases like DED and allergic conjunctivitis, in vitro models of corneal epithelial cells, conjunctival fibroblasts, and lacrimal gland cells provide controlled systems to study barrier function, inflammation, and tear film dynamics. Advanced techniques, such as 3D organoid culture, further enhance model relevance by replicating tissue architecture and intercellular interactions, while integration with artificial intelligence (AI) enables high-throughput image analysis and phenotypic quantification, accelerating research workflows and improving data accuracy.

Our Services

Protheragen's ophthalmic disease model development services are designed to meet the complex needs of modern preclinical research. Our commitment to scientific excellence, customization, and ethical practice positions us as a leader in the field, supporting the advancement of ophthalmic disease research and the development of new therapeutics.

Ocular Surface Disease Model Development

We can develop customized models for a full spectrum of ocular surface disorders to support preclinical research on disease mechanisms and therapeutic interventions. For dry eye disease (DED), models can be induced by environmental factors (low humidity, sleep deprivation), chemical agents (benzalkonium chloride, atropine), or meibomian gland dysfunction (MGD), recapitulating key phenotypes such as reduced tear production, corneal epithelial damage, and inflammation. Services also cover models for allergic conjunctivitis, corneal diseases (viral/fungal keratitis, corneal neovascularization, neurotrophic keratitis), and bullous keratopathy (corneal endothelial injury models). Each model is validated using clinical-grade endpoints, including tear film break-up time, corneal staining, conjunctival hyperemia assessment, and histological analysis of ocular surface tissues, to ensure relevance to human disease.

Posterior Segment Disease Model Development

Specialized model development for posterior segment disorders—leading causes of irreversible blindness—is available to support research across therapeutic modalities. For age-related macular degeneration (AMD), models of both dry (drusen accumulation, retinal pigment epithelial atrophy) and wet (choroidal neovascularization) subtypes can be created, optimized for evaluating anti-VEGF therapies, neuroprotective agents, and gene therapies. For diabetic retinopathy (DR), models of non-proliferative and proliferative disease are developed by inducing retinal vascular leakage, microaneurysm formation, and neuronal dysfunction, facilitating research into glucose-lowering agents, anti-inflammatory therapies, and vascular protective compounds. Additional posterior segment models include those for inherited retinal diseases (IRDs) such as retinitis pigmentosa, Stargardt disease, and Leber congenital amaurosis, retinal central vein occlusion, retinopathy of prematurity, and choroidal neovascularization.

Glaucoma and Anterior Segment Disease Model Development

Tailored models for glaucoma and anterior segment disorders are developed to replicate disease-specific pathophysiology and functional deficits. For glaucoma, hypertensive models (via steroid application, episcleral vein cauterization, or corneal microbead injection) and normotensive models can be constructed, recapitulating elevated intraocular pressure (IOP), retinal ganglion cell loss, optic nerve cupping, and visual function impairment. These models are validated using IOP measurement, optical coherence tomography (OCT) for retinal nerve fiber layer thickness assessment, electroretinography (ERG), and optokinetic response testing to evaluate therapeutic efficacy. Anterior segment model services also include cataract models (induced by sodium selenite, age-related, or diabetic conditions) and uveitis models (endotoxin-induced uveitis, EIU), enabling preclinical research into cataract therapies, anti-inflammatory agents, and immunomodulatory interventions.

Genetic and Custom Model Development

Specialized genetic model development services support research into inherited ophthalmic disorders and target validation. Using state-of-the-art gene-editing technologies (CRISPR-Cas9, TALENs), custom knockout, knock-in, and transgenic animal models (rodents, zebrafish) can be created for specific genetic variants associated with IRDs, glaucoma, and other inherited ocular diseases. For client-specific research needs, tailored models using patient-derived induced pluripotent stem cells (iPSCs) are developed, generating retinal organoids, retinal pigment epithelial cells, and photoreceptors that recapitulate disease phenotypes at the cellular level. These models are ideal for target validation, mechanistic studies, and high-throughput screening of candidate compounds, as they retain the genetic background of human disease. Comprehensive characterization of genetic models—including genotyping, phenotypic analysis, and functional assessment—is provided to ensure suitability for clients' research objectives.

In Vitro and Organoid Model Development

In vitro and organoid model development services complement in vivo systems, supporting high-throughput screening and mechanistic research. Services include primary and immortalized cell cultures of ocular tissues (corneal epithelial cells, conjunctival fibroblasts, retinal pigment epithelial cells, retinal ganglion cells, Müller glial cells) and 3D co-culture systems that replicate tissue-tissue interactions. For advanced translational research, iPSC-derived retinal organoids and corneal organoids are developed, recapitulating the anatomical structure and functional properties of human ocular tissues. These organoid models enable studies of early disease development, cellular crosstalk, and therapeutic response in a human-relevant system, reducing reliance on animal models and improving translational predictivity. In vitro models can also be optimized for high-throughput drug screening, integrating automated imaging and artificial intelligence (AI)-driven phenotypic analysis to accelerate compound evaluation.

Model Validation and Phenotypic Characterization Services

Comprehensive validation and phenotypic characterization services using advanced ophthalmic technologies ensure the reliability and translational relevance of developed models. Services include in vivo imaging (slit-lamp photography, color fundoscopy, fundus autofluorescence, fluorescein angiography, OCT), functional assessments (ERG, visual evoked potential, optokinetic response, pupillometry), and histological/molecular analysis (tissue sectioning, immunohistochemistry, gene expression profiling, protein analysis). For each model, robust validation criteria aligned with clinical endpoints are established, ensuring key disease phenotypes are consistently replicated and measurable. This rigorous characterization enables clients to evaluate therapeutic efficacy, target engagement, and safety profiles confidently, supporting data-driven decisions in preclinical drug development. Validation services adhere to Good Laboratory Practice (GLP) standards, ensuring compliance with regulatory requirements for global preclinical submissions.

Cell-based Models Development Service

• Cell Line Development Service
• Primary Cell Development Service
• iPSC Development Service

Organoid Models Development Service

• Adult Stem Cell (ASC) Source
• Embryonic Stem Cell (ASC) Source
• Induced Pluripotent Stem Cell (iPSC) Source
• Patient-derived Organoid (POD)

Animal Models Development Services

• Surgical Models
• Genetically Engineered Models
• Humanized Animal Models
• Customized Animal Models

We offer customized model development services to meet the specific needs of each research project. Our team works closely with clients to understand their requirements and to design models that accurately replicate the disease of interest. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

Reference

• Solanki, Aum, et al. "Ocular drug delivery: impact of in vitro cell culture models." Nano-Biomaterials For Ophthalmic Drug Delivery (2016): 483-494.