Ophthalmic Disease Therapeutics Development Services

The development of ophthalmic therapeutics includes drug discovery, formulation development, and preclinical testing to ensure safety and efficacy. As a research service provider, Protheragen is committed to providing global pharmaceutical companies with one-stop solutions for the development of therapies for ophthalmic diseases.

Therapeutics Development in Ophthalmic Diseases

Ophthalmic disease therapeutics development encompasses preclinical research and translational efforts focused on creating safe, effective interventions to treat, manage, or halt the progression of vision-threatening ocular disorders. These therapies target a broad spectrum of conditions—including degenerative (age-related macular degeneration, glaucoma), genetic (retinitis pigmentosa, Leber congenital amaurosis), inflammatory (uveitis, scleritis), infectious (keratitis, endophthalmitis), and neoplastic (uveal melanoma, retinoblastoma) diseases—across all ocular tissues. Preclinical development in this field prioritizes elucidating disease pathogenesis, validating therapeutic targets, optimizing drug delivery to overcome ocular barriers, and evaluating efficacy and safety in disease-relevant models before advancing to clinical trials.

The preclinical pipeline for ophthalmic therapeutics is defined by its focus on addressing unique ocular challenges, such as the blood-ocular barrier (BOB) and blood-retinal barrier (BRB) that restrict systemic drug penetration, the need for tissue-specific targeting to minimize off-target damage to delicate structures like the retina and optic nerve, and the requirement for long-term efficacy to counter progressive vision loss. Core objectives include enhancing intraocular bioavailability, modulating disease-specific molecular pathways, preserving ocular function, and mitigating adverse effects—critical for regulatory approval and clinical adoption. Preclinical studies integrate in vitro cellular assays, ex vivo ocular tissue models, and in vivo disease models to generate robust data on pharmacokinetics (PK), pharmacodynamics (PD), efficacy endpoints, and safety profiles, forming the foundation for Investigational New Drug (IND) applications.

Fig.1 Nanotechnology-based strategies for ophthalmic disease therapeutics development. (Weng Y., et al., 2017)

This development process is tailored to the heterogeneous nature of ophthalmic diseases, each requiring distinct therapeutic strategies. For example, genetic ocular disorders often demand gene-based therapies to correct pathogenic variants, while degenerative conditions may rely on neuroprotective agents or anti-angiogenic compounds. Inflammatory diseases require immune-modulating therapies to suppress aberrant immune responses, while infectious conditions necessitate pathogen-specific antimicrobials. Preclinical validation prioritizes target engagement, dose-response relationships, and translation of efficacy from animal models to humans, as species-specific differences in ocular anatomy and disease progression can impact clinical predictability.

Key methodologies in preclinical ophthalmic therapeutics development include target validation via gene editing or knockdown systems, drug formulation optimization for ocular delivery routes (topical, intravitreal, periocular), and efficacy assessment using functional endpoints (electroretinography, visual acuity testing) and histopathological analysis. Preclinical studies also evaluate drug stability under physiological conditions, potential immunogenicity of biologics or gene therapies, and the risk of long-term ocular toxicity—all critical for ensuring patient safety. For combination therapies, preclinical work assesses synergistic effects and optimal dosing regimens to maximize efficacy while minimizing cumulative toxicity.

Preclinical development also emphasizes alignment with clinical needs, including addressing unmet medical needs for diseases with limited treatment options (e.g., dry age-related macular degeneration) and optimizing therapies for patient-centric delivery (e.g., sustained-release formulations to reduce injection frequency). By rigorously addressing these preclinical considerations, developers can accelerate the translation of therapies from the lab to the clinic, improving outcomes for patients with vision-threatening ocular conditions.

Ophthalmic Diseases Therapeutics Development

The field of ophthalmic drug discovery has witnessed a remarkable surge of advancements in the past decade, as evidenced by the steady stream of FDA approvals for groundbreaking therapeutics. Notably, the emergence of innovative therapeutic strategies, including gene therapy, stem cell therapy, and targeted modulation of pathways like the complement system, visual cycle, and mTOR signaling, has opened up new avenues for tissue repair, regeneration, and achieving lasting therapeutic benefits.

Table 1. Summary of active ocular gene therapy programs. (Rodrigues G. A., et al., 2019)

Disclaimer: Protheragen focuses on providing preclinical research service. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.

Our Services

Protheragen is at the forefront of ophthalmic disease therapeutics development, offering a comprehensive suite of services designed to accelerate the discovery and delivery of novel therapeutics for eye diseases.

The services we can provide include but are not limited to:

• Therapeutic Target Validation
  Protheragen provides comprehensive target validation services to confirm the biological relevance and druggability of candidate targets in ophthalmic diseases. This includes functional validation via gene editing (CRISPR/Cas9, siRNA/shRNA) and overexpression systems in ocular cell lines (RPE, photoreceptor, corneal epithelial cells) and ex vivo ocular tissues. We evaluate target modulation effects on disease-related phenotypes, such as cell survival, inflammation, angiogenesis, and neurodegeneration, using high-content imaging, flow cytometry, and molecular assays (qPCR, Western blotting). Clients can access support for target engagement studies, including binding affinity analysis for small molecules and biologics, and downstream pathway characterization to validate the mechanism of action, ensuring targets are clinically relevant and therapeutically actionable.
• Ocular Formulation and Delivery Optimization
  We specialize in developing and optimizing ocular formulations to enhance bioavailability, target specificity, and therapeutic efficacy. For gene therapies, we optimize AAV vector capsid engineering, payload design, and delivery routes (intravitreal, subretinal) to improve transduction efficiency and tropism for target ocular tissues. For small molecules and biologics, we develop nanotherapeutic formulations (LNPs, polymeric micelles, hydrogels) and sustained-release systems (implants, microspheres) to overcome ocular barriers and prolong intraocular retention. We evaluate formulation performance via in vitro permeation assays (corneal, scleral, RPE models), release kinetic studies, and in vivo distribution analysis, measuring drug concentrations in target tissues (retina, uvea, vitreous humor) and systemic organs to optimize dosing and minimize off-target exposure.
• Preclinical Efficacy Evaluation
  Our preclinical efficacy services assess therapeutic performance in disease-relevant in vitro, ex vivo, and in vivo models. In vitro assays include cell viability, apoptosis, inflammation, and angiogenesis testing to evaluate candidate therapies in ocular cell lines and 3D organoids (retinal, corneal). Ex vivo studies use freshly isolated ocular tissues (human donor eyes, rabbit uvea, mouse retinas) to evaluate therapeutic penetration and efficacy in a physiological context. In vivo efficacy studies employ gold-standard disease models—including EAU (uveitis), rd1/rd10 (retinitis pigmentosa), laser-induced choroidal neovascularization (wet AMD), and murine glaucoma models—with assessments via clinical scoring, functional endpoints (electroretinography, visual acuity), histopathological analysis, and molecular profiling to quantify therapeutic effects on disease progression.
• Gene Therapy Preclinical Development
  Protheragen offers specialized preclinical services for gene-based ophthalmic therapies, including gene editing, gene augmentation, and gene silencing. We support vector design and optimization (AAV, lentivirus, adenovirus), payload engineering (CRISPR constructs, cDNA, siRNA), and transduction efficiency testing in ocular cell lines and in vivo models. We evaluate gene editing precision (on-target/off-target effects) via high-throughput sequencing, transgene expression levels via qPCR and immunofluorescence, and functional efficacy via phenotypic rescue assays. Additionally, we assess immunogenicity of viral vectors and gene editing tools, measuring immune cell infiltration and cytokine production in in vivo models to mitigate safety risks.
• Preclinical Safety and Toxicology Assessment
  We conduct comprehensive preclinical safety studies to support IND submissions, adhering to GLP standards and global regulatory guidelines. Ocular safety testing includes local irritation evaluation, histopathological analysis of ocular tissues (retina, cornea, optic nerve) to assess structural damage or inflammation, and functional assessments to evaluate vision preservation. Systemic toxicity studies measure hematological parameters, organ function, and body weight changes, with a focus on off-target organ exposure. For biologics and gene therapies, we evaluate immunogenicity (anti-drug antibody detection) and hypersensitivity responses. All studies generate detailed reports with statistically robust data, including dose-response relationships and safety margins, to support regulatory review and clinical trial design.
• Pharmacokinetics (PK) and Pharmacodynamics (PD) Profiling
  Protheragen provides PK/PD profiling services to characterize the absorption, distribution, metabolism, and excretion of ophthalmic therapeutics, as well as their relationship to therapeutic efficacy. We measure drug concentrations in ocular fluids (aqueous humor, vitreous humor) and tissues (retina, uvea, cornea) and systemic matrices (plasma, organs) via LC-MS/MS and other analytical techniques. PD assessments include target engagement, downstream pathway modulation, and correlation with efficacy endpoints (e.g., VEGF reduction in AMD models, photoreceptor survival in degenerative models). We also evaluate PK/PD relationships across different doses and delivery routes to optimize dosing regimens, ensuring therapeutic concentrations are maintained at target sites while minimizing toxicity.

Types of Ophthalmic Diseases Therapeutics

By Therapeutic Strategies

• Antibiotic Development
• Antifungal Drug Development
• Antivatival Drug Development
• Anti-inflammatory Drug Development
• Targeted Therapy Development

By Molecule Types

• Small Molecule Drug Development
• Cell Therapy Development
• Gene Therapy Development
• Therapeutic Antibody Development
• Therapeutic Peptide Development
• Therapeutic Protein Development

We utilize state-of-the-art equipment and techniques, including high-throughput screening platforms, advanced analytical instrumentation, and specialized in vitro and in vivo models, to drive the discovery and development of innovative ocular therapies. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

References

• Weng Yuhua, et al. "Nanotechnology-based strategies for treatment of ocular disease." Acta pharmaceutica sinica B 7.3 (2017): 281-291.
• Rodrigues, Gerard A., et al. "Pharmaceutical development of AAV-based gene therapy products for the eye." Pharmaceutical research 36.2 (2019): 29.