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Ophthalmological Diseases

Research and development into rare ophthalmological diseases is often limited, making the development of appropriate drugs and therapies challenging. Our company is dedicated to addressing this gap by providing comprehensive drug and therapy development services for rare ophthalmological diseases.

Overview of Ophthalmological Diseases

Ophthalmological diseases encompass a wide range of conditions that affect the eyes and visual system. These diseases can be categorized into various subtypes, including but not limited to, ocular surface disorders, corneal diseases, retinal disorders, glaucoma, uveitis, and genetic eye diseases. Ophthalmological diseases can lead to visual impairment or blindness if left untreated, making their diagnosis and therapy of utmost importance.

  • Rare Ophthalmological Diseases
    Rare ophthalmological diseases are a subset of ophthalmological conditions that affect a small population of individuals. These diseases often have a genetic basis and are characterized by significant visual impairment or loss. As of the current update, within the European list of rare diseases, there are 53 ophthalmological diseases categorized as rare diseases. Additionally, 103 systemic diseases involve ophthalmological manifestations. This accounts for a portion of the overall 7,000 rare diseases listed.
Schemes of the eye structure, common ocular diseases, and ocular drug administration routes. Fig.1 Schemes of the eye structure, common ocular diseases, and ocular drug administration routes. (Nhàn N T T, et al., 2023)

Therapy Development for Ophthalmological Diseases

Cell-based Therapies

These therapies involve the transplantation of healthy cells or stem cells to replace damaged or degenerated cells in the retina or cornea. Stem cell-based therapies hold immense potential for regenerating damaged retinal cells and restoring vision in diseases like retinitis pigmentosa and Stargardt disease.

Gene Therapies

Many rare ophthalmological diseases are caused by specific genetic mutations. Gene therapies aim to correct these mutations or provide functional copies of the defective genes to restore vision. This approach has shown success for certain rare genetic eye diseases and holds great potential.

Drug Therapies

This includes the development of small molecules, biologics, and gene therapies that target specific molecular pathways involved in the disease progression. These therapies can be administered topically, orally, or through intravitreal injections, depending on the disease and its location.

Neovascularization and Angiogenesis

Conditions like age-related macular degeneration (AMD) and diabetic retinopathy are characterized by abnormal blood vessel growth in the retina. Therapies targeting angiogenesis pathways aim to inhibit the growth of these vessels and preserve visual function.

Our Services

The development of therapies for ophthalmological diseases involves targeting specific molecular pathways and cellular mechanisms involved in the disease progression. Our company is at the forefront of ophthalmological disease diagnostics and therapy development. We offer a wide range of services to aid in the development of novel therapies for ophthalmological diseases.

Rare Types of Ophthalmological Diseases

Therapy Development Platforms

We understand the importance of reliable animal models in eye disease research. Our experts have successfully developed and characterized a variety of animal models that mimic human eye diseases. Our comprehensive preclinical testing services include rigorous in vitro and in vivo evaluation of potential therapeutics. We utilize state-of-the-art technologies and methods to evaluate the efficacy, safety, and pharmacokinetics of drug candidates.

If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Nhàn, Nguyễn Thị Thanh, Daniel E. Maidana, and Kaori H. Yamada. "Ocular delivery of therapeutic agents by cell-penetrating peptides." Cells 12.7 (2023): 1071.
  • Sburlan, Elena Angelica, et al. "Rare ophthalmology diseases." Romanian Journal of Ophthalmology 63.1 (2019): 10.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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