Ophthalmological rare diseases are mostly inherited blinding eye diseases that severely damage the structure and function of the eye and seriously affect the physical and mental health of patients and their offspring. Ophthalmic diseases designated as orphan conditions have heterogeneous causes, including inherited genetic defects (e.g., Leber’s congenital claustrophobia (LCA) and Stargardt’s disease), autoimmune syndromes, or neovascular damage.
In recent years, the underlying genetic factors of a range of ophthalmological rare diseases have been identified, and the development of relevant animal models has led to significant breakthroughs in preclinical research. The establishment and availability of stable and reliable animal models for ophthalmological rare diseases have incalculable potential for disease pathogenesis studies, drug target studies, and evaluation of therapeutic effects. Current orphan drug designations in this therapeutic area cover a wide range of therapeutic strategies, including gene therapy, peptides that induce immune tolerance, and neovascularization.
Ophthalmological Rare Diseases Animal Models
l Animal Models for Retinal Degeneration
Orphan diseases characterized by retinal degeneration include porcine psychogenic retinitis, LCA, and Stargardt’s disease. Their etiology is closely related to one or more underlying genetic defects that cause photoreceptor dysfunction. Our team of experts provides clients with a variety of animal models for the development of therapies for rare retinal degenerative diseases, including
Animal Models for Retinitis Pigmentosa
Animal Models for Stargardt’s Disease
Animal Models for Leber’s Congenital Amaurosis
l Animal Models for Ocular Autoimmune Diseases
Non-infectious chronic uveitis and keratoconjunctivitis are two ocular autoimmune diseases designated as orphan diseases. We are committed to providing customized animal models for these two ocular autoimmune diseases and performing various tests and assays to evaluate potential treatments’ efficacy.
Animal Models for Non-infectious Chronic Uveitis
Animal Models for Keratoconjunctivitis
l Animal Models for Ocular Neovascularization
Retinopathy of prematurity (ROP) and neovascular glaucoma (NVG) are ocular neovascularization diseases for which therapies have been granted orphan status by the COMP.
Animal Models for Retinopathy of Prematurity
Animal Models for Neovascular Glaucoma
Advantages of Our Ophthalmological Rare Diseases Modeling
l Multiple available animal models including mice, rats, dogs, canines, pigs, and non-human primates
l Custom model development to meet specific client requirements
l Professional animal model characterization
l Extensive experience in preclinical research
l Competitive pricing and fast turnaround time
For rare ophthalmic diseases such as retinitis pigmentosa and non-infectious chronic uveitis, our company offers not only a variety of naturally occurring disease models but has also developed a series of eye-related gene editing models. These models have good phenotypic data and can be genetically stable and applied to various studies of ophthalmological rare diseases. Meanwhile, our professional and first-class analysis platform can provide the whole process of ophthalmic pharmacodynamic analysis including ocular injection administration, ocular biopsy, ocular tissue sampling, pathology analysis, and molecular testing of gene and protein expression.
Reference
Nhàn, N. T. T.; et al. Ocular delivery of therapeutic agents by cell-penetrating peptides. Cells, 2023, 12(7): 1071.
