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.

Background

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.

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