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Gene therapy refers to the introduction of normal exogenous genes into target cells to correct or compensate the diseases caused by defective and abnormal genes for the purpose of therapy. The eye is a closed sphere, and the drug is injected into the local eyeball only, which has little influence on the whole body, so it is suitable for gene therapy. In addition, there are more than 220 genes associated with ophthalmology, and any mutation may cause vision loss and blindness.
The world's first drug to treat ophthalmic genetic disease is LUXTURNA®, a drug developed by Spark Therapeutics of the United States and used to treat Leber congenital amaurosis (LCA2, RPE65 gene mutation), which was approved by the U.S. FDA in December 2017.It is seen as an important milestone in human gene therapy field. Gene therapy using safe viral vectors to deliver nucleic acid drugs to correct genetic abnormalities or express normal gene products is one of the most promising strategies for the treatment of blind-causing eye diseases.
As the first ophthalmic gene therapy company in China, Neurophth is committed to the research, development and industrialization of gene therapy drugs with adeno-associated virus vectors, to explore and develop new therapies for patients with ophthalmic diseases worldwide.