LEBER CONGENITAL AMAUROSIS MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2024-2031)

Leber Congenital Amaurosis Market, By Treatment Type (Gene Therapy, Pharmaceutical Drugs, Retinal Prosthesis, Assistive Devices, and Others), By Target Gene (RPE65, GUCY2D, AIPL1, RPGRIP1, CEP290, and Other Genes), By End User (Hospitals, Specialized Eye Clinics, Ophthalmology Research Centers, Home Care Settings, and Others), By Geography (North America, Latin America, Europe, Asia Pacific, Middle East & Africa)

In April 2023, Researchers from the National Eye Institute found that Reserpine, an FDA-approved drug originally used for high blood pressure, shows potential for treating Leber congenital amaurosis type 10 (LCA10). In laboratory models, Reserpine helped preserve light-sensitive photoreceptors affected by LCA10, a genetic disorder caused by CEP290 mutations.
In November 2022, Editas Medicine presented clinical data from the Phase 1/2 BRILLIANCE trial of EDIT-101, a CRISPR/Cas9-based therapy for Leber congenital amaurosis 10 (LCA10), a rare retinal disorder. The trial showed that three of the 14 patients treated experienced significant improvements in best corrected visual acuity (BCVA) and other visual function measures. EDIT-101 targets the IVS26 CEP290 mutation, affecting around 1,500 patients in the U.S., for which there is currently no effective treatment.