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Gene therapy for hereditary blindness

Start project: 2018
Involved institutes: LUMC
Stage: Preclinical
Status: Completed

Description: Hereditary retinitis pigmentosa (RP) is a rare genetic disease of the eye, which causes vision loss in 1 in 4,000 people, due to mutations in the CRB1 gene. An AAV-based gene therapy is being developed for the treatment of RP. After the successful execution of the experiments with the funding from Starfish Innovations, this technology was in April 2020 licensed out to Horama to further advance to clinical studies.