Gene Therapy for hereditary blindness
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. This project aims to conduct gate keeper experiments in preparation to advance to Phase IIa clinical studies.
A Starfish Innovations project