Opus Genetics, a Penn startup based on technology developed by Jean Bennett, MD, PhD, the F.M. Kirby Emeritus Professor of Ophthalmology at Penn Medicine, and colleagues announced encouraging one-month data from its first pediatric patient treated with OPGx-LCA5, a gene therapy targeting LCA5-related inherited retinal disease. The Phase 1/2 clinical trial is taking place at Penn.
According to the data, the 16-year-old patient, who was treated in February, showed “meaningful improvement in visual function” after receiving a single subretinal injection and did not experience drug-related adverse events. Read more here.