Saving vision with gene therapies

Vinit Mahajan, MD, PhD, associate professor of ophthalmology and vice-chair for research, is leading two human gene therapy trials, one for dry age-related macular degeneration (AMD) and a second for an inherited form of retinal disease caused by the retinitis pigmentosa GTPase regulator gene. Diseases often originate from mutated genes, and gene therapy is a therapeutic approach that places healthy genes into patient cells to prevent and treat disease. Mahajan’s trials are sponsored by two clinical-stage gene therapy companies, Gyroscope Therapeutics Limited and MeiraGTx UK II Ltd. Currently, they are screening patients with AMD and inherited retinal disease via saliva sampling genetic testing kits in preparation for the intervention phase. Qualifying participants who carry genes of interest are enrolled into a natural history study, which closely evaluates disease progression. In the coming months, they hope to perform surgery to deliver the approved gene therapy drug to patients. To find out more, send an email to VisionGenetics@stanford.edu. An up-to-date list of all clinical trials can be viewed here.