Gene therapy can be used to treat retinitis pigmentosa, an inherited eye disease, and there will likely be an increase in approved gene therapies for ophthalmic conditions, explained Michel Michaelides, MD, FACP, consultant ophthalmologist and professor of ophthalmology at the University. Department of Genetics at the College London Institute of Ophthalmology.
Michel Michaelides, MD, FACP, explains how gene therapy can be used to treat retinitis pigmentosa, an inherited eye disease, and how it could be used in the future of eye care. Michaelides is Consultant Ophthalmologist at Moorfields Eye Hospital in the Department of Medical Retina, Inherited Eye Diseases and Pediatric Ophthalmology and Professor of Ophthalmology in the Department of Genetics at University College London Institute of Ophthalmology.
What is the meaning of RPGR (GTPase regulator) in retinitis pigmentosa and how can gene therapy address it?
Retinitis pigmentosa [RP] is genetically highly variable with over 100 different genes causing RP. RPGR is one of the most common causes of RP. It tends to be one of the most serious forms as well. It is therefore particularly significant that we are on the verge of potentially having an approvable drug for RPGR. So, fingers crossed for the phase 3 study that is currently underway.
How do you think gene therapy for the treatment of retinal diseases and other ophthalmic conditions will develop in the future?
So I’m very optimistic…there have been a few studies that haven’t quite reached their primary endpoint recently, but I think that’s to be expected in drug development. We have an approved therapy for one of our rarest diseases. And I’m sure there will be an increase in approved drugs over the next 5 years to really have a positive impact on the lives of our patients.