Michel Michaelides, MD, FACP, on Gene Therapy's Potential in Retinitis Pigmentosa


The professor of ophthalmology at the University College London Institute of Ophthalmology discussed the likely rise of gene therapies in inherited retinal diseases.

This content originally appeared on our sister site, AJMC.

AJMC spoke with Michel Michaelides, MD, FACP, about the potential of gene therapy in treating retinitis pigmentosa, an inherited eye disease, and how it could be used in the future of eye care. Michaelides serves a consultant ophthalmologist at Moorfields Eye Hospital in the Department of Medical Retina, Inherited Eye Disease and Paediatric Ophthalmology and is a professor of ophthalmology at the University College London Institute of Ophthalmology's Genetics Department.


What is the significance of the RPGR (GTPase regulator) gene as it pertains to retinitis pigmentosa and how can gene therapy address it?

Retinitis pigmentosa [RP] is genetically very variable with more than 100 different genes that cause RP. RPGR is one of the more common causes of RP. It tends to be one of the more severe forms as well. So it's particularly meaningful that we are on the cusp of potentially having an approvable drug for RPGR. So, fingers crossed for the phase 3 study that is being undertaken currently.

How do you expect for gene therapy for the treatment of retinal diseases and other ophthalmic conditions to develop in the future?

So, I'm very optimistic....there have been a couple of studies that haven't quite achieved their primary end point recently, but I think that's to be expected in drug development. We have an approved therapy for one of our rarer conditions. And I'm certain there will be an increase in approved drugs over the next 5 years to really make a positive impact on our patients lives.

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