Dr Michel Michaelides Speaks About Implications of Gene Therapy Study Results in Ophthalmology

The results of a study showing improved functional vision and retinal sensitivity have implications for gene therapy in ophthalmological conditions, explained Michel Michaelides, MD, FACP, a consultant ophthalmologist and a professor of ophthalmology at the University College London Institute of Ophthalmology's Genetics Department.

Michel Michaelides, MD, FACP, explained what research findings presented at the American Academy of Ophthalmology (AAO) 2022 Conference could mean for the potential of gene therapy to improve functional vision and retinal sensitivity in patients with retinitis pigmentosa.


What were some of the results from your study that was presented at AAO 2022 and what do they say about the potential for the investigational gene therapy botaretigene sparoparvovec (bota-vec)?

We presented our phase 1/2 data of the entire trial and also including the randomized controlled expansion phase. And what was exciting was that, in addition to the safety that we've previously alluded to, we were able to show that, comparing treated patients to a concurrent control group, there were significant improvements in both retinal sensitivity and functional vision. And I was particularly excited about the functional vision because that's what patients can do with the improvement in their retinal sensitivity and, arguably, that's the most important thing.

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