Ophthalmology has long been a fruitful area for biotechnology innovation. In highly prevalent conditions associated with ageing, such as age-related macular degeneration, or with chronic disease, such as diabetic macular oedema, vascular endothelial growth factor inhibitors have made important contributions to stabilizing vision over the past two decades. Incremental innovation has steadily improved efficacy while lowering the frequency of injections. And newer targets, such as components of the complement cascade and angiopoietin, are extending the range of therapeutic options available.

Progress in inherited retinal disease (IRD) has been slower, however. No gene therapy has gained approval in an IRD since Luxturna (voretigene neparvovec) did so in 2017. Moreover, Luxturna, which is approved for treating biallelic RPE65 mutation-associated retinal dystrophy, has not been a commercial success, while some of the bigger players have written off billions of dollars in R&D investments because of acquisitions that have failed to deliver.

Classical gene replacement approaches continue to be challenging because of the huge variety of genes associated with IRD – about 270 have been described. Developing individual gene therapies for each is not feasible with current technologies. A newer wave of ‘mutation-agnostic’ gene therapies has moved into the clinic in the last year. These do not address a specific disease-causing mutation but aim to slow the rate of vision loss by improving the metabolic status of damaged photoreceptor cells. Exon editing and cellular reprogramming are at an earlier stage of development, but could also provide ways of addressing multiple disease-associated mutations with a single therapy. The field has never been short of creative ideas – but it needs some clinical success.

Companies mentioned in this episode:
Ascidian Therapeutics, Beacon Therapeutics, Biogen, Editas Medicine, Endogena Therapeutics, Genentech, Gyroscope Therapeutics, Johnson & Johnson, MeiraGTx, Nightstar Therapeutics, Novartis, Ocugen, Ophthotech, Pfizer, Rezolute, Roche, SparingVision, Spark Therapeutics, Thrombogenics, ViGeneron

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