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Subspecialties Retina, Glaucoma, Business and Innovation, Health Economics and Policy

Realizing Retinal Regeneration

Approximately 1 in 2,000 people worldwide are affected by inherited retinopathies (1) but few treatment options are available for retinal degeneration. There is also no cure for glaucoma, which affects 60 million people worldwide (2) and is caused by degeneration of retinal ganglion cells (RGCs) and their axon bundles that form the optic nerve. The FDA’s 2017 approval of LUXTURNA to treat inherited retinal degeneration caused by biallelic mutations in RPE65 has established viral vectors as a viable clinical therapy to monogenic retinal disease. Furthermore, advances in pluripotent stem cell techniques have enabled retinal pigment epithelium (RPE) to reach clinical trials as a cell therapy for treating age-related macular degeneration (AMD) (3). Now it is time to talk about in vivo reprogramming: a novel therapeutic approach that will circumvent the issues with subretinal transplantation of cell therapies and the challenge of targeting every monogenic condition with gene therapy.

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About the Authors

Evdokia Paza

Research Associate at Mogrify, Cambridge, UK.


Alice Lightowlers

Scientist at Mogrify, Cambridge, UK.


Tim Landy

Corporate Development Associate at Mogrify, Cambridge, UK.


Geraint Parfitt

Principal Scientist at Mogrify, Cambridge, UK.

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