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Ophthalmology

According to the World Health Organization, there are approximately 2.2 billion people affected by visual impairment globally. Eye conditions are varied and may have a genetic cause or develop as a result of aging, injury or other health conditions.

Whilst many ocular conditions can be treated effectively, some can only be partially treated or have no treatment at all. Approximately 1 in 2,000 people in the world have an inherited retinal degeneration. For a small percentage of these patients, in particular those with mutations in RPE65, one gene therapy may be a suitable treatment, but for the majority, no effective therapy exists. Similarly, of the 10.4 million people worldwide living with age-related macular degeneration, geographic atrophy, the most severe form of dry age-related macular degeneration, accounts for approximately 5 million cases. Unlike for patients with wet age-related macular degeneration, there is no treatment available for dry age-related macular degeneration. For a number of corneal diseases and the patients affected, corneal transplants can be an effective treatment, however, this option relies on the quality and availability of donor corneal tissue.

As an immune-privileged organ, which is easily accessible for the delivery of treatment and for the monitoring of disease, the eye is an ideal candidate for cell and gene therapies. Furthermore, the regulatory approvals of an autologous stem-cell therapy and gene therapy, within the last decade, have paved the way for future cell and gene therapies to achieve market authorization.

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Cell Reprogramming for Ophthalmology

Here we discuss cellular reprogramming in the context of ophthalmology, and in particular, the reprogramming of ocular cell types to support the development of ex vivo transplants and novel in vivo reprogramming therapies for retinal degeneration and other eye conditions.

White Paper
Realizing Retinal Regeneration: How can data-driven in vivo reprogramming be used to treat retinal and optic nerve degeneration?
2020   |   Evdokia Paza, Alice Lightowlers, Tim Landy, Geraint Parfitt
Approximately 1 in 2,000 people worldwide are affected by inherited retinopathies but few treatment options are available for retinal degeneration. The FDA’s 2017 approval of LUXTURNA to treat inherited retinal dege...