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October 27, 2017

Could groundbreaking gene therapies offer a breakthrough to retinal disease sufferers?

Hereditary retinal disorders are a result of genetic defects, and there are no approved treatments for most of these conditions.

Hereditary retinal disorders are a result of genetic defects, and there are no approved treatments for most of these conditions.

However, it is expected that gene therapies that are currently being developed for these diseases may soon become available, enabling the treatment of vision loss and the slowing of disease progression.

New treatment approaches, including gene therapies, have been gaining significant traction in preclinical and clinical trials in ophthalmology.

A novel approach

In gene therapy, a specific gene is delivered to a patient’s target cells via a viral vector.

These viral vectors are converted from infectious agents, such as retroviruses and adenoviruses, into gene delivery vehicles.

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Although there are no gene therapies in the ophthalmology market, they make up a notable proportion of the ophthalmology pipeline, accounting for the fourth largest share of products in development.

Promising products set to emerge

Gene therapies have shown encouraging levels of safety and effectiveness, and therefore represent strong commercial opportunities.

Recently, Spark Therapeutics put forward for review its orphan drug Luxturna, for the treatment of biallelic RPE65-mediated inherited retinal disease.

The drug is the first adeno-associated viral vector to reach this stage.

On October 12, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee unanimously recommended its approval.

If, as expected, the FDA approves Luxturna in 2018, it will become the first pharma treatment for an inherited retinal disease, and the first FDA-approved gene therapy for any inherited disease.

A new chapter in retinal treatment?

There are gene therapies in active development for a number of ophthalmological diseases, including age-related macular degeneration, diabetic retinopathy, glaucoma and retinitis pigmentosa.

These developments are very promising, and the potential applications of gene therapy within ophthalmology are broad.

This could mean a bright future both for patients suffering from retinal disorders, and for companies willing to explore this unconventional treatment approach.

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