Retinitis Pigmentosa Case
Retinitis pigmentosa is a rare genetic disorder that causes trouble with seeing at night and reduced peripheral vision. It is a slow, progressive disorder of the eye’s retina. In France, a company developed a gene therapy that may cure the disease.
The French company, GenSight Biologics, used a technique called optogenetics. The technique allows scientists to control living cells, in this case, neurons, using light to cure this form of blindness. The optogenetics technique is originally acquired from single-celled algae to help the organism detect light. The gene therapy’s Clinical Trial Application has been accepted by the UK Medicines and Healthcare Regulatory Agency. It’s first-in-man, multi-center, open label dose-escalation study is named PIONEER for phase I and II of GS030 patients. The patients will be administered with a single intravitreal injection and given a wearable optronic visual stimulation.
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This represents a major milestone for the Company. For the first time ever in ophthalmology, an optogenetic approach combined with a medical device will be tested in humans,” said Bernard Gilly, the CEO, and co-founder of the company.
What is Retinitis Pigmentosa
Retinitis pigmentosa occurs when any of one of more than 50 genes changes or mutates. These genes are vital in keeping the cells in the retina called photoreceptors functional. However, changes or mutations in these can lead to three possible effects:
- Severe changes inhibit the gene’s production of a specific protein needed by the photoreceptors.
- Mutations can lead to the production of a protein that is toxic to the
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