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Algal genes to be used in new therapy

31/01/2018By Matthew Woodley • Staff Journalist
Genes from light-seeking algae will be inserted into the eyes of blind patients as part of human trials for a new type of optogenetics treatment.

French biopharma company GenSight Biologics received approval from the UK Medicines and Healthcare Regulation Agency last month to start its Pioneer Phase I/II trials for the radical new gene therapy GS030. The trial will specifically target patients with end-stage non-syndromic retinitis pigmentosa with vision not better than “counting fingers”.


“For the first time ever in ophthalmology, an optogenetic approach combined with a medical device will be tested in humans. If proven safe and effective, this therapy could be transferable from retinitis pigmentosa to dry-AMD.”
Dr Bernard Gilly, GenSight co-founder

GenSight co-founder Dr Bernard Gilly said approval for the trial represented a major milestone for the company.

“I am particularly excited to have GS030 moving into the clinic,” he said.

“For the first time ever in ophthalmology, an optogenetic approach combined with a medical device will be tested in humans. If proven safe and effective, this therapy could be transferable from retinitis pigmentosa to dry-AMD.”

The trial will analyse the safety and tolerability of the therapy at one year post-injection and is expected to begin in the first quarter of 2018.

Three cohorts of three subjects each will be administered an increasing dose of the therapy via a single intravitreal injection in their worse affected eye, while a fourth extension cohort will receive the highest tolerated dose. In addition to the injections, patients will also wear an optronic visual stimulation device.

The aim of the treatment is to convert nerve cells into light sensitive cells – a technique that has been used before on animals, but whose effects on humans are unknown.

According to New Scientist, optogenetics is usually used on animals to illuminate certain areas of the brain in an effort to document brain behaviour and better understand how it works. However, this trial will aim to boost the detection of red light in the human eye.

Should it prove successful, it is hoped patients will then be able to use special goggles to improve their overall vision.

Image courtesy: Flickr | Amy Anderson

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