Gene therapy enters the eye
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Gene therapy enters the eye

09.04.2013 - A new French start-up secured a seed financing of €32m. Based on an optogenetics/gene therapy approach, it aims to cure eye diseases.

The field of gene therapies is experiencing a serious tailwind these days. On 8 April, GenSight Biologics announced the closure of a €32m Series A financing. The Parisian biopharmaceutical company works on the development of ophthalmic therapeutics – using gene therapy. The financing was co-led by Novartis Venture Fund, Abingworth LLP, Versant Ventures and Index Ventures. GenSight will use the funds to develop a gene replacement therapy for Leber's hereditary optic neuropathy (LHON) and an optogenetic therapy for retinitis pigmentosa (RP).

The lead product is expected to enter the clinic in 2013 in LHON patients. The co-founder and CEO of GenSight, Bernard Gilly, explains why ophthalmology is one of the most best indications for gene therapies: "It is promising because of the safety and efficacy demonstrated in certain trials in this field." Other advantages for GenSight are its "unique, proprietary approach to targeting the mitochondria in LHON developed at the Vision Institute in Paris", and  – Gilly adds – "the exclusive access to key intellectual property from Novartis for using optogenetics to treat RP patients."

The new therapy would enter an anbandoned field as the MAA of Santhera’s synthetic coenzyme Q10 derivative Raxone had to be withdrawn due to EMA concerns only in early 2013.



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