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GenSight, a French biotechnology company, has announced the receipt of FDA orphan designation for retinitis pigmentosa gene therapy treatment in the US.

GenSight Biologics S.A. (Euronext: SIGHT) has announced the receipt of FDA Orphan Drug Designation in the US for its experimental retinitis pigmentosa (RP) treatment, “GS030”. The announcement follows a similar orhan designation grant in the EU, announced in Q4 of 2016. According to the company, GS030, is an optogenetic therapy delivered using adeno-associated viral (AAV) […]

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Phase IIa trial recruits first patient for novel therapy directed towards treatment of Stargardt’s disease.

A Phase IIa human study, sponsored by Acucela Inc., based in Seattle, Washington, has announced the recruitment of its first patient to evaluate emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. The experimental small molecule treatment, a visual cycle modulator, will be tested in a multicenter, randomized, blinded study to investigate the […]

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Potential biomarker for diabetic retinopathy uncovered through genome-wide mRNA analysis

Researchers based at the Institute of Ophthalmology, Conde de Valenciana, Mexico City, have published the results of a genome wide mRNA screen which suggest a potential link between alternative splice forms of the TUBD1 gene, and a risk for developing diabetic retinopathy (DR). While the study was performed on a relatively small number of diabetic […]

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