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Genable Receives FDA Orphan Drug Status for Ocular Disease Drug

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Genable Technologies, an Irish biopharmaceutical firm, announced last week that it has received orphan drug status from the US Food and Drug Administration for its expressed RNAi-based treatment for retinitis pigmentosa.

The disease is rare degenerative eye condition that can lead to blindness. Last summer, the company took a license to Benitec Biopharma’s expressed RNAi technology for use in developing a treatment for the condition (GSN 7/19/2012).

Genable’s drug, dubbed GT038, comprises an AAV vector to deliver an shRNA that silences both the mutant and wild-type rhodopsin gene, which is mutated in retinitis pigmentosa. The company, which previously received orphan drug status for the agent from European regulators, anticipates a phase I trial of GT038 could begin in 2014.

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