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Genable Receives FDA Orphan Drug Status for Ocular Disease Drug


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.