Benitec Biopharma

Gene silencing with DNA-directed RNA interference (ddRNAi)

Benitec Biopharma is Australia's only listed gene therapy company, pioneering DNA-directed RNA interference (ddRNAi) — a technology that combines gene therapy with RNA interference for permanent gene silencing from a single treatment. Their lead program BB-301 targets oculopharyngeal muscular dystrophy (OPMD), a rare genetic disease with no approved treatments. Unlike traditional RNAi therapies that require repeated dosing, Benitec's ddRNAi approach delivers a DNA template via AAV that continuously produces silencing RNA molecules, offering a potential one-time cure.