Key information

🔎 Fund Overview

Benitec Biopharma Inc is a clinical-stage biotechnology company. The Company is focused on the advancement of novel genetic medicines. Its proprietary Silence and Replace deoxyribonucleic acid (DNA) -directed ribonucleic acid (RNA) interference platform combines RNA interference, or RNAi, with gene therapy to create medicines that simultaneously facilitate sustained silencing of disease-causing genes and concomitant delivery of wildtype replacement genes following a single administration of the therapeutic construct. The Company is developing Silence and Replace-based therapeutics (BB-301) for chronic and life-threatening human conditions including Oculopharyngeal Muscular Dystrophy (OPMD). BB-301 is an adeno-associated viral vector (AAV)-based gene therapy designed to silence the expression of disease-causing genes (to slow, or halt, the underlying mechanism of disease progression) and to simultaneously replace the mutant genes with normal, wildtype genes.