베니텍 바이오파머

Benitec Biopharma Inc., a clinical-stage biotechnology company, focuses on the development of novel genetic medicines in the United States. Its Silence and Replace, a DNA-directedRNA interference platform, that combines RNA interference with gene therapy to create medicines which 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 BB-301, an AAV-based gene therapy designed to silence the expression of the disease-causing gene, which is in phase 1b/2a for the treatment of oculopharyngeal muscular dystrophy related dysphagia. Benitec Biopharma Inc. is headquartered in Hayward, California.