Founded in 1997 and headquartered in Melbourne, Australia, Benitec Biopharma plc is a clinical-stage biotechnology company focused on the development of gene-silencing therapies. The company specializes in DNA-directed RNA interference (ddRNAi), a platform designed to silence disease-causing genes at the mRNA level. Benitec’s approach employs engineered DNA constructs that instruct cells to produce short interfering RNAs (siRNAs), offering a potentially durable and precise treatment modality.
Benitec has established strategic collaborations with leading academic institutions and biopharmaceutical organizations to advance its ddRNAi programs. The technology is adaptable across multiple delivery platforms, notably adeno-associated virus (AAV) vectors, which facilitate targeted delivery of genetic constructs. This flexibility underpins the company’s efforts to address a range of therapeutic areas with high unmet medical need.
The company’s pipeline includes a lead candidate in chronic hepatitis B aimed at achieving sustained viral suppression through targeted gene silencing. In parallel, Benitec is exploring ddRNAi-based approaches for HTLV-1-associated diseases and fibrotic disorders such as idiopathic pulmonary fibrosis, with the goal of modulating pathological gene expression drivers. Earlier-stage research has also investigated applications in neurodegenerative conditions, reflecting the broad applicability of RNA interference strategies in both hepatology and neurology.
Benitec serves global markets with operations in Australia and the United States, supported by a network of collaborators in Europe and Asia. The company is governed by a board of directors and an experienced management team with deep expertise in gene therapy development, regulatory strategy and commercialization. Through its ddRNAi platform, Benitec Biopharma continues to advance novel therapeutic candidates toward clinical evaluation, addressing diseases with significant unmet patient needs.
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