Benitec Biopharma

Antibiotics and Infectious diseasesGeneticsImmunologicalsNeurologicalOncologyAustralia

Benitec Biopharma

Australia-based Benitec Biopharma (ASX: BLT) specializes in expressed RNA interference (RNAi) technology.

Australia-based Benitec Biopharma (ASX: BLT) specializes in expressed RNA interference (RNAi) technology.

Benitec’'s patented gene silencing technology, ddRNAi, has the potential to deliver cure as well as treatment of chronic and life-threatening diseases, due to its long lasting effects. Its unique platform, ddRNAi, has distinct advantages over other methods of gene silencing such as siRNA, anti-sense and zinc finger nucleases.

Benitec's patents cover the use of RNAi resulting from the introduction of a DNA construct to the target cell nucleus in vivo to effect long-lasting genetic silencing. The synthetic RNA interference approach based on siRNA (small interfering RNA) introduces externally-manufactured dsRNA silencing molecules into a target cell, where they enter a natural RNAi pathway to silence a particular gene. Because dsRNA is fragile, unless modified, it is difficult to deliver to target cells in target organs without the risk of degradation. Even if successful delivery of intact RNA molecules is achieved, the treatment effect will be transient, lasting hours or maybe days. This means that, for siRNA to be effective, relatively high and repeated doses are needed. These difficulties may explain why several programs based on siRNA have been discontinued in recent years.

Due to its demonstrated ability to effect long-lasting gene silencing, once proven in human trials, ddRNAi technology has significant potential to treat or cure a broad range of human diseases and conditions. These include cancers, neurological diseases, infectious diseases, autoimmune diseases, genetic diseases and other afflictions that are associated with the expression of a small number of key genes. The company has pipeline drugs for HIV/AIDS, hepatitis C and B, drug resistant lung cancer, cancer-associated chronic pain, muscular dystrophy, age-related macular degeneration, retinitis pigmentosa and Huntington's Disease.


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