Why Benitec is a sound investment
Benitec is a biotechnology company developing a novel, proprietary therapeutic technology platform that combines gene silencing and gene therapy with a goal of providing sustained, long-lasting silencing of disease-causing genes from a single administration. We believe our technology has the potential to be a "one shot" cure for a wide range of diseases that are currently addressed by strict ongoing treatment regimens or that have no effective treatment or only palliative care options. We are using our technology, called DNA-directed RNA interference, or ddRNAi, to develop our pipeline of product candidates for the treatment of several chronic and life-threatening human diseases, such as hepatitis B, age-related macular degeneration, or AMD and oculopharyngeal muscular dystrophy, or OPMD.
Our objective is to become the leader in discovering, developing, clinically validating and commercializing ddRNAi-based therapeutics for a range of human diseases with high unmet clinical need or large patient populations, and to thereby provide a better life for patients with these diseases.
Novel Gene Silencing Platform
Our ddRNAi technology is designed to utilize the specificity and gene silencing effect of RNA interference while overcoming many of the limitations associated with the ongoing administration of siRNA. Our ddRNAi approach combines RNA interference with gene therapy. Unlike siRNA, our ddRNAi technology starts with a DNA construct. Gene therapy vectors, which are carrier molecules, often viruses, that deliver genetic material into the cell, are used to deliver the DNA construct to the nucleus of the targeted cells. The DNA construct then generates double-stranded short hairpin RNAs, or shRNAs, which are processed by the cell into siRNAs, which in turn silence the disease-associated genes. Advantages of our ddRNAi approach include:
- ddRNAi is designed to produce sustained, long-lasting silencing of the disease-causing gene, following a single administration, leading to the potential for "one shot" cures for a wide range of diseases, which could eliminate the requirement for patient compliance to take regular doses of medicine for long-term management of their disease;
- ddRNAi technology can target a wide range of tissues, including, but not limited to, the liver;
- ddRNAi uses the cell's own transcriptional mechanisms to produce a constant level of shRNA so that intracellular levels of siRNA do not fall below threshold levels required for disease suppression;
- the level of shRNA in the cells can potentially be fine-tuned to achieve optimal concentrations;
- off-tissue effects can be minimized;
- the DNA constructs are shielded in gene therapy vectors that are designed to avoid activating the interferon response;
- ddRNAi provides the option to both silence the defective gene and replace the defective gene with a normal version;
- ddRNAi can be designed to express multiple siRNAs in the same cell, targeting either a single gene at several different sites to minimize the risk of viral resistance, or multiple genes in distinct cellular pathways, potentially enabling treatment of complex genetic diseases such as cancer, diabetes and heart disease; and
- ddRNAi can elicit long-term response by continued expression of siRNA from a single administration, potentially preventing viral reinfection.
We are pursuing preclinical research in HBV, AMD and OPMD and plan to submit IND applications for those product candidates. We expect to complete preclinical in vivo proof-of-concept studies for our HBV product candidate in the second quarter of 2016, and for our AMD and OPMD product candidates in the fourth quarter of 2016. Based on the potential market opportunity and considerable interest from pharmaceutical companies, we plan to prioritize the HBV program as our next candidate for clinical development.
Each of our three current core indications are severe diseases with high unmet medical need or large patient populations. We believe there is a strong rationale for treating these diseases and other diseases that have well-characterized gene targets that can be silenced, thus preventing the disease-causing gene from being expressed. We also intend to develop ddRNAi applications in novel technologies including immunotherapy, such as chimeric antigen receptor T cells, or CAR T.
The adaptability of our platform also presents an opportunity for us to selectively form collaborations to expand our capabilities and product offerings into a range of diseases and potentially to accelerate the development and commercialization of ddRNAi therapeutics more broadly. We will continue to expand our franchise of ddRNAi-based therapeutics by out-licensing, where appropriate, applications of ddRNAi for the development of a range of therapeutics outside of our immediate focus. Once clinical proof of concept has been achieved for a product candidate, we plan, where appropriate, to enter into collaborations with pharmaceutical companies to develop that product candidate and ultimately commercialize it if it receives regulatory approval.
Strong IP Position
We have rights to intellectual property that includes a patent portfolio protecting our ddRNAi technology platform in numerous jurisdictions through 2019, and a growing portfolio of patents protecting improvements to our ddRNAi technology and product candidates in numerous jurisdictions through at least 2025. In addition to progressing our pipeline of product candidates, we will further develop and improve our ddRNAi platform technology and its associated intellectual property through in-house development and in-licensing of complementary technologies.