Sydney-based Benitec Biopharma (ASX: BLT) has announced a new exploratory cellular therapy program including exosome-based delivery utilising the company’s proprietary ddRNAi technology.
It has also announced plans for a US listing, on the NASDAQ Global Market Select, through which it plans to raise US$60-70 million. The company said it will use the capital raised to advance its therapeutic programs.
Benitec holds global patents for expressed RNA interference (also called DNA-directed RNAi – ddRNAi), positioning the company in the development of therapies associated with specific genes and genetic disorders.
The new cellular therapy program will be done in collaboration with UK-based stem cell therapeutics company, ReNeuron.
According to the company, the program aims to utilise Benitec’s ddRNAi technology to address serious diseases using derived exosomes as delivery agents.
"This announcement follows promising initial data that has been generated in work performed at both companies indicating the feasibility of this approach," it said. "Benitec and ReNeuron have completed a series of in vitro experiments that demonstrate that stem cells transduced with ddRNAi-expression constructs produce exosomes that are effective at delivering expressed shRNAs to target (cancer) cells and to knock down a specific gene in those cells. The data also indicated that ReNeuron’s stem cells could be more effective than mesenchymal stem cells for this purpose."
In a statement, Benitec said that, while the results are preliminary, they could open the potential for both companies’ platform technologies to be extended into a wide range of new therapeutic applications.
“We are delighted to be extending our research collaboration with ReNeuron," said Benitec’ CEO Dr Peter French.
"The application of ddRNAi to stem cells and derived exosomes represents a unique combination of stem cell biology, gene therapy and gene silencing. It opens up a wide array of possibilities for extending the utility of core technologies from both companies for application into new therapeutic areas.
"Having ReNeuron as a collaborator on this approach increases the chances of success as their knowledge of stem cell science coupled with their unique therapeutic platforms are tremendous assets in this area.”
ReNeuron’s Chief Executive Officer, Mr Olav Hellebø, said, “It is exciting to be working with Benitec to explore the potential of our technology as a delivery system for Benitec’s unique gene silencing technology targeting drug resistant cancers. Benitec’s extensive knowledge of expressed RNA interference is invaluable in this novel approach. Whilst the work is at an early stage we are committed to developing the program in conjunction with Benitec to determine the optimal design and use of therapeutics deploying this promising and highly novel technology combination.”
Benitec also confirmed the appointment of Mr Bob Atwill, Senior Vice President Cellular Therapies, to progress this and other potential opportunities in cellular and immunotherapies, such as ddRNAi-modified CAR-T cells.
Mr Atwill has extensive experience in the stem cell industry and was the former CEO of the Regenerative Medicine Division of Allied Healthcare Group, now Admedus.