In the field of RNAi therapeutics, effective delivery continues to be the primary hurdle to overcome. Minicircles, developed by Mark A. Kay, M.D., Ph.D., Professor of Pediatrics and Genetics at Stanford University and Strategic Consultant to Benitec, provides a significant advance over current RNAi non-viral systems and expands the repertoire of non-viral delivery methods available to researchers undertaking therapeutic programs.

With this new technology, we have demonstrated therapeutic levels of transgene product in animal models for up to 10 months, with no toxicity from this non-integrating vector stated Dr. Kay. We designed a unique plasmid to allow for easy purification and large-scale production of Minicircles, making this a feasible vector for clinical use.

Minicircles result in gene expression up to 500 times greater than that seen with traditional plasmid DNA (Chen, et al. Mol Ther 2001; 3:403-410) and were created from the discovery in the Kay laboratory that bacterial sequences present in normal plasmid DNA result in significant down-regulation of gene expression in animal models.

Benitec retains exclusive, sub-licensable rights to all RNAi therapeutic uses of Minicircles. The commercial advancement of this innovative technology builds on the relationship we established with Stanford through the license of Dr. Kay ™s RNAi patent applications. We consider our continuing involvement with Stanford and the Kay laboratory to be a tremendous competitive advantage said Sara Cunningham, COO of Benitec, Inc.

This license advances Benitec ™s ability to build effective RNAi therapeutics for our in-house disease programs and supports our pathfinder approach to the overall development of RNAi therapeutics. We are very pleased at the strengthening of our ties with Stanford. Having ourselves pioneered the development of ddRNAi, we recognise the need for innovation in this exciting and fast-developing field. We will therefore continue to identify in-licensing opportunities for Benitec in delivery methods and clinical targets from other leading centers of innovation in both the United States and abroad, stated John McKinley, Executive Chairman and CEO of Benitec.

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