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> Technology provides a unique approach to gene editing and DNA delivery
> Enables Horizon to use gene editing directly as a therapeutic
> Immediate applications in biomanufacturing, diagnostics, cell and gene therapy
Horizon Discovery Group plc (LSE: HZD) ("Horizon" or “the Company”), the world leader in the application of gene editing technologies, today announces that it has gained exclusive worldwide rights to use a novel transposon-based technology platform that will broaden Horizon’s gene editing capabilities. This new gene editing technology, co-invented by Horizon’s Head of Innovation Dr. Tilmann Buerckstuemmer together with three other European Scientists, has been acquired on terms that include a small upfront payment with additional downstream royalty payments to the third party co-inventors against future product sales and sub-license deals.
Access to this technology provides a unique approach to the modification of genes and will allow Horizon to operate across all applications including product development, service provision, and therapeutic development. The technology is based on Helitrons, a type of transposon that provides two unique benefits over alternative gene editing approaches. Current transposon technologies transfer DNA by using a ‘cut and paste’ mechanism, whereas the Helitron-based technology covered by this patent instead uses a ‘copy and paste’ mechanism that allows multiple copies of a DNA sequence to be incorporated into a genome.
This technology also enables the highly efficient delivery of DNA into a genome. A common method for DNA delivery is through the use of retroviruses or lentiviruses, which can be challenging to manufacture and are covered by associated IP that require expensive licenses. The Helitron IP provides a far simpler and less expensive approach to manufacturing, promising Horizon the opportunity to realise significant operational efficiencies in cell engineering, and cell and gene therapy.
Applications of this technology for Horizon include:
> Biomanufacturing: supports delivery of the DNA sequence of the protein to be manufactured into bioproduction cell lines, and holds the potential to enhance the productivity of cells by having multiple copies of the target sequence present.
> Diagnostics: to be used to efficiently develop reference standards for assays designed to measure the copy number of a gene (e.g. Her2 in breast cancer).
> Therapeutics: provides an alternative mechanism to deliver a gene of interest into a genome, and opens up a potentially revolutionary approach to therapeutic development where multiple copies of an expressing gene can drive the effectiveness of therapy.
Cell and gene therapy represents a particularly large opportunity. In 2015, public and private companies raised almost £8 billion to support development programmes in cell and gene therapy1, and the market has been estimated to be worth up to £14 billion by 20252.
Dr. Darrin M Disley, Chief Executive Officer, Horizon Discovery Group, commented: “Horizon’s access to the best gene editing technologies, and our deep expertise in their application, has been a cornerstone in our becoming the ‘Cell Builders’. Through this license, we now have access to a powerful new technology that promises to enable the development of new products in high value areas such as biomanufacturing and diagnostics, to drive operational efficiencies, and also has the potential to be used directly as a therapeutic in the area of cell and gene therapy.”
Transposons, also called ‘jumping genes’ are discrete pieces of DNA that are able to move from one site to another within one genome and function both as a driver of evolution by contributing to the formation of new genes, and serve to support certain regulatory functions in cells. Given their relatively simple design and inherent ability to move DNA, transposons have been widely adopted as genetic tools, primarily to help remove and integrate genetic sequences. The most commonly used transposon technologies for gene editing are Sleeping Beauty and PiggyBac, both of which use a cut/paste mechanism, where a sequence is removed from where it sits and is then inserted randomly elsewhere in the genome. Poseida Therapeutics raised up to $30 million in December 2016 to exploit PiggyBac to develop novel CAR-T cell therapy and gene therapy approaches for amongst others Jannssen Pharmaceuticals.
2. Cell and Gene Therapy market data, ABPI, PWC October 2016