22 May 2017
Horizon Discovery Announces New Gene Editing Technology Platform for Research and Therapeutic Applications
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
Cambridge, UK, 22 May 2017: 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.
“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.”
Dr. Darrin M DisleyChief Executive Officer, Horizon Discovery Group
- Cell engineering: The development of cell lines that reflect the genetics of disease found in real patients by use of gene editing
- Biomanufacturing: A type of manufacturing that utilises biological systems to produce commercially important biomaterials and biomolecules for use in medicines
- Cell therapy: the administration of live whole cells (potentially gene edited) to a patient for the treatment of a disease
- Gene therapy: the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders
- Genome: an organism's complete set of DNA, including all of its genes
- Helitron: a type of transposable element found in eukaryotes
- Reference standard: a standardised substance (e.g. DNA, protein) which is used as a measurement base or control for an assay
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.
- Cell and Gene Therapy market data, ABPI, PWC October 2016
For further information from Horizon Discovery Group plc, please contact:
Horizon Discovery Group plc
Darrin Disley, Chief Executive Officer
Richard Vellacott, Chief Financial Officer
Chris Claxton, VP Investor Relations
Tel: +44 (0) 1223 655 580
Numis Securities Limited (Broker and NOMAD)
Michael Meade / Freddie Barnfield
Tel: +44 (0) 207 260 1000
Consilium Strategic Communications (Financial Media and Investor Relations)
Mary-Jane Elliott / Susan Stuart / Matthew Neal / Melissa Gardiner
Tel: +44 (0) 20 3709 5701
Zyme Communications (Trade and Regional Media)
Tel: +44 (0)7787 502 947
About Horizon Discovery Group plc www.horizondiscovery.com
Horizon Discovery Group plc (LSE: HZD) ("Horizon"), is a world-leading gene editing company that designs and engineers genetically-modified cells and then applies them in research and clinical applications that advance human health.
Horizon’s core capabilities are built around its proprietary translational genomics platform, a highly precise and flexible suite of gene editing tools (rAAV, ZFN, CRISPR and Transposon) able to alter almost any gene sequence in human or mammalian cell-lines.
Horizon offers over 23,000 catalogue products and related research services, almost all of which are based on the generation and application of cell and animal models that accurately recapitulate the disease-causing genetic anomalies found in diseases like cancer. Horizon’s commercial offering has been adopted by over 1,600 unique research organisations in over 50 countries as well as in the Company’s own R&D pipeline to support a greater understanding of the genetic drivers of disease and the development of molecular, cell and gene therapies that can be prescribed on a personalised basis.
Horizon is headquartered in Cambridge, UK, and is listed on the London Stock Exchange’s AIM market under the ticker “HZD”.