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Cambridge, UK, 05 July 2017: Horizon Discovery Group plc (LSE: HZD) ("Horizon" or “the Company”), the world leader in the application of gene editing technologies, today announces the completion of licensing agreements with all joint patent holders to provide exclusive worldwide rights to a novel transposon-based gene editing technology platform.
The license broadens Horizon’s gene editing capabilities, with immediate applications in biomanufacturing, diagnostics and cell therapy. This release follows an announcement on 22 May 2017 describing Horizon gaining initial rights to this technology.
The gene editing technology is based on a type of transposon (Helitrons), which uses a ‘copy and paste’ mechanism to allow multiple copies of a DNA sequence to be incorporated into a genome, either immediately, or by reactivating the transposon machinery to increase the copy number at a later time. This is in contrast to current transposon technologies (e.g Sleeping Beauty or PiggyBac), which transfer DNA by a ‘cut and paste’ mechanism, where a sequence is removed from one part of a genome and randomly inserted elsewhere.
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 realize significant operational efficiencies in cell engineering, and cell and gene therapy.
The Helitron patent is held by four inventors: Zoltan Ivics (Max Delbrück Center for Molecular Medicine (MDC) and the Paul Ehrlich Institute), Ivana Grabundzija (MDC), Vladimir Kapitonov (Genetic Information Research Institute), and Tilmann Buerckstuemmer (Global Head of Innovation, Horizon Discovery). Horizon has now been granted an exclusive license from all co-owners for use of the technology, providing freedom to operate for all applications including tool development, service provision, and therapeutic development.
Applications of this technology for Horizon include:
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: “At Horizon we understand that the improvement of existing gene editing technologies, as well as development of completely novel ones, is critical to our lasting success leading the way in the gene editing space. Through the completion of gaining exclusive rights from all parties, Horizon now has access to a powerful new gene editing platform that provides a unique approach to gene editing and DNA delivery, strengthening our capabilities in bioproduction and diagnostics, and for the first time directly as a cell or 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
The Max Delbrück Center for Molecular Medicine (MDC)
The Max Delbrück Center for Molecular Medicine (MDC) in the Helmholtz Association was founded in Berlin in 1992. It is named after the German-American biophysicist Max Delbrück who was awarded the 1969 Nobel Prize in Physiology and Medicine. The MDC’s mission is to study molecular mechanisms in order to diagnose, prevent and fight disease more effectively. Currently, more than 1,600 scientists, staff and guests from 60 countries are working at the MDC. www.mdc-berlin.de/en
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”.
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