The licence 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 licence 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:
- Biomanufacturing: supports delivery of the DNA sequence of the protein to be manufactured into bioproduction cell lines (e.g. CHO), 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 therapy (1), and the market has been estimated to be worth up to £14 billion by 2025 (2).
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.”
Glossary:
- 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
About transposons
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
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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
Zyme Communications (Trade and Regional Media)
Katie Odgaard
Tel: +44 (0)7787 502 947
Email: katie.odgaard@zymecommunications.com
Consilium Strategic Communications (Financial Media and Investor Relations)
Mary-Jane Elliott / Susan Stuart / Matthew Neal / Melissa Gardiner
Tel: +44 (0) 20 3709 5701
Email: horizon@consilium-comms.com
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