Date: 2017-07-05

Type of information: Licensing agreement

Compound: novel transposon-based gene editing technology platform

Company: Horizon Discovery (UK)

Therapeutic area: Technology - Services

Type agreement: licensing

Action mechanism:

  • gene editing 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 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. 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.  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 develop reference standards for assays designed to measure the copy number of a gene
  • 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.



  • • On July 5, 2017, Horizon Discovery, a leader in the application of gene editing technologies, announced 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.
  • 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.
  • • On May 22, 2017, Horizon Discovery announced that it has gained exclusive worldwide rights to use a novel transposon-based technology platform that will broaden Horizon’s gene editing capabilities. The technology is based on Helitrons, a type of transposon that provides two unique benefits over alternative gene editing approaches. 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.

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