Horizon Discovery gains exclusive rights to 'novel' gene editing tech

The AIM-traded firm said the new gene editing technology, co-invented by Horizon's head of innovation Dr Tilmann Buerckstuemmer together with three other European scientists, had been acquired on terms that included 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 the technology provided a unique approach to the modification of genes and would allow Horizon to operate across all applications including product development, service provision, and therapeutic development, the company’s board explained.

It said the technology was based on Helitrons, a type of transposon that provided two unique benefits over alternative gene editing approaches.

Current transposon technologies transferred DNA by using a 'cut and paste' mechanism, whereas the Helitron-based technology covered by the patent instead used a 'copy and paste' mechanism that allowed multiple copies of a DNA sequence to be incorporated into a genome.

The technology also enabled the “highly efficient delivery” of DNA into a genome, the board said.

A common method for DNA delivery was through the use of retroviruses or lentiviruses, which could be challenging to manufacture and was covered by associated IP that require expensive licenses, Horizon said.

The Helitron IP provided a far simpler and less expensive approach to manufacturing, the board said, promising Horizon the opportunity to realise “significant operational efficiencies” in cell engineering, and cell and gene therapy.

“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',” said CEO Darrin M Disley.

“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 Disley added.