UK-based Horizon Discovery Group has collaborated with Rutgers University in the US to develop and commercialise a new gene editing technology, called base editing.
The technology has the potential for use in the development of new cell therapies.
Base editing is designed to engineer DNA or genes in cells, enabling the correction of errors or mutations in the DNA.
The technology guides a non-nuclease DNA modifying enzyme to the disease-causing gene by leveraging a nuclease-deficient CRISPR protein and an RNA-based recruitment mechanism.
Upon reaching the target site, the enzyme is said to correct or modify the gene while reducing the risk of oncogenic DNA breaks.
The company claims that the new technology can be used for more accurate gene editing and leads to fewer unintentional genomic changes, compared to existing approaches such as CRISPR/Cas9.
Horizon Discovery CEO Terry Pizzie said: “Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment.
“By extending our scientific and IP capabilities, Horizon will now be able to more fully support our pharma, biotech and academic partners to deliver better cell therapy solutions to patients.”
As part of the agreement, Horizon Discovery made a non-material payment to Rutgers to gain the option for exclusive licensing of the base editing technology in all therapeutic applications.
In addition, the company will fund additional base editing research at Rutgers University, and carry out in-house assessment and proof of concept studies.
Dr Shengkan Jin from the university added that the cytidine deaminase version of the new technology can be used to develop ex-vivo therapeutics, such as HIV resistant cells for AIDS.
The technology has the potential for therapeutic application in diseases caused by a single genetic alteration as well as those where permanent targeting of a disease-related gene could be beneficial.