Horizon Discovery Group has forged a new alliance with Rutgers University to develop the next generation of gene editing technology for therapeutic use.
The gene editing specialist has exclusively licensed the University’s base editing technology, which could boost the development of new cell therapies.
Base editing builds on the highly adaptable CRISPR approach, which is fast becoming a standard tool in many labs due to its precision and low costs, although it produces many challenges.
First generation CRISPR technology creates a double stranded cut in DNA breaks (which are naturally tumour prone) and often requires cells to have homologous dependent repair activity to achieve therapeutic effect.
However, homologous dependent repair activity is by and large absent in diseased tissues, which presents a barrier when using CRISPR as a therapeutic tool.
Unlike CRISPR however, base editing utilises a nuclease-deficient CRISPR protein and an RNA-based recruitment mechanism to guide a non-nuclease DNA modifying enzyme to the disease-causing gene.
The enzyme can then effectively correct or modify the gene in the disease tissue while minimising the potential of oncogenic DNA breaks, allowing for an accurate gene-editing procedure while reducing negative effects due to unintentional genomic alterations.
“Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment,” said Terry Pizzie (pictured below), Horizon’s chief executive officer.
He adds: “We are very excited to partner with Rutgers University. 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.”
The new partnership builds on Horizon’s five-year strategy, which focuses on investing in high value technologies and according to Pizzie, base editing is “a perfect example of that”.
Dr. David Kimball, Interim Senior Vice President for Research and Economic Development at Rutgers University, added: “Gene editing technology has truly revolutionised how scientists think about their search for better options and outcomes in the treatment of disease. We look forward to advancing the shared goals of further developing this novel base editing platform and improving human health through this collaboration with Horizon.”
As part of the agreement, Horizon has made a non-material payment to Rutgers to exclusively license the tech for intention to use in all therapeutic applications.
Horizon has also agreed to fund further research in base editing at the University.