Horizon Discovery Group (HZD) has entered into an exclusive partnership with Rutgers university to develop and commercialise a new gene editing technology, known as base editing.
Base editing is a new technology platform for engineering DNA or genes in cells, with the potential to correct errors or mutations in the DNA by modifying genes with an enzyme.
Terry Pizzie, Horizon’s Chief Executive Officer, 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.”
Horizon is to collaborate with Rutgers to develop the new base editing platform from the laboratory of Dr. Shengkan Jin, associate professor of pharmacology at Rutgers Robert Wood Johnson Medical School.
Additionally, Horizon made a non-material payment to Rutgers for the exclusive license of the base editing technology in all therapeutic applications. It will also fund further research in base editing at Rutgers while undertaking evaluation and proof of concept studies.
Mr. Pizzie mentioned he is excited to partner with Rutgers because it is a world leader in the field of gene editing and gene modulation, both in research and applied markets. He said that Horizon will now be able to more fully support its pharma, biotech and academic partners to deliver better cell therapy solutions to patients.
Photo by Cambridge Independent
Dr. Shengkan ‘Victor’ Jin of Rutgers University said: “The potential is enormous”
“The cytidine deaminase version of the technology alone could potentially be used for developing ex vivo therapeutics such as gene modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, and over-the-shelf CAR-T cells for leukemia, as well as in vivo therapeutics for inherited genetic diseases.”
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.”
Compared with currently available gene editing methodologies such as CRISPR/Cas9, which creates “cuts” in the gene that can lead to adverse or negative effects, the new technology allows for more accurate gene editing while reducing unintended genomic changes, the company said.
The technology will have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialisation.
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