Horizon Discovery, Rutgers Join Forces for Gene Editing

January 28, 2019
Pharmaceutical Technology Editors
Pharmaceutical Technology's In the Lab eNewsletter
Volume 14, Issue 2

Horizon and Rutgers University will partner to develop and commercialize new gene editing technology for therapeutic and research applications.

On Jan. 28, 2019, gene editing company Horizon Discovery Group announced that it has entered into an exclusive strategic partnership with Rutgers, The State University of New Jersey, to develop and commercialize a gene editing technology known as base editing.

Under the partnership, Horizon and Rutgers will further develop the base editing platform from the laboratory of Dr. Shengkan Jin, associate professor of pharmacology at Rutgers Robert Wood Johnson Medical School. As part of the agreement, Horizon has made a non-material payment to Rutgers for an option to exclusively license the base editing technology for use in all therapeutic applications. Horizon will also fund further research in base editing at Rutgers while undertaking evaluation and proof-of-concept studies at Horizon. According to Horizon, the technology has potential applications in the development of new cell therapies and will expand the company’s research tools and services.

Base editing is a technology platform for engineering DNA or genes in cells that has the potential to correct errors or mutations in the DNA by modifying genes using an enzyme. Horizon reports that compared to currently available gene editing methodologies such as CRISPR/Cas9-which creates ‘cuts’ in the gene that can lead to adverse or negative effects-this new technology allows for more accurate gene editing while reducing unintended genomic changes. The technology is expected to have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialization.

“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 chimeric antigen receptor [CAR]-T cells for leukemia, as well as in-vivo therapeutics for inherited genetic diseases,” Dr. Jin stated in a company press release. “The potential is enormous. In addition to the ‘simple’ diseases caused by a single genetic alteration event, the therapeutic strategy, in principle, could also be useful for treating diseases where permanently targeting a disease-related gene is beneficial.”

Source: Horizon Discovery Group