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Horizon Discovery, Rutgers Collaborate to Commercialize CRISPR Base Editing Technology

NEW YORK (GenomeWeb) – Gene editing firm Horizon Discovery and Rutgers University announced today that they have signed a partnership deal to commercialize a gene editing technology developed in the lab of Rutgers Robert Wood Johnson Medical School pharmacology associate professor Shengkan Jin.

The base editing platform — which modifies genes by creating single point mutations in DNA without making double-stranded breaks — has potential applications in the development of new cell therapies and will augment Horizon's research tools and services, the company said.

Horizon plans to collaborate with Rutgers to further develop the technology, and work toward commercializing it. 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's own researchers will also conduct evaluation and proof of concept studies while the firm funds further research in base editing at Rutgers.

"Base editing is potentially transformative for all gene editing technologies with the potential to help target many diseases that to date have no treatment. As a world leader in the field of gene editing and gene modulation, both in research and applied markets, we are very excited to partner with Dr. Jin and Rutgers University," Horizon CEO Terry Pizzie said in a statement.

"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," Pizzie added. "As part of our five-year investment strategy, Horizon committed to investing in high-value technologies that maintain our market leadership; base editing technology is a perfect example of that."

Jin also noted that the cytidine deaminase version of the technology could be useful in creating ex vivo therapeutics for sickle cell anemia and beta thalassemia, HIV-resistant cells, and CAR-T cells for leukemia, as well as in vivo therapeutics for inherited genetic diseases.