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Rutgers Licenses Base Editing Tech to Horizon Discovery

NEW YORK – Horizon Discovery announced on Tuesday that it has exercised its option to exclusively license a novel base editing technology from Rutgers University for use in therapeutic, diagnostic, and service applications.

The company and the university signed an option and research agreement in January 2019 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.

Horizon planned to collaborate with Rutgers to further develop the technology and work toward commercializing it. As part of the agreement, Horizon's own researchers also planned to conduct evaluation and proof-of-concept studies while the firm funded further research in base editing at Rutgers.

Now, Horizon said it has exercised its option and Rutgers has granted it an exclusive license to the base editing technology. The license includes the right for Horizon to sublicense the technology to entities seeking to use it for therapeutic development. Financial terms of the deal have not been disclosed.

"Exercising our option for this technology is potentially transformative for Horizon," Horizon CEO Terry Pizzie said in a statement. "Base editing has the potential to revolutionize the treatment of genetic diseases. Over the past year, we completed a detailed evaluation phase of Rutgers' technology and are excited to now have an exclusive license for use in therapeutic, diagnostic and service applications."

In a separate announcement on Tuesday, Horizon said it will provide access to the base editing technology to business partners for the development of more effective multi-gene knockout cell therapy programs.

The company is seeking early-access customers to assess the technology and lead its development, which could enable the development of therapeutics that rely on engineering cells either directly in the body or externally before transplanting back into the patient. The technology could also be used in cell therapy programs that require more effective multi-gene knockouts such as CAR-T cells with an improved safety profile, the company said.

Horizon has various internal programs that are meant to accelerate the clinical uptake of the base editing technology, and the firm is also seeking three to five partners to assess the development of the base editing platform.

"The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialization," Jonathan Frampton, Horizon's corporate development partner, said in a statement. "Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organizations will help us to drive innovation and deliver the best therapy for the patient."