Exercises Option to Exclusively License Base Editing Technology from Rutgers, The State University of New Jersey

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In January 2019, Horizon and Rutgers signed an Option Agreement and a Research Agreement, which granted Horizon an option to obtain an exclusive license to Rutgers’ proprietary Base Editing technology for therapeutic, diagnostic and service applications. Under the terms of the agreement, Horizon and Rutgers agreed to collaborate to further develop the Base Editing technology, which included the Group undertaking its own evaluation and proof-of-concept studies. Horizon has several internal programs designed to accelerate the clinical uptake of this technology and make it widely available for therapeutic applications. 

Horizon has now exercised its option and Rutgers has granted it an exclusive license to Rutgers’ proprietary 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 are confidential and have not been disclosed.

Base Editing is a novel technology that enables scientists to make edits to base pairs in DNA by recruiting specific Base Editing enzymes (deaminases).

Horizon is now seeking early access customers to assess and shape the development of this Base Editing platform, which could enable the development of novel therapeutics that rely on engineering cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy).  This novel technology could also be applied in cell therapies programs that require more effective multi-gene knockouts (such as CAR-T cells) with an improved safety profile. Customers will also gain access to Horizon’s expertise in genome engineering of different cell types, influence over the direction of future development and access to early technical data from the platform.

For more scientific detail on Base Editing, please see below in the notes to editors.

Base Editing is a novel technology platform for engineering DNA or genes in cells, which allows scientists to make specific edits to base pairs in DNA or RNA by using an enzyme to change a single base of DNA into another. 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, this new technology allows for more accurate gene editing while reducing unintended genomic changes.

Many human diseases have a simple and known cause, i.e. a single genetic alteration caused through heredity genetics or by an infectious agent.    Examples include genetic diseases such as sickle cell anaemia and Duchenne muscular dystrophy, as well as infectious diseases such as AIDS and hepatitis B.  Despite the clear and simple cause, a cure is understandably difficult because the approved drugs, as well as most drugs under development, target a disease-associated target or protein rather than impacting the disease-causing gene itself. This is largely due to a deficiency in understanding of the etiological cause of disease as well as the lack of effective DNA-targeting technology.

CRISPR gene editing technology allows targeted recognition and modification of specific disease-causing DNA sequences in the genes of cells. However, first generation CRISPR technology generates double-strand DNA breaks and often requires disease tissues or cells to have homologous dependent repair activity to achieve optimum therapeutic effect. As double strand DNA breaks are oncogenic in nature and homologous dependent repair activity is by and large absent in diseased tissues, the first generation CRISPR technology arguably has major hurdles to overcome for developing therapeutic vehicles or agents; in particular for developing in vivo therapeutics since it could generate potentially oncogenic DNA breaks and usually requires homology-dependent repair activity which is absent in most disease- affected organs. 

Base Editing has the potential to overcome the issues above by utilizing a nuclease-deficient CRISPR protein and an RNA-based recruitment mechanism to guide a non-nuclease DNA modifying enzyme, such as a cytidine deaminase, to the disease-causing gene, where the enzyme effectively corrects or modifies the gene in the disease tissues while minimizing the generation of the potential oncogenic DNA breaks. This allows more accurate editing of genes with reduced negative effects due to unintentional genomic changes. 

Horizon Discovery Group plc (LSE: HZD) ("Horizon") drives the application of gene editing and gene modulation within the global life science market – supporting scientists on the path from research to therapy.

Built upon more than a decade of experience in the engineering of cell lines, Horizon offers an unmatched portfolio of tools and services to help scientists gain a greater understanding of gene function, identify genetic drivers behind human disease, deliver biotherapeutics, cellular and gene therapies for precision medicine as well as develop and validate diagnostic workflows.

Horizon’s solutions enable almost any gene to be altered, or its function modulated, in human and other mammalian cell lines.

The Group’s customers include many of the world’s foremost academic institutes, global pharmaceutical and biotechnology companies as well as clinical diagnostic laboratories. Insight into the challenges faced by these organizations enables Horizon to focus efforts on development of innovative solutions that not only differentiate the Group’s offering, but also fuel development of the next wave of precision medicines.

Horizon is headquartered in Cambridge, UK with offices in USA and Japan.  The Group is listed on the London Stock Exchange's AIM market under the ticker HZD.

Rutgers, The State University of New Jersey, is a leading national research university and the state of New Jersey's preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth oldest higher education institution in the United States. More than 69,000 students and 22,500 full- and part-time faculty and staff learn, work, and serve the public at Rutgers locations across New Jersey and around the world.

More news and information about Horizon Discovery Group plc

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Globe Newswire: 12:00 GMT Tuesday 14th January 2020

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