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Horizon Discovery to Provide Access to Novel Base Editing Technology

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Horizon Discovery, a company specializing in the application of gene editing and gene modulation technologies, has announced that it will provide access to a novel base editing technology licensed from Rutgers, The State University of New Jersey, for exclusive use in therapeutic, diagnostic and services applications. This technology is incorporated into Horizon’s next-generation gene editing platform to enable the development of novel therapeutics that rely on engineering patient’s cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy). This platform will also expand the Company’s research tools and service provisions.

The Company formed an exclusive partnership with Rutgers in January 2019 to further develop the novel base editing technology invented by Dr. Shengkan Jin, associate professor of pharmacology, and co-inventor Dr. Juan C. Collantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School, and has since been funding research in base editing at the University while undertaking its own evaluation and proof-of-concept studies. Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking 3–5 partners to assess and shape the development of its Pin-point™ base editing platform.

Horizon will offer partners access to a novel system that could be used to progress more effective multi-gene knockout cell therapy programs through clinical development with an improved safety profile. Partners will also gain access to the Company’s expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Base editing is a novel technology for engineering DNA in cells, which the potential to correct certain errors or mutations in the DNA, or inactivate disease-causing genes. Compared with currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates “cuts” in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

Dr. Shengkan 'Victor' Jin of Rutgers University stated: “The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation. Other applications could include use as gene therapies for inherited genetic diseases including antitrypsin deficiency and Duchenne muscular dystrophy. In addition, we intend to take full advantage of the unique modularity and versatility features of Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well defined. At the top of this disease list are Alzheimer’s disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia.”