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Horizon Discovery and Rutgers University form exclusive partnership to develop next generation novel gene editing technology.

08:23 EST 28 Jan 2019 | Zyme Communications

  • Horizon will work with Rutgers University to develop and commercialise base editing platform for therapeutic and research applications
  • New gene editing technology builds on CRISPR and has the potential to assist in the development and treatment of a number of genetic diseases and cancers

Cambridge, UK, 28 January 2019: Horizon Discovery Group plc (LSE: HZD) (“Horizon” or “the Group”), a global leader in gene editing and gene modulation technologies, today announces that it has entered into an exclusive strategic partnership with Rutgers, The State University of New Jersey (US), to develop and commercialise a novel gene editing technology, known as base editing. The technology potentially has applications in the development of new cell therapies and will augment Horizon’s research tools and services.

Horizon will collaborate with Rutgers University to further develop the novel 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. As part of the collaboration, Horizon will also fund further research in base editing at Rutgers University while undertaking evaluation and proof of concept studies at Horizon. 

Base editing is a novel 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. 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. The technology will have a significant impact in enabling cell therapies to be progressed through clinical development and towards commercialisation. For more scientific detail on base editing, please see below in the notes to editors.

Terry Pizzie, Horizon’s Chief Executive Officer, said: “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. 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. 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. We look forward to updating on the progress of the partnership and potential future next steps.”

Dr. Shengkan ‘Victor’ Jin of Rutgers University stated: “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 CAR-T cells for leukemia, as well as in vivo therapeutics for inherited genetic diseases. 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.”

Dr. David Kimball, Interim Senior Vice President for Research and Economic Development at Rutgers University, added: “Gene editing technology has truly revolutionised how scientists think about their search for better options and outcomes in the treatment of disease. We look forward to advancing the shared goals of further developing this novel base editing platform and improving human health through this collaboration with Horizon.”

Base editing

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 anemia 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 utilising 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. 

About Horizon Discovery Group plc www.horizondiscovery.com

Horizon Discovery Group plc (LSE: HZD) ("Horizon") is a world leader in gene editing and gene modulation technologies. Horizon designs and engineers cells using its translational genomics platform, a highly precise and flexible suite of DNA editing tools (rAAV, ZFN, CRISPR and Transposon) and, following the acquisition of Dharmacon, Inc., its functional genomics platform comprising gene knockdown (RNAi) and gene [removed]cDNA, ORF) tools, for research and clinical applications that advance human health. Horizon's platforms and capabilities enable researchers to alter almost any gene or modulate its function in human or mammalian cell-lines.

Horizon offers an extensive range of catalogue products and related research services to support a greater understanding of the function of genes across all species and the genetic drivers of human disease and the development of personalised molecular, cell and gene therapies. These have been adopted by over 10,000 academic, drug discovery, drug manufacturing and clinical diagnostics customers around the globe, as well as in the Group's own R&D pipeline.

Horizon is headquartered in Cambridge, UK, and is listed on the London Stock Exchange's AIM market under the ticker HZD

About Rutgers University

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.

For further information from Horizon Discovery Group plc, please contact: Zyme Communications (Trade and Regional Media) Katie Odgaard Tel: +44 (0)7787 502 947 Email: katie.odgaard@zymecommunications.com Horizon Discovery Group plc Terry Pizzie, Chief Executive Officer Tel: +44 (0) 1223 655 580 Consilium Strategic Communications (Financial Media and Investor Relations) Mary-Jane Elliott / Matthew Neal / Melissa Gardiner Tel: +44 (0) 20 3709 5700 Email: horizon@consilium-comms.com NEXT ARTICLE

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