Powering personalized medicine from sequence to treatment
Horizon Discovery (LSE: HZD) is a world-leading gene editing company headquartered in the Cambridge, UK biotech cluster. Horizon builds human disease models and reagents derived from gene edited cells that our customers use, or that we deploy on their behalf to: gain knowledge of the genetic drivers of disease; develop novel drugs or cell therapies targeted at these genetic drivers; and develop companion diagnostics that predict patient response in the clinic. In this way we apply the right genetics, in the right model, in the right context to provide biological insights that translate to improved research and development outcomes for drug developers and treatment regimens that better serve patients.
Our platform comprises multiple precision gene editing technologies (our proprietary technology rAAV together with CRISPR and ZFN), and is underpinned by an extensive intellectual property estate of owned or in-licensed rights.
Horizon participates in similar markets to life science tool and service stocks such as Abcam and Evotec, and uses gene editing to underpin the business similar to CRISPR stocks such as Editas and Intellia.
What is Gene Editing?
Gene editing is the process by which specific changes are made to the sequence of a gene within the context of a host cell. By editing the code of a patient-derived cell to introduce or repair a genetic anomaly believed to drive disease, the phenotype of a patient’s disease can be reproduced in a laboratory setting, allowing researchers to ask important biological questions of potential drugs or cell therapies earlier in the drug discovery process.
By deploying a world-leading position in gene editing Horizon is able to design, engineer and apply cells to advance human health.
Through a powerful combination and a decade of experience, where over 4,000 genes have yielded a catalogue in excess of 23,000 products, and with our gene editing platform, we are able to introduce virtually any modification to any cell according to our own, or our customers’, exact specifications.
Once built, engineered cells can also act as product manufacturing engines, yielding related cell and reagent products that can be used by customers as research tools or molecular diagnostic reference standards or as a means to generate advanced in vivo models, or even as factories for the manufacturing of protein-based therapeutics.
In this way, the cells we build power Horizon’s entire business from Products, Services to high value Research Biotech initiatives.
Horizon’s Products business unit offers a broad and expanding range of off the shelf products including gene edited cell lines and reference standards designed to drive research programs and improve clinical testing workflows through precisely engineered disease models.
Horizon’s Services business unit performs on-demand research services according to the specific needs of clients, and the generation of novel genetically-defined cell lines and in vivo models that can be deployed in the broader commercial offering.
Horizon’s Research Biotech business unit deploys our extensive intellectual property, gene editing platform, products, services and know-how alongside targeted investment to develop our immunotherapyo-oncology, synthetic lethality and cell and gene therapy ventures. This activity offers significant upside exposure to the business, providing access to high- value investment areas such as that those realized by cancer immunotherapy companies, gene editing companies and gene therapy companies.