Life Sciences Focus: Drug Discovery
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Gene editing and gene modulation technologies support drug discovery
Horizon Discovery has developed a broad range of gene editing and gene modulation technologies that enable scientists to gain a greater understanding of gene function and apply this knowledge to the development of novel biotherapeutics, gene therapies and diagnostic workflows. This article reviews recent developments at the company.
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eadquartered in Cambridge, UK and with offices in the US and Japan, Horizon Discovery Group plc specializes in the application of gene editing and gene modulation technologies that support drug discovery and development within the global life sciences sector, offering a portfolio of tools and services to help scientists gain a greater understanding of gene function, identify genetic drivers behind human disease and deliver biotherapeutics, cellular and gene therapies for precision medicine as well as for the development and validation of diagnostic workflows. The company’s solutions are designed to enable almost any gene to be altered, or its function modulated, in human and other mammalian cell lines. Its clients include leading academic institutes, global pharmaceutical and biotechnology companies and clinical diagnostic laboratories. Horizon says that its insight into the challenges faced by these organisations enables it to focus its efforts on the development of innovative solutions that not only differentiate its offering, but that also fuel development of the next wave of precision medicines.
Gene knockout and gene activation studies Earlier this month (July 2020), the company introduced its stably expressing Cas9 and dCas9-VPR cell lines to help accelerate gene knockout and gene activation experiments, respectively. The
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Horizon Discovery is now offering an arrayed CRISPR knockout screening service for primary human B cells, cells that are freshly isolated from donors and known to be difficult to study in the lab. Pictured is a B-lymphocyte antibody-producing immune cell. (Image from Shutterstock © Kateryna Kon)
cell lines are optimized to work alongside the company’s Edit-R predesigned synthetic single guide RNA (sgRNA) and CRISPRa guide RNA, offering researchers a complete solution to simplify and streamline CRISPR gene editing and modulation workflows. Horizon’s Cas9 and dCas9- VPR stable cell lines were generated using its Edit-R Lentiviral particles with a blasticidin resistance cassette and are provided in pooled
format. The cell lines are QC-verified and validated to ensure stable expression and functionality of Cas9 or dCas9- VPR endonuclease in a range of common cell backgrounds. Both cell lines are available in the same background to enable loss-of-function and gain-of- function studies to be performed in parallel without the need to engineer a cell line specifically for this purpose. Horizon says that removing the time-intensive
step of generating a stable cell line and the cost associated with purchasing a nuclease could help researchers increase R&D productivity and allow novice users to gain a better understanding of the CRISPR workflow.
Production of highly complex proteins In a separate recent development, in May of this year, biopharmaceuticals CDMO
Summer 2020
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