Horizon Discovery introduces single cell RNAseq-linked CRISPR screening service

• CRISPR-based screening technology platform to address critical gaps in target ID and validation

• Single cell RNAseq-linked pooled CRISPR screening offers high-quality screening data and biological insight

Cambridge, UK, 04 August 2020: Horizon Discovery Group plc (LSE: HZD) ("Horizon", "the Company", or "the Group"), a global leader in the application of gene editing and gene modulation for cell line engineering, today announced the addition of single cell RNAseq-linked pooled CRISPR screening to its CRISPR screening services portfolio. The platform offers high-quality screening data and biological insight to address critical gaps in target identification and validation.

This launch is the result of a collaboration, announced in 2018, to develop and apply this novel research tool to discover new biology essential for the development of future therapeutics. The biological insight gained from Horizon’s single cell RNAseq-linked CRISPR screening platform could help researchers to gain further understanding into complex biological models and speed up the time from discovery to validation by integrating the effect of gene editing with complex gene expression mapping.

Horizon’s pooled format screens offer researchers access to highly robust whole-genome level analyses that yield quality data outputs. Currently, it can be challenging to adequately multiplex the data from these screens to evaluate complex biological phenomena occurring in a subset of edited cells. Coupling pooled CRISPR screening to single cell RNAseq allows the impact of CRISPR-based gene modification to be examined on a global transcriptomic scale at single cell resolution. This could enable scientists to address critical gaps in target identification and validation as they work to develop new and more effective therapeutics.

“Pooled CRISPR-Cas9 knockout screens have become a linchpin in drug target identification and validation. The development of our single cell RNAseq-linked pooled CRISPR screening service provides a substantial advance to Horizon’s screening capabilities,” said Terry Pizzie, Chief Executive Officer, Horizon Discovery. “Our continuing development of cutting-edge services, including CRISPR screens in primary human immune cells, means we can offer our customers unparalleled insights into the biology that will help to underpin future drug and cell-based therapeutics.” For further information about Horizon’s cell-based screening services, please visit: https://horizondiscovery.com/navigation/screening/functional-genomic-screening

ENDS

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Horizon Discovery’s single cell RNAseq-linked pooled	Terry Pizzie, CEO, Horizon Discovery
CRISPR screening maps transcriptomic changes

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About Horizon Discovery Group plc - horizondiscovery.com 

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 Company’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 the Company to focus efforts on development of innovative solutions that not only differentiate the Company’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.

Follow Horizon Discovery on Twitter @HZDiscovery and LinkedIn @Horizon Discovery

About Horizon’s High-throughput Functional Genomic Screening platform

Horizon has established a core expertise in functional genomic screening using CRISPR-Cas9 and RNAi screening platforms. These techniques are used to explore the function of genes by interrupting their sequence or disrupting the process by which they lead to the generation of protein. In this way, these screens can be used to find and validate novel drug targets, to identify mechanisms of drug resistance or sensitivity, and to aid with patient stratification. Horizon performs these screens in both normal human cell lines and also in haploid cell lines, which represent a simplified and efficient model with which to explore gene function as these cells have only one copy of each gene. Horizon’s CRISPR platform includes CRISPR knockout, which uses gene editing to effect function, as well as CRISPRi and CRISPRa. CRISPRi, which induces gene repression, and CRISPRa, which activates gene expression, provide powerful applications for CRISPR screening. CRISPRi screening is especially well suited to study the function of genes that when knocked out are essential for cell survival, or that are amplified to provide a functional effect, both classes of genes that are not amenable to CRISPR knockout screens. CRISPRa screening enables, for the first time, the study of activation-linked responses on genome-wide level.

• RNA sequencing (RNAseq): The using of next generation sequencing to quantitatively study the expression level of specific genes via the level of mRNA in the cell

• CRISPR: A gene editing technology that enables genomic modifications in a wide variety of organisms and tissues with high efficiency

• CRISPRa and CRISPRi: Nuclease deficient versions of Cas9 that enable the repression or overexpression respectively of target genes

• Functional genomic screening platform: A platform used by Horizon to identify and validate genes as targets for novel therapeutics using CRISPR based screening and other approaches

• Knockout: Genetic technique in which one of an organism’s genes is made inoperative.