Horizon Discovery announces progress of its gene and cell therapy platform

Horizon Discovery Group plc, the world leader in the application of gene editing technologies, today announces progress of its gene and cell therapy platform for contract service and therapeutic applications. The new offering will make use of Horizon’s extensive suite of gene editing technologies, including CRISPR and its proprietary rAAV and Transposon technologies, to edit pluripotent stem cells (iPSC) and primary human lymphocytes.

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Horizon is already working with three clients to support preclinical development across a variety of gene and cell therapy treatments in areas of high unmet medical need. This work is expected to contribute more than $2 million in revenue in 2017.

In addition, Horizon’s research biotech business is looking to exploit its unique IP and expertise to develop a universal cell therapy platform to support companies focused on developing autologous and allogeneic (“off the shelf”) cell therapy projects in areas of high unmet medical need. These include immuno-oncology, autoimmune diseases, rare genetic diseases, metabolic, neurological and blood disorders.

Dr Darrin M Disley, Chief Executive Officer, Horizon Discovery Group, commented: “Horizon is a world leader in gene editing, and our deep expertise combined with our suite of proprietary technologies makes us the perfect “go-to” partner for companies targeting gene and cell therapies across a range of diseases. The use of genetic screening approaches for novel target identification and precision gene editing for the subsequent development of gene and cell therapies are areas of burgeoning interest in biotech and pharma. Repairing the direct causes of genetic diseases, either at the DNA or cellular level will enable the personalised treatment of diseases at a lower cost and with better therapeutic results.”

Glossary:

  • Cell engineering/editing: The development of cell lines that reflect the genetics of disease found in real patients by use of gene editing
  • CRISPR: A gene editing technology that enables genomic modifications in a wide variety of organisms and tissues with high efficiency
  • Cell therapy: the administration of live whole cells (potentially gene edited) to a patient for the treatment of a disease
  • Gene therapy: the administration of vector to a patient for the treatment of a disease by direct correction (via gene editing) of a disease
  • Induced Pluripotent Stem Cells (iPS): cells derived from skin or blood that have been reprogrammed back into a basic state which can then be transformed into any type of human cell needed for research or therapeutic purposes
  •  rAAV: A gene editing technology, exclusive to Horizon for non-therapeutic applications, which allows cells to be edited with extreme precision and virtually no risk of unforeseen errors
  • Transposon gene editing: a type of ‘jumping gene’ that performs gene editing through a ‘copy and paste’ mechanism that allows multiple copies of a DNA sequence to be incorporated into a genome


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For further information from Horizon Discovery Group plc, please contact:

Horizon Discovery Group plc
Darrin Disley, Chief Executive Officer
Richard Vellacott, Chief Financial Officer
Chris Claxton, VP Investor Relations
Tel: +44 (0) 1223 655 580

Consilium Strategic Communications (Financial Media and Investor Relations)
Mary-Jane Elliott / Susan Stuart / Matthew Neal / Melissa Gardiner
Tel: +44 (0) 20 3709 5701
Email: horizon@consilium-comms.com

Zyme Communications (Trade and Regional Media)
Katie Odgaard
Tel: +44 (0)7787 502 947
Email: katie.odgaard@zymecommunications.com



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