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Horizon and RFUMS to Develop Cystic Fibrosis Cellular Disease Models

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Horizon Discovery (Horizon) has announced its collaboration with the Rosalind Franklin University of Medical Sciences (RFUMS) to develop a human cellular disease model of cystic fibrosis.

The agreement focuses on the use of Horizon’s GENESIS™ precision genome editing technology to reconstitute a specific mutation in the CFTR (cystic fibrosis transmembrane conductance regulator) protein which is associated with approximately 90% of cystic fibrosis patients, in the endogenous genome of a lung cancer cell line.

Under the terms of the agreement, RFUMS will receive a perpetual academic research use license to the materials.

Horizon will retain rights for commercial distribution of the cell lines to third parties, including biotech and pharmaceutical companies.

Horizon also intends to develop, through its Horizon Diagnostics division, commercially available reference standards based on the modified cell line, for quality assessment of cystic fibrosis diagnostic tests.

The project is funded through a grant awarded to Drs Neil Bradbury and Robert Bridges at RFUMS by Cystic Fibrosis Foundation Therapeutics Inc.

“Approval for the first CFTR therapeutic was recently granted by the FDA, however, fewer than 5% of patients carry the genetic profile for which this drug was approved. We hope that by creating this tool targeting the more common CFTR mutation, we can ultimately improve the lives of a far greater proportion of cystic fibrosis sufferers,” explained Dr Neil Bradbury, lead investigator at RFUMS.

Dr Bradbury continued, “Studies using non-human cell models have identified several, potential CFTR therapeutics from high-throughput screens. We hope to improve the efficiency of this process by creating a more informative screening tool with Horizon’s isogenic disease model cell lines, which we believe will also be useful for the broader research community.”

Dr Darrin Disley, CEO, Horizon, said: “Horizon’s precision genome editing technology, which enables the recapitulation of genetic mutations in cell models with unprecedented accuracy, exactly as they occur in the primary tissue, has an important role to play in progressing research in all therapeutic areas. Horizon’s mission is to shape the next generation of personalized medicines across many different therapeutic areas. We’re very pleased that RFUMS chose to partner with Horizon on this program.”

Horizon’s X-MAN™ isogenic cell line catalogue currently consists of over 400 different genetically defined paired mutant and normal cell lines, and includes cancer and HIV related cell lines.