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Horizon Discovery, RFUMS Collaborate on Disease Model of Cystic Fibrosis

NEW YORK (GenomeWeb News) – Horizon Discover today said it is collaborating with the Rosalind Franklin University of Medical Sciences to develop a human cellular disease model of cystic fibrosis.

The deal covers use of Horizon's Genesis precision genome editing technology to "reconstitute a specific mutation" in the cystic fibrosis transmembrane conductance regulator, or CFTR, protein in the endogenous genome of a lung cancer cell line.

The specific mutation in the CFTR protein is associated with about 90 percent of cystic fibrosis patients, Horizon said.

RFUMS will receive a perpetual academic research-use license to the materials under the terms of the agreement, while Horizon will keep the rights to commercially distribute the cell lines to third parties, including biotech and pharma firms. Through its Horizon Diagnostics division, the company also plans to develop reference standards based on the modified cell lines for assessing cystic fibrosis diagnostic tests.

Financial terms of the deal were not disclosed. The project is funded through a grant to Neil Bradbury and Robert Bridges at RFUMS by Cystic Fibrosis Foundation Therapeutics.

Bradbury said in a statement that while the US Food and Drug Administration approved the first CFTR therapeutic recently, less than 5 percent of patients carry the genetic profile for which the drug was approved.

"Studies using non-human cell models have identified several, potential CFTR therapeutics from high-throughput screens," he said. "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."