Date: 2012-03-12
Type of information: R&D agreement
Compound: human cellular disease model of cystic fibrosis
Company: Horizon Discovery (UK) Rosalind Franklin University Medical Sciences - RFUMS (USA)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: R&D
Action mechanism:
Disease: cystic fibrosis
Details: Horizon Discovery Limited, a leading provider of research tools to support the development and prescription of personalized medicines, 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.
Financial terms: The project is funded through a grant awarded to Drs Neil Bradbury and Robert Bridges at RFUMS by Cystic Fibrosis Foundation Therapeutics Inc.
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