Editas Medicine has announced a three-year agreement with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the Cystic Fibrosis Foundation, in which CFFT will pay up to $5 million to Editas Medicine to support the discovery and development of CRISPR/Cas9-based medicines for the treatment of cystic fibrosis (CF). In addition, Editas will access CFFT’s extensive network of CF scientific advisors and clinical researchers.
“We are delighted to work with Cystic Fibrosis Foundation Therapeutics to discover and develop medicines for people with cystic fibrosis,” said Katrine Bosley, President and Chief Executive Officer, Editas Medicine. “While significant medical and scientific progress has been made in recent years, a great deal of work remains. I’m hopeful that our genome editing approach will one day create important new therapies that can address the genetic mutations that cause this disease.”
"We believe that the CRISPR approach to gene editing holds significant promise for repairing the underlying cause of cystic fibrosis," said Preston W. Campbell III, M.D., President and Chief Executive Officer, CF Foundation. "We’re pleased to work with Editas Medicine and are excited by the possibilities of what can be accomplished on behalf of people with CF.”
The gene that causes CF encodes the cystic fibrosis transmembrane conductance regulator protein (CFTR), which helps maintain the water balance within the lung and in other tissues. Mutations in this gene lead to problems with the flow of fluids and salt into and out of cells. This causes a thick buildup of mucus in the lungs, pancreas and other organs. The mucus clogs the airways and traps bacteria, leading to chronic infections and inflammation of the airways. There are more than 1,800 known mutations within the CFTR gene. As part of this agreement, Editas Medicine will explore targeting both common mutations as well as mutations not addressed by conventional approaches.