Cystic Fibrosis

The Disease:

  • Approximately one in 3,700 babies is born with CF in the U.S.
  • Causes persistent lung infections and progressively limits ability to breathe
  • Morbidity and mortality primarily caused by severity of lung diseases

The Editas Approach

In May 2016, we entered into 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 to financial support, CFFT will provide access to its extensive network of CF scientific advisors and clinical researchers.

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, we will explore targeting both common mutations as well as mutations not addressed by conventional approaches.