Project Overview

We are developing base editors capable of correcting a mutation in cystic fibrosis (CF) that is not responsive to current drugs, and engineering delivery vehicles tailored to the treatment of CF.

The discovery of pharmacologic cystic fibrosis transmembrane conductance regulator (CFTR) modulators has transformed the landscape for cystic fibrosis (CF) treatment worldwide. A substantial fraction of CF patients can benefit from the small molecule ivacaftor and its partner drugs. However, around 10 percent of patients are not eligible for such therapy, either because their CFTR genotype is refractory to existing drugs or because they cannot tolerate modulator therapy. For these patients, there is an urgent need for alternative strategies to restore CFTR function.

Our goals for this project are to (1) develop base editors corresponding to CFTR mutations that are currently beyond the reach of ivacaftor and its partner drugs, (2) develop a pipeline for cell-specific assessment of the editing performed by the CF-specific base editors developed in Goal 1, and (3) use an animal model of CF for pre-clinical testing of approaches developed and refined in Goals 1 and 2.

Learn more about the IGI Delivery Collective, a collaborative effort working to develop new technology for delivery of CRISPR-based therapies.