Promising early stage clinical trial results for three next-generation CFTR modulator therapies that target the underlying defect in cystic fibrosis were announced.

The studies, led by Vertex Pharmaceuticals Inc., highlight the potential of next-generation combination therapies to be significantly more effective than current FDA-approved modulators and to also benefit more people with CF than ever before. Data from the trials, along with results from another study expected to begin later this year, will help Vertex decide which next-generation modulator candidate or candidates to move forward into Phase 3 studies in early 2018. We are especially excited about this treatment since this medication will benefit KATIE and all those with this CF gene mutation!!!