Cystic fibrosis is a common life-threatening genetic disease in the United States. The disease is caused by over 2,000 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are over 70,000 patients world-wide.
There are no FDA-approved drugs that can treat all 2,000 CFTR mutations. The FDA has approved three CFTR modulator therapies, Kalydeco®, Orkambi® and Symdeko® (Vertex Pharmaceuticals Inc.), to treat fewer than 40 cystic fibrosis-causing mutations. These drugs do not treat the underlying genetic cause of cystic fibrosis.
This allows airway cells to produce functional CFTR protein using their normal cellular machinery. This approach will be the first to treat the underlying defect that causes cystic fibrosis⎯dysfunctional or absent CFTR protein⎯in all patients, regardless of mutation type.
We have completed preclinical proof of concept studies, demonstrating that LUNAR is able to deliver mRNA efficiently into lung epithelial cells in animals and is compatible with nebulization. In cell-based assays, we have also emonstrated that our lead CFTR mRNA has improved functional activity and longer duration of protein expression compared to a reference CFTR mRNA.