Sionna Therapeutics Sionna is leveraging more than a decade of extensive research to advance first-in-class small molecules engineered to stabilize CFTR’s nucleotide-binding domain 1 (NBD1), where the most common CF genetic mutation, F508del, resides. Despite having long been identified as a critical component for proper CFTR function, NBD1 has been considered “undruggable”, and none of the currently approved CF therapies directly stabilizes it. Sionna is also developing a portfolio of complementary CFTR modulators that are designed to work synergistically with its NBD1 stabilizers to improve CFTR activity. Sionna believes stabilizing NBD1 is central to unlocking dramatic improvements in clinical outcomes and quality of life for CF patients.
The Foundation supported the development of this type of modulator therapy for more than a decade by funding early-stage F508del corrector discovery work at Genzyme/Sanofi that eventually led to Sionna’s current pipeline. Since 2011, the Foundation has provided about $51 million to this research, including about $13 million directly to Sionna.