Sionna Therapeutics showcases novel CFTR stabilizers at NACFC 2023

At the forefront of cystic fibrosis research, Sionna Therapeutics recently unveiled critical preclinical findings on their innovative Series 2 NBD1 stabilizers at the 2023 North American Cystic Fibrosis Conference. The company’s stabilizers, SION-719 and SION-451, have demonstrated the potential to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in preclinical models.

Targeting the Core of CF’s Most Common Mutation

Focusing on the challenging ΔF508 mutation, the root cause of the majority of CF cases, Sionna Therapeutics is developing its Series 2 NBD1 stabilizers to directly address this mutation’s destabilizing effects. Currently, Sionna is progressing with a Phase 1 clinical trial for SION-638, while SION-719 and SION-451 are progressing toward Investigational New Drug (IND) application readiness.

“Our science team’s dedication over a decade has culminated in the discovery of first-in-class modulators that directly stabilize NBD1,” shared Dr. Greg Hurlbut, celebrating the company’s long-term research efforts.

Preclinical Data Indicates a Major Advance in CF Treatment

Preclinical results presented by Sionna showed that their Series 2 stabilizers, in combination with other modulators, achieved full wild-type levels of ΔF508-NBD1 stability and functionality—a substantial finding for the CF treatment landscape.

Sionna Therapeutics’ Strategic Vision for CF Care Improvement

Mike Cloonan, President, and CEO of Sionna, stressed the importance of Sionna’s research in improving upon current CF treatments, aiming to achieve normalized CFTR function for the majority of patients. With the advancement of SION-719 and SION-451 to clinical stages, the company is making strides toward a new era of CF therapy.