Core Insights - Sionna Therapeutics is focused on developing novel medicines to normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, aiming to revolutionize the treatment paradigm for cystic fibrosis (CF) [1][4] Company Overview - Sionna Therapeutics is a clinical-stage biopharmaceutical company dedicated to creating therapies that restore CFTR function, particularly targeting the nucleotide-binding domain 1 (NBD1) [4] - The company has a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation, which is prevalent in CF patients [4] Research and Development - Sionna has developed NBD1 stabilizers, SION-719 and SION-451, which have shown the ability to increase the stability of isolated ΔF508-NBD1 by 16°C, surpassing wild-type NBD1 levels [5] - Preclinical data indicate that SION-719 and SION-451 can correct F508del-CFTR maturation to wild-type levels when combined with other modulators, SION-2222 and SION-109 [5] - The company plans to advance SION-719 into a Phase 2a proof-of-concept trial and SION-451 into a Phase 1 trial, both expected to start in the second half of 2025 [3] Conference Presentation - Sionna presented preclinical data at the 48th European Cystic Fibrosis Conference, showcasing the effectiveness of dual combinations of NBD1 stabilizers with proprietary modulators [1][3]