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]

Sionna Therapeutics Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, performance or other events to be materially different from any future results, performance or other events expressed or implied by the forward-looking statements. Given these uncertainties, you should not place undue reliance on forward-looking statements. Our actual future results, performance or other events may be materially different from what we expect. Exc ...

SION-719 and SION-451 were generally well tolerated and achieved desired pharmacokinetic targets that reinforce their potential as either an add-on to standard of care or in a Sionna dual combination Positive Phase 1 Results for SION-719 and SION-451 The Phase 1 randomized, double-blind, placebo-controlled clinical trials evaluated the safety, tolerability, and PK profiles of single ascending doses (SAD) and multiple ascending doses (MAD) of SION-719 and SION-451 in healthy volunteers. The effect of food on ...

WALTHAM, Mass., May 22, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that preclinical data assessing combinations of Sionna’s nucleotide-binding domain 1 (NBD1) stabilizers, SION-451 and SION-719, with complement ...

WALTHAM, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the prese ...

WALTHAM, Mass., April 30, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for cystic fibrosis (CF) by developing novel medicines that normalize the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, today announced that management will present at The Citizens Life Sciences Conference on Wednesday, May 7, 2025 at 12:30 p.m. ET. A live webcast of the prese ...

Vertex Pharmaceuticals (VRTX) announced that the European Commission (EC) has expanded the label of its blockbuster cystic fibrosis (CF) drug, Kaftrio, to treat all patients aged two years and older who have at least one non-class I mutation in the CFTR gene.This approval was expected as the EMA’s Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending Kaftrio’s approval in February for the above indication. With this expanded label, nearly 4,000 more CF patients livin ...