Core Insights - Sionna Therapeutics is focused on developing novel medicines to improve the treatment of cystic fibrosis (CF) by normalizing the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1][3] Group 1: Upcoming Presentation - Data will be presented at the 2025 North American Cystic Fibrosis Conference (NACFC) in Seattle from October 22-25, 2025 [1] - The presentation will cover the safety, tolerability, and pharmacokinetics of novel NBD1 stabilizers SION-719 and SION-451 from two phase 1 first-in-human studies [2] - The session will take place on October 24, 2025, from 10:15 a.m. to 12:15 p.m. PT [2] Group 2: Research Focus - Sionna Therapeutics aims to restore CFTR function to near-normal levels by stabilizing CFTR's nucleotide binding domain 1 (NBD1) [3] - The company is advancing a pipeline of small molecules designed to correct defects caused by the F508del genetic mutation, which affects NBD1 [3] - Sionna is also developing complementary CFTR modulators to work synergistically with its NBD1 stabilizers to enhance CFTR function [3]
Sionna Therapeutics to Present Data at the 2025 North American Cystic Fibrosis Conference