Sionna Therapeutics Showcases Breakthrough Findings in CF Research

Insights into Sionna Therapeutics' Groundbreaking Research on Cystic Fibrosis

Sionna Therapeutics, Inc. (Nasdaq: SION), a leading clinical-stage biopharmaceutical company, is on a transformative journey to change how cystic fibrosis (CF) is treated. Their innovative approach aims to develop medicines that can restore the normal function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a significant breakthrough for those battling this condition.

Upcoming Presentation at the 2025 North American Cystic Fibrosis Conference

Excitingly, Sionna Therapeutics has announced its participation in the upcoming 2025 North American Cystic Fibrosis Conference. This prestigious event, taking place from October 22-25, 2025, will serve as a platform for Sionna to showcase critical findings from their latest studies. The conference is eagerly anticipated as it will bring together experts and advocates in the cystic fibrosis community.

Research Focus and Key Study Highlights

One of the central presentations at the conference will delve into the safety, tolerability, and pharmacokinetics of novel NBD1 stabilizers, SION-719 and SION-451. These findings emerge from two pivotal phase 1 first-in-human studies, which are fundamental in understanding the potential benefits of these drugs for cystic fibrosis patients.

Dr. Jason H. Maley, Senior Director of Clinical Development at Sionna, will present these findings during a dedicated session titled "W20 - Cutting-edge Clinical Trials: Insights & Updates Workshop". This session is scheduled for Friday, October 24, 2025, from 10:15 a.m. to 12:15 p.m. PT, during which attendees can engage with Sionna’s advances in CF treatment.

Targeting CFTR: A Purposeful Approach

Another impactful study that Sionna will present focuses on small molecule stabilizers of CFTR NBD1. This research highlights how these stabilizers can significantly extend the half-life of the apical glycoform of F508del-CFTR, bringing it closer to the baseline function seen in wild-type CFTR. The authors of this poster, who include several esteemed researchers alongside Gregory Hurlburt, Ph.D., co-founder of Sionna, illustrate through their findings the potential of these medicines to provide significantly better outcomes for patients.

Sionna Therapeutics: Committed to Advancing CF Care

The mission of Sionna Therapeutics is clear: they are dedicated to revolutionizing CF treatment through innovative therapies. By directly addressing the malfunctioning CFTR, specifically through stabilizing the NBD1 domain, Sionna aims to restore and enhance CFTR function. This advancement could mean dramatic improvements in both clinical outcomes and the overall quality of life for those affected by cystic fibrosis.

Advancing a New Era in Cystic Fibrosis Treatment

With over a decade of focused research into NBD1, Sionna is forging a promising path ahead. The company's pipeline features a range of small molecules specifically engineered to correct defects linked to the F508del mutation within the NBD1 region. Additionally, Sionna is developing a comprehensive portfolio of complementary CFTR modulators that are designed to synergize with their NBD1 stabilizers, further enhancing efficacy in CF treatment.

Stay Informed with Sionna Therapeutics

Those interested in the latest updates from Sionna Therapeutics can find the presentations and posters from the conference on their website, specifically under the “Scientific Presentations” section. This is a valuable resource for anyone seeking detailed insights into Sionna's contributions to cystic fibrosis research.

Contact Information for Further Inquiries

For media inquiries, Adam Daley from CG Life can be reached at 212.253.8881. Investors looking for more information can contact Juliet Labadorf through the designated investor relations email.

Frequently Asked Questions

What is the focus of Sionna Therapeutics' research?

Sionna Therapeutics focuses on developing innovative treatments for cystic fibrosis, specifically targeting the CFTR protein to restore its function.

Where will Sionna present its research findings?

Sionna will showcase its research at the North American Cystic Fibrosis Conference, which is set to take place in late October.

Who is the presenting author for Sionna’s research?

The presenting author for the key studies is Dr. Jason H. Maley, Senior Director of Clinical Development at Sionna Therapeutics.

What are NBD1 stabilizers?

NBD1 stabilizers are novel compounds developed by Sionna that aim to enhance the stability and function of the CFTR protein, specifically addressing mutations like F508del.

How can I learn more about Sionna Therapeutics?

For more information about Sionna Therapeutics and their ongoing research, visit their official website.

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