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Sionna Therapeutics
Sionna Therapeutics (SION) investor relations material
Sionna Therapeutics Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Guggenheim Securities Emerging Outlook: Biotech Summit 2026 summary11 Feb, 2026
Company background and mission
Focused on revolutionizing cystic fibrosis (CF) treatment using first-in-class NBD1 stabilizers, aiming to disrupt a market long dominated by a single player.
Originated from Genzyme and Sanofi science, with Sionna formed in 2019 to accelerate CF research.
Currently advancing multiple NBD1 programs and complementary mechanisms in clinical development.
Scientific rationale and innovation
NBD1 is a critical domain in the CFTR protein, with the F508del mutation affecting 90% of CF patients and causing protein instability.
Existing therapies like TRIKAFTA do not directly stabilize NBD1, leaving room for improvement.
NBD1 was long considered undruggable due to shallow binding pockets, but Sionna has developed two clinical-stage NBD1 stabilizers.
Preclinical data show NBD1 stabilization can restore CFTR protein half-life to normal levels.
Clinical development and study design
The phase 2a PreciSION-CF study tests SION-719 added to TRIKAFTA in F508del homozygous patients, aiming to show sweat chloride improvement as a biomarker of CFTR function.
Study uses a crossover design with each patient as their own control, targeting a 10 mmol/L sweat chloride improvement, considered clinically meaningful.
Patient population is carefully selected for homogeneity and room for improvement on TRIKAFTA.
Data readouts for both the PreciSION-CF and dual combination studies are expected in mid-2026, with more precise timing to be announced after full enrollment.
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Next Sionna Therapeutics earnings date
Q4 202520 Mar, 2026
Sionna Therapeutics
Next Sionna Therapeutics earnings date
Q4 202520 Mar, 2026
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Frequently asked questions
Sionna Therapeutics Inc is a biotechnology company focused on the research and development of therapies targeting genetic diseases. The company specializes in discovering small molecule treatments designed to modulate protein function and address the underlying causes of certain conditions. Sionna Therapeutics conducts preclinical and clinical research to advance its pipeline of drug candidates, primarily in the area of rare and inherited disorders. The company collaborates with research institutions and healthcare partners to support its development efforts. Sionna Therapeutics Inc is headquartered in Boston, Massachusetts, and its shares are listed on the NASDAQ.
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