Sionna Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results

Ongoing PreciSION CF Phase 2a proof-of-concept trial evaluating NBD1 stabilizer SION-719 as an add-on to standard of care in participants with ...

SpliSense Granted EMA EARLY PRIME Designation for SPL84 in Cystic Fibrosis

SpliSense, a clinical-stage biotechnology company developing transformative RNA-based therapies for pulmonary diseases, today announced that the European Medicines Agency (EMA) has granted early ...
Nasdaq

Sionna Therapeutics Presents Preclinical Data on Novel CFTR Modulators at the 48th European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
Healio

Cystic fibrosis triple therapy outcomes improve regardless of prior modulators

Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...
Cystic Fibrosis Foundation

Our Thoughts on Changing CF Care

Cystic fibrosis care is changing quickly — with better treatments, longer lives, and new tools like telehealth. This is pushing us to rethink what high quality, individualized care should look like.
Business Insider

Krystal Biotech Announces Positive Interim Clinical Update from KB407 Phase 1 CORAL-1 Study with Confirmation of Wild-Type CFTR Delivery to the Lungs of Patients with Cystic ...

Confirmed wild-type CFTR delivery and expression in conducting airway cells of patients with class I mutations KB407 transduction confirmed in all six patients with successful bronchoscopies ...
Healio

CFTR modulator use during pregnancy preserves maternal lung function

Please provide your email address to receive an email when new articles are posted on . “[These findings] help clinicians be more supportive of a female with CF becoming pregnant,” Raksha Jain, MD, ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
Business Wire

Vertex Announces Investigational New Drug (IND) Application for VX-522, mRNA Therapy for People With Cystic Fibrosis, Cleared by FDA

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for VX ...
MedPage Today

Next-in-Class Once-Daily Pill Approved for Cystic Fibrosis

The FDA approved a once-daily oral combination of vanzacaftor/tezacaftor/deutivacaftor (Alyftrek) for cystic fibrosis (CF) in adults and children 6 years and up ...
Nasdaq

Sionna Therapeutics to Present Preclinical Data on CFTR Modulators at European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. announced that preclinical data on its novel cystic fibrosis (CF) treatment candidates, SION-451 and SION-719, will be presented at the European Cystic Fibrosis Society’s ...