“This Phase 2a trial evaluated the potential effect of an oral compound to improve trafficking of the defective CFTR protein, and its results represent an encouraging step forward in the development ...

Phase 1 data of first-in-class NBD1 stabilizers, SION-719 and SION-451, demonstrated they were generally well tolerated and exceeded desired pharmacokinetic targets New preclinical data show that NBD1 ...

- For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease– -12,000 people with one ...

- With this reimbursement agreement in place, more than 2,200 people with CF will have PBS-funded access to TRIKAFTA®, including more than 700 who will now have access to a CFTR modulator therapy for ...

Vertex Pharmaceuticals Incorporated VRTX today announced interim results from the first part of a Phase 2 study designed to evaluate multiple combination regimens of VX-770 and VX-809, Vertex's lead ...

BOSTON--Vertex Pharmaceuticals Incorporated (VRTX) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization ...

LONDON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has validated a Type II Variation Marketing Authorization Application ...

WALTHAM, Mass., Oct. 24, 2025 (GLOBE NEWSWIRE) -- Sionna Therapeutics, Inc. (Nasdaq: SION), a clinical-stage biopharmaceutical company on a mission to revolutionize the current treatment paradigm for ...