Up to four years of treatment with Symdeko (tezacaftor/izacaftor) is safe and effective in people with cystic fibrosis (CF) with one or two copies of F508del, a common CF-causing mutation. That’s according to new long-term data from an open-label Phase 3 trial that tested the approved CF therapy…
The European Commission (EC) has granted orphan drug designation to KB407, Krystal Biotech’s potential inhaled gene therapy for cystic fibrosis (CF). Orphan drug status is intended to encourage the development of therapies for rare, life-threatening, or chronically debilitating conditions, through a series of benefits, such as 10 years of market exclusivity…
A Phase 2 trial of ACG-701, an investigational oral treatment for pulmonary exacerbations in people with cystic fibrosis (CF), is underway at clinical sites in the U.S. The REPRIEVE trial (NCT05641298), launched in December, is evaluating the safety, pharmacokinetics, and effectiveness of ACG-701, being developed by Aceragen, when added…
Children with cystic fibrosis (CF) show significant differences from healthy children in the quality of their food choices and, subsequently, the microbes in their gut and respiratory tract, with evidence of greater intestinal inflammation in these young patients, a recent study in Australia found. Certain microbes in the gut associated…
Trikafta (elexacaftor/tezacaftor/ivacaftor) was seen to keep lung function from getting worse in people with cystic fibrosis (CF) for up to two years, a study found. These findings counter previous studies wherein the active ingredients in Trikafta, alone or in combinations of two, were seen to slow, but not…
The first patient has been dosed in a Phase 1b/2 clinical trial testing WRAIR-PAM-CF1, an investigational bacteriophage therapy, in adult cystic fibrosis (CF) patients with chronic Pseudomonas aeruginosa lung infections. The PHAGE trial (NCT05453578) is recruiting an estimated 72 such patients who tested positive for a P. aeruginosa infection…
The Cystic Fibrosis Foundation is investing up to $15 million in ReCode Therapeutics to support the development of the company’s inhaled mRNA-based therapy for cystic fibrosis (CF). “We are excited to expand our relationship with the CF Foundation with this investment in our CFTR mRNA program for cystic…
Renexxion is seeking clearance from the U.S. Food and Drug Administration (FDA) for clinical testing of the treatment candidate naronapride in people with cystic fibrosis (CF) and gastrointestinal (GI) problems. The Ireland-based biopharmaceutical company, which specializes in treatments for gastrointestinal disorders, announced the opening of an investigational new…
Sionna Therapeutics is forging ahead in the new year with its preclinical and clinical programs aimed at developing small molecules — such as SION-638 — as possible treatments for cystic fibrosis (CF). An ongoing Phase 1 trial, now enrolling participants, is testing SION-638 in healthy volunteers. Dosing…
A novel inhaled antibiotic that Spexis is developing to treat infections of Pseudomonas aeruginosa — the key bacterial agent of lung infections in cystic fibrosis (CF) — showed a good safety profile in a first trial conducted in healthy volunteers, the company announced. “The first clinical data with our inhaled…
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