News

Researchers have discovered that monocytes, a type of white blood cell, drive persistent and sustained inflammation that leads to lung damage in people with cystic fibrosis (CF), a study reported. The researchers showed that, in a CF mouse model, reducing monocyte activity lowered the number of tissue-damaging immune cells…

SpliSense has launched a Phase 1/2 clinical trial to test SPL84, its inhaled treatment candidate for cystic fibrosis (CF) patients carrying a common disease-causing splicing mutation. As of now, no specific treatment is available for people with CF who carry this particular mutation. Called 3849+10 kb C-to-T, it…

The Cystic Fibrosis Foundation has invested another $4.85 million — termed an equity investment — in Aridis Pharmaceuticals to advance the clinical development of AR-501, an inhaled treatment for chronic lung infections in people with cystic fibrosis (CF). The total funding from the CF Foundation for the clinical…

Vertex Pharmaceuticals is launching a clinical trial to test VX-522, its inhaled messenger RNA (mRNA) therapy for lung disease in cystic fibrosis (CF) patients who are not eligible for treatment with an existing CFTR modulator. The study is expected to open soon, and it will assess VX-522’s safety and…

People with cystic fibrosis (CF) who are treated with Orkambi and Symkevi (Symdeko) in real-world settings tend to experience less decline of lung function, though results may be more modest and variable than what was reported in clinical trials for these therapies, a new study reports. “The…

The first participant has been dosed in a Phase 1 clinical trial evaluating Sionna Therapeutics‘ SION-638, an investigational oral treatment for cystic fibrosis (CF). The trial aims to assess the safety and pharmacokinetics — the movement into, through, and out of the body — of SION-638 in healthy volunteers.

First Wave Biopharma has selected the initial sites for the Phase 2 clinical trial of a new adrulipase formulation to treat exocrine pancreatic insufficiency (EPI) in people with cystic fibrosis (CF). “We believe that our reformulation of adrulipase offers the potential to provide a dramatically improved treatment option…

The U.K. National Institute for Health and Care Research awarded £1.5 million (about $1.8 million) for research on an at-home monitoring system for cystic fibrosis (CF) patients. The study will start in early 2023 and enroll patients across the U.K., who will use small wearable devices to collect health…

CF Youth, a new exercise program created by Beam for children living with cystic fibrosis (CF), was launched on the company’s online platform. The initiative, created in collaboration with CF Yogi and Johns Hopkins Cystic Fibrosis Center, can be accessed by caregivers and healthcare providers. Regular exercise has been…

A 30-year-old woman with cystic fibrosis (CF) and poor lung function gave birth to a healthy child while being treated with Trikafta, reportedly the first such successful CF pregnancy in Switzerland. The mother’s percent predicted forced expiratory volume in one second (ppFEV1) — meaning her expected ability to…