Andrea Lobo, PhD,  science writer—

A Phase 2 clinical trial of an optimized formulation of adrulipase, a yeast-derived enzyme to treat exocrine pancreatic insufficiency (EPI) in adults with cystic fibrosis (CF), has screened its first patient. First Wave BioPharma, the therapy’s developer, anticipates dosing to start soon, with top-line results expected by mid-year.

A Phase 1 trial of ARCT-032, an inhaled experimental treatment for cystic fibrosis (CF), has been cleared to start in New Zealand. Developed by Arcturus Therapeutics, ARCT-032 contains the messenger RNA (mRNA) that provides instructions for lung cells to produce the CFTR protein, restoring its activity. Mutations in…

A higher body mass index (BMI) and recent exposure to certain antibiotics are risk factors for asymptomatic intestinal colonization with Clostridioides difficile in children with cystic fibrosis (CF), a new study suggests. The study, “Prevalence, Risk Factors, and Sequelae of Asymptomatic Clostridioides difficile Colonization in Children with Cystic…

Despite widespread use of telehealth, fewer cystic fibrosis (CF) patients in the U.S. met the recommendations for routine follow-up during the COVID-19 pandemic in 2020 than in 2019. However, the use of telehealth was related to higher adherence to aspects of CF care that can be evaluated remotely. “In…

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…

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…

The Phase 2a clinical trial testing Aridis Pharmaceuticals’ experimental inhaled therapy AR-501 for chronic lung infections in people with cystic fibrosis (CF) is fully enrolled. AR-501, designed to be given once a week, can be self-administered within minutes using a hand-held nebulizer that delivers the medication directly to…

The antibiotic Xenleta (lefamulin) was well tolerated in a Phase 1 clinical trial in adults with cystic fibrosis (CF) and showed a similar pharmacological profile to healthy volunteers. “We are excited to share positive topline results from this important study of Xenleta in patients with CF,” said Christine…

The Cold Spring Harbor Laboratory (CSHL) has licensed an artificial intelligence platform, developed by Epistemic AI, to accelerate cystic fibrosis (CF)-related discoveries. The platform, which also contains CF-specific resources, will help the lab’s scientists to accelerate their research by providing easy access to publications and clinical trials,…