News

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…

First Wave BioPharma has asked the U.S. Food and Drug Administration (FDA) for permission to start a Phase 2 clinical trial that would test the company’s new formulation of adrulipase in people with cystic fibrosis (CF) who have exocrine pancreatic insufficiency. Submitting the application “is an important step…

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…

A novel gene-editing tool that allows scientists to “drag-and-drop” sequences of DNA into the genome, without inducing large cuts, could aid in treating genetic diseases like cystic fibrosis (CF), a study reported. The technology, aptly called PASTE (for programmable addition via site-specific targeting elements), “expands the capabilities of genome…

Enrollment of adults at sites across the U.S. is continuing for a Phase 2a trial of AR-501 (gallium citrate), Aridis Pharmaceuticals‘ investigational inhaled therapy for chronic lung infections due to cystic fibrosis (CF). As an inhaled treatment, AR-501 is intended to be self-administered weekly using a hand-held nebulizer that…