Marisa Wexler, MS,  senior science writer—

Having cystic fibrosis (CF) can put a tremendous financial strain on patients: In the U.K., the typical family affected by the genetic disease will lose more than £6,500 (about $8,000) every year as a consequence. That’s according to a new report, “The Financial Costs of Cystic…

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…

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…

The Phase 1b/2a clinical trial SWARM–Pa, testing Armata Pharmaceuticals‘ experimental phage therapy AP-PA02 in people with cystic fibrosis (CF) who have chronic Pseudomonas aeruginosa infections, is complete, Armata announced. Data will now be analyzed, and top-line results are expected early in the new year. “We are very pleased to…

Claire’s Place Foundation has announced the launch of its fundraising campaign, called #CPFoneinamillion, to support families with cystic fibrosis (CF) during extended hospital stays. Since its founding in 2011, the nonprofit has given $875,000 in Extended Hospital Stay Grants to people affected by CF. The new…

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…

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…

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…

Vertex Pharmaceuticals recently filed an application with the U.S. Food and Drug Administration (FDA) asking that Trikafta, its triple-combination modulator therapy, be approved for children with cystic fibrosis (CF) ages 2 to 5. The company is planning to file similar requests in the European Union and U.K.

A new test that measures the excretion of bicarbonate in urine may be used to assess the effectiveness of treatments for cystic fibrosis (CF), according to a new study. “The goal is for the urine test to be used as a clinical tool to determine both the severity of…