Patricia Inácio, PhD, science writer —

Arrowhead Pharmaceuticals has filed for approval to launch a Phase 1/2a clinical trial in New Zealand testing its candidate inhaled therapy for cystic fibrosis (CF), called ARO-ENaC, the company announced. ARO-ENaC is an investigational RNA therapy designed to lower the production of the epithelial sodium channel alpha subunit (αENaC) in the…

ReCode Therapeutics has raised $80 million in Series A financing to support the preclinical development of its lead targeted RNA therapy for cystic fibrosis (CF). The funding also will be used to support the development of RNA therapies for primary ciliary dyskinesia (PCD), a hereditary condition that impairs the function…

A new variation of the gene-editing technology CRISPR-Cas9 can correct mutations in the CFTR gene — the genetic cause of cystic fibrosis (CF) — in stem cells from CF patients, a study shows. The new approach has the ability to correct mutations without the need to excise the affected region,…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

Amplifiers, a potential new class of therapies that modify CFTR — the protein whose defects cause cystic fibrosis (CF) — do so by stabilizing the template used for its production so the protein works better in cells and there’s more of it, a study into the mechanisms of amplifiers reported.

Girls with cystic fibrosis (CF) under the age of six have higher levels of a tiny RNA molecule, miR-885-5p, in their blood compared with boys of the same age, which could be linked to the worse clinical outcomes normally seen in women with CF, a study suggests. The study,…

Developing a way to destroy persister cells — bacterial cells that enter a dormant phase and are able to resist antibiotics — is the goal of Mehmet Orman, a professor at the University of Houston. The cells can cause recurrent lung infections in cystic fibrosis (CF) patients. “If…

A project aimed at personalized medicine based on the use of organoids, or “mini-organs,” from cystic fibrosis (CF) patients with rare mutations is on track to collect samples from 500 people with CF by the end of April, Proteostasis Therapeutics announced. The organoids will be used as a…

Cystic fibrosis carriers — individuals who have certain genetic mutations in one of the copies of the CFTR gene — have a significantly increased risk compared with healthy people for several CF-related conditions, a new study shows. The study, “Cystic fibrosis carriers are at increased risk for…

Genetic variations that lower the expression levels of a gene called RNF5 may explain why some people with cystic fibrosis (CF) are at lesser risk of developing lung infections, a study reports. The study, “Systematic genetic analysis of the MHC region reveals mechanistic underpinnings of HLA type associations with…