Forest Ray PhD,  —

Spirovant Sciences‘ lead gene therapy candidate for select cystic fibrosis (CF) patients, SPIRO-2101, was given rare pediatric disease and orphan drug designations by the U.S. Food and Drug Administration (FDA) to support its development, according to a recent press release. Orphan drug designation applies to medicines targeting…

The airways of cystic fibrosis (CF) patients show unusual levels of an antibody called BPI-ANCA, and they correlate with the amount of Pseudomonas aeruginosa bacteria found in their sputum, according to a recent study. Evidence of both these antibodies and this bacteria in…

Moderna Therapeutics and Vertex Pharmaceuticals have entered a three-year research and licensing agreement aimed at developing gene-editing delivery techniques for the treatment of cystic fibrosis (CF). While Moderna will focus on ways to deliver gene-editing therapies to lung cells, Vertex will concentrate on other gene-editing components —…

The U.S. Food and Drug Administration (FDA) has agreed to simplify Aridis Pharmaceuticals‘ planned Phase 2 trial of AR-501, an investigational inhaled treatment for chronic lung infections in patients with cystic fibrosis (CF). The decision was based on positive safety data from a Phase 1 trial in…

Savara Pharmaceuticals is stopping its Phase 2a exploratory clinical study of Molgradex (molgramostim nebulizer solution) to treat nontuberculous mycobacterial (NTM) lung infection in people with cystic fibrosis (CF), the company announced in a press release. The decision to discontinue the open-label ENCORE trial (NCT03597347) stems from an inability to achieve the study’s primary outcome. That goal…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

People with cystic fibrosis (CF) in the U.K. have grown more numerous, older, and more diverse, according to the U.K. Cystic Fibrosis Registry’s annual data report for 2019. The report, meant to present a snapshot of both the gains as well as the challenges still faced by…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…

Poorer oxygen uptake and transport may underlie skeletal muscle differences that affect exercise tolerance in children and adolescents with cystic fibrosis (CF) relative to healthy peers, a recent study suggests. The study, “Tissue oxygenation in peripheral muscles and functional capacity in cystic fibrosis: A cross‐sectional study,”…

Enterprise Therapeutics announced that dosing has begun in a Phase 1 clinical trial testing ETD002, a potential inhaled treatment for cystic fibrosis (CF). The study (NCT04488705) will assess the safety and tolerability of EDT002 in ascending single and multiple doses in healthy people as compared with a matching placebo.