Daniela Semedo, PhD,  —

David M. Rodman is the new chief development strategy officer at ProQR Therapeutics, a leader in RNA therapies for cystic fibrosis (CF) and other diseases. Rodman, a medical doctor, will be tasked with advancing ProQR’s treatment candidates. Dr. David M. Rodman. Photo credit: ProQR A key therapy…

Canon BioMedical has developed 21 Novallele genotyping assays that can detect changes within the cystic fibrosis transmembrane conductance regulator (CFTR) — the defective gene in cystic fibrosis (CF) patients. Different types of genetic mutations in the CFTR gene cause diseases. These range from single nucleotide polymorphisms (SNPs, the most common type of…

Vertex Pharmaceuticals is acquiring CTP-656, a potentiator treatment for the protein that is defective in cystic fibrosis, from Concert Pharmaceuticals. CTP-656, a deuterium-modified version of the therapy ivacaftor, is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, meaning that it enhances the activity of the CFTR protein. CTP-656 has received…

Researchers conducting a Phase 1 study for cystic fibrosis (CF) have dosed their first healthy volunteer with a combination of the novel drugs GLPG2222 (a corrector) and GLPG2451 (a potentiator), the Belgian company Galapagos announced. The randomized, placebo-controlled study (NCT02788721) involves at least 40 healthy participants in Belgium.

Researchers are developing a compact respiratory device that will allow children whose lungs have failed from cystic fibrosis (CF) or other diseases to move about a hospital rather than having to stay in a bed during treatment or while awaiting a transplant. The Ambulatory Assist Lung for Children project…

Online vlogs — the video equivalents of web journals or blogs — can be an important platform for patients and families living with chronic diseases like cystic fibrosis (CF), a way of connecting with and learning from others in similar circumstances, researchers report. These vlogs, done by patients and addressing all aspects of life with a…

The top 10 priorities for cystic fibrosis research — set by CF patients and specialists alike — range from learning more about treatments that delay or prevent disease progression, to simplifying therapies so they’re less burdensome and getting patients to keep using a prescribed medication, according to a survey exercise led by scientists at The University…

Corbus Pharmaceuticals has completed its Phase 2 clinical trial  evaluating Resunab (JBT-101) for the treatment of adults with cystic fibrosis (CF), and expects to soon report topline results. “We look forward to having our first safety data on JBT-101 in CF,” Barbara White, MD, chief medical officer at Corbus,…

A number of important discoveries, therapeutic developments, and events related to cystic fibrosis (CF) were reported daily by Cystic Fibrosis News Today throughout 2016. Now that the year is over, it is time to briefly review the articles that appealed most to our readers. Here are the top 10 most-read…

Galapagos NV recently reported positive topline results from SAPHIRA 1, a Phase 2 clinical trial assessing the company’s potentiator molecule, GLPG1837, in patients with cystic fibrosis (CF) who have the G551D mutation. Data showed results comparable to an approved CF treatment, the company announced. SAPHIRA 1 (NCT02707562) was an…