Alice Melão, MSc,  —

Articles by Alice Melão

ProQR Completes Phase 1b Trial of Inhaled Therapy for CF Patients with F508del CFTR Mutation

ProQR Therapeutics has finished a dose-escalating Phase 1b clinical trial evaluating the safety, stability and distribution throughout the body of its investigational therapy QR-010 in cystic fibrosis (CF) patients with a confirmed F508del CFTR gene mutation. Interim top-line results should be released by late September. A missing amino acid in the 508 position of the…

Two of Proteostasis’ Three Cystic Fibrosis Therapies Are in Clinical Trials, and the Third Is Heading for Trials

Two of the three therapies that Proteostasis Therapeutics has developed to correct the underlying problem in cystic fibrosis are going through clinical trials and a third is heading for trials, the company reports. That underlying problem is a mutation in a gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR. It produces…

Phase 3 Study of Sollpura, Enzyme Replacement Therapy for CF Patients with EPI, Recruiting in Europe and US

A Phase 3 study of Sollpura (liprotamase), a non-porcine enzyme replacement therapy aiming to treat exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF) better than existing porcine-derived therapies, is now recruiting about 150 pediatric and adult patients in the United States, Europe and Israel. Specifically, the expanded trial will evaluate the efficacy — or non-inferiority — of Sollpura compared…