Cystetic Medicines begins Phase 1 trial of ‘molecular prosthetics’ for cystic fibrosis

Cystetic Medicines, a company that makes small-molecule channels to be used as molecular prosthetics for the cystic fibrosis transmembrane conductance regulator (CFTR) protein in people with cystic fibrosis, has dosed its first volunteer in a Phase 1 trial. In people with cystic fibrosis, the CFTR protein is faulty, scarce, or absent. Cystetic’s approach may treat the approximately 10% of those with CF who produce little to none of the protein and therefore aren’t helped by the class of drugs that can improve the function of faulty CFTR proteins, the firm says.