News

Kaftrio Rescues Chloride, Bicarbonate Deficits in GI Tissues

The triple combination therapy Kaftrio restores the function of CFTR — the defective protein in cystic fibrosis (CF) — and associated chloride and bicarbonate flow in gastrointestinal “mini-organs” derived from a patient carrying the most common CF-causing mutation, a study shows. These findings highlight the benefits of Vertex…

Partial Monocyte Transplant Improves Survival in Mouse Study

Monocyte immune cells from bone marrow partially transplanted into a mouse model of cystic fibrosis (CF) improved survival and reduced inflammation, a study found. Meanwhile, transplanting monocytes from CF mice into healthy mice triggered CF-like symptoms and limited survival, suggesting that these defective immune cells may cause symptoms, the…

New Capsule Form of Adrulipase Alfa for EPI May Soon Enter Trial

First Wave BioPharma has filed two new patents related to adrulipase, a yeast-derived enzyme that the company is developing to treat exocrine pancreatic insufficiency in people with cystic fibrosis (CF). One application is related to dosage forms, methods of treatment, and manufacturing methods for the investigational medication. This includes a…