Krystal Biotech disclosed early clinical data suggesting its mutation-agnostic approach can deliver native protein to cystic fibrosis patients and signaled plans to move directly into a pivotal trial. The company presented results that it says demonstrate proof of concept for its gene therapy modality. BioCentury’s trial round-up and Krystal statements indicate the company is seeking an expedited regulatory pathway based on early efficacy signals and a mutation-agnostic mechanism. The approach aims to reach patients beyond mutation‑specific modulators in a large CF population. Investors and competitors will track the design and endpoints of any pivotal move, along with safety data and manufacturability, to judge whether Krystal’s strategy could change the CF therapeutic landscape.