Krystal Biotech disclosed early clinical findings for a mutation‑agnostic gene therapy intended to deliver native protein to cystic fibrosis patients, and the company signaled plans to pursue a pivotal trial pathway. The data showed proof‑of‑concept for delivering therapeutic protein across CF mutations, prompting Krystal to propose a more accelerated path into registrational testing. Krystal framed the approach as mutation‑agnostic—seeking to address the broader CF population rather than single‑variant corrections—and said the data support moving directly toward a pivotal study. The company’s announcement has implications for gene therapy regulators and competitors in CF, where variant‑specific approaches have dominated development to date.