Krystal Biotech disclosed early clinical data suggesting its mutation‑agnostic approach can deliver native CFTR protein to cystic fibrosis patients, and the company signaled plans to advance directly toward a pivotal trial. Company statements and a clinical report outlined proof‑of‑concept signals that Krystal believes support accelerated development. The filings and clinical report have reignited debate over mutation‑agnostic gene therapies for CF, particularly around regulatory pathways, manufacturing scale and how native protein delivery compares with existing modulator and gene‑editing strategies.