Krystal Biotech disclosed early clinical proof‑of‑concept data for a mutation‑agnostic gene therapy approach in cystic fibrosis and said it plans to pursue a pivotal development path. The company presented clinical updates indicating native protein delivery to CF patients and proposed a direct route toward registrational trials based on observed functional effects. Krystal’s announcement follows an industry trend of mutation‑agnostic strategies in CF and highlights regulatory and development choices around moving from early efficacy signals into pivotal designs. Management framed the data as supportive for accelerated development but noted additional confirmatory data and regulatory engagement remain critical next steps.