Krystal Biotech disclosed early clinical data indicating its gene therapy can deliver native protein to cystic fibrosis patients, and the company proposed advancing directly into a pivotal trial. Krystal described the therapy as mutation‑agnostic and presented clinical observations that supported a proof‑of‑concept claim. Management is seeking regulatory engagement to expedite pivotal development. The firm’s data suggest meaningful target engagement in treated subjects; Krystal argues that a mutation‑agnostic approach could broaden patient eligibility compared with mutation‑specific gene edits or modulators. The company’s pivot toward a pivotal path raises questions about trial design, endpoints, and the regulatory standard for confirmatory evidence in gene-replacement strategies. If regulators accept Krystal’s pivotal proposal, the program could compress timelines for bringing a broadly applicable CF gene therapy into late‑stage testing—an outcome that would reshape competitive positioning in cystic fibrosis therapeutics.