Krystal pursues mutation‑agnostic CF gene therapy: early clinical signals

Krystal Biotech disclosed early clinical proof‑of‑concept data for a mutation‑agnostic cystic fibrosis gene therapy and said it plans to move directly into a pivotal trial. The company reported the ability to deliver native protein to patients and advocates a streamlined path to registration. Clinical details released alongside the company’s development plan underscore Krystal’s push to advance a broad‑spectrum gene therapy approach in CF.