Krystal Biotech, Inc. ((KRYS)) announced an update on their ongoing clinical study.
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Krystal Biotech, Inc. is conducting a Phase 1 clinical study titled KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis. The study aims to evaluate the safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis, a significant step in addressing this chronic genetic condition.
The intervention being tested is KB407, a nebulized biological treatment designed to deliver a replication-defective HSV-1 vector expressing the full-length human CFTR gene. This approach seeks to address the underlying cause of cystic fibrosis by targeting the defective CFTR protein.
The study is interventional with a non-randomized, sequential model and no masking. Its primary purpose is treatment, focusing on the administration of KB407 in increasing doses across three open-label cohorts.
The study began on August 15, 2022, with a primary completion date yet to be determined. The latest update was submitted on August 19, 2025, indicating ongoing recruitment and progress.
This update could positively influence Krystal Biotech’s stock performance by showcasing their commitment to innovative treatments for cystic fibrosis. Investors may view this as a promising development, potentially boosting confidence in the company’s pipeline. The competitive landscape includes other biotech firms exploring gene therapy solutions, highlighting the importance of Krystal’s advancements.
The study remains active, with further details available on the ClinicalTrials portal.