the US Food and Drug Administration (FDA) has granted clearance to Krystal Biotech’s Investigational New Drug (IND) application for KB408, a potential gene therapy for alpha-1 antitrypsin deficiency (AATD), a rare genetic disease. Submitted in August, the application sought approval for the commencement of a Phase I clinical trial for KB408.
After conducting a thorough 30-day review of the application, the FDA has notified Krystal Biotech of the successful clearance of its IND.
Hubert Chen, Senior Vice President of Clinical Development at Krystal Biotech, expressed excitement about advancing KB408 into clinical trials, specifically the Serpentine-1 study. He emphasized that this milestone is crucial as it aims to address a severe lung disease that currently has limited treatment options. Moreover, it allows them to demonstrate their gene delivery platform’s potential in treating lung epithelial cells.
Krystal Biotech is set to administer the first dose to a participant in the trial during the first quarter of the upcoming year. The study is designed as a single-dose escalation, open-label investigation, aiming to evaluate the pharmacodynamics and safety of KB408 in adults with AATD who have a PIZZ genotype.*
The trial will further assess the efficacy, safety, and tolerability of three different dosages of KB408 in each cohort, consisting of three patients each.
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