FDA Gives Go-Ahead for New Gene Therapy Clinical Trial in Cystic Fibrosis

Anna Osborne
2 August 2022
Clinical Trials
Gene Therapy
Stem Cells
Viral/Non-Viral Vectors
Pivotal-stage gene therapy company Krystal Biotech has announced the FDA has accepted its Investigational New Drug application for the clinical trial of the gene therapy, KB407 for cystic fibrosis (CF).

Krystal Biotech submitted its Investigational New Drug application to the FDA on July 1st 2022. The FDA’s approval will now see the Company initiate a Phase I clinical trial of KB407 in patients with CF.

Suffered by 40,000 people in the U.S. alone, CF is a genetic disease caused by mutations in the cystic fibrosis conductance regulator (CFTR) gene.

Faulty or absent CFTR protein causes mucus build-up in the lungs, pancreas, and other organs. In the lungs, this mucus build-up causes a loss of function and ultimately results in respiratory failure.

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Currently, approximately 10-15% of CF patients carry genetic mutations that are not responsive to available treatments.

KB407 has been developed to treat patients with cystic fibrosis regardless of the underlying mutation.

Hubert Chen, Senior Vice President of Clinical Development at Krystal Biotech, commented, “We are excited to advance KB407, our investigational gene therapy for patients with CF, into the clinic. It is designed to treat the root cause of the disease regardless of an individual patient’s mutation by giving the body instructions to produce its own functional protein. In addition, this IND acceptance represents an important milestone for us as it allows us to demonstrate the power of our platform to deliver genes, beyond skin cells, to respiratory cells.”


This redosable therapy is built from a modified HSV-1 vector that carries two copies of the cystic fibrosis conductance regulator (CFTR) gene to the respiratory cells, enabling the cells to then express functional copies of the CFTR protein. This restores ion and water flow into and out of secretary airway epithelial cells and preventing mucus build-up.

Krystal’s Phase I trial will see KB407 delivered to 3 cohorts of a total of 20 adult patients with CF, at ascending doses, via nebulized administration. The safety and tolerability of the nebulized gene therapy will be assessed as the study’s primary endpoint.

Lung function in the trial will be evaluated from baseline levels by forced expiratory volume in 1 second.

Source: Krystal Biotech Press Release


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