U.S. company Krystal Biotech is set to start a clinical trial for cystic fibrosis after the United States Food and Drug Administration (FDA) accepted its investigational new drug (IND) application.
The gene therapy company will be evaluating KB407, a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.
By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has the potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation.
“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,” said Hubert Chen, senior vice president of clinical development at Krystal Biotech.
“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,
The post FDA gives ok to Krystal Biotech cystic fibrosis clinical trial appeared first on Labiotech.eu.
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