KRYS - Krystal gets FDA nod to start trial of gene therapy KB407 for cystic fibrosis
- Krystal Biotech ( NASDAQ: KRYS ) said the U.S. Food and Drug Administration (FDA) accepted its investigational new drug application to start a trial of its gene therapy KB407 for cystic fibrosis (CF), a type of lung disorder.
- The company noted that KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs.
- The phase 1 trial, which is expected to start n H2 2022, will utilize nebulized administration to deliver KB407 in up to 20 adults with CF, according to the company's Aug. 1 press release.
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Krystal gets FDA nod to start trial of gene therapy KB407 for cystic fibrosis