Krystal Biotech said that the FDA has cleared an IND for KB408 nebulized gene therapy for the treatment of alpha-1 antitrypsin deficiency, and the company said that it plans to initiate a Phase 1 trial in early 2024. According to Krystal, the study will evaluate 3 ascending doses of KB408 in adult patients with AAT deficiency who have a PI*ZZ genotype.
KB408 is based on a vector derived from HSV-1 that carries 2 copies of the SERPINA1 gene, which encodes for AAT. The FDA granted orphan drug designation to KB408 for AAT deficiency earlier this month.
Krystal Biotech Senior VP of Clinical Development Hubert Chen commented, “We are excited to advance KB408, our investigational gene therapy for patients with alpha-1 antitrypsin deficiency, into the clinic in our Serpentine-1 study. This IND acceptance represents an important milestone for us as we work to address a serious lung disease with limited treatment options, and also allows us to demonstrate the potential of our platform to deliver genes repeatedly to epithelial cells of the lung.”
Read the Krystal Biotech press release.
