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Krystal Biotech Initiates Phase 1 Clinical Trial of KB407 for Cystic Fibrosis Treatment, Administering First Dose to Patient

Monday, July 03, 2023

Krystal Biotech, a biotechnology company listed on NASDAQ as KRYS, has announced a significant milestone in their research efforts. They have administered the first dose of their experimental treatment KB407 in a Phase 1 clinical trial at the Cystic Fibrosis Institute of Chicago. The trial, known as CORAL-1/US, aims to evaluate the efficacy of KB407, an engineered HSV-1-based genetic medicine delivered via aerosol, for the treatment of cystic fibrosis (CF).

The potential of KB407 lies in its ability to address the underlying genetic defect in CF by delivering full-length copies of the CFTR gene directly to the lungs through nebulization. Unlike other therapies, KB407 is designed to be mutation-agnostic, which means it may offer benefits to all individuals affected by cystic fibrosis, regardless of their specific CFTR mutation.

The CORAL-1/US study is a dose-escalation trial being conducted at multiple centers and will involve approximately 20 CF patients. Each administration of KB407 will be nebulized in under 30 minutes. The study consists of three cohorts, with the first two cohorts enrolling five subjects each and the final cohort enrolling ten subjects.

Cystic fibrosis is a genetic disease caused by mutations in the CFTR gene, resulting in the production of dysfunctional or absent CFTR protein. This leads to the accumulation of mucus in the lungs, persistent lung infections, and progressive pulmonary disease. The Cystic Fibrosis Foundation estimates that there are nearly 40,000 children and adults living with CF in the US, and over 105,000 diagnosed cases worldwide across 94 countries.

KB407 has received orphan drug designation from both the US Food and Drug Administration (FDA) and the European Commission for the treatment of cystic fibrosis, highlighting the potential therapeutic value of this investigational gene therapy.



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