US-based biotechnology company Krystal Biotech has dosed the first patient in its Phase I CORAL-1/US clinical trial of KB407, an investigational gene therapy designed to treat cystic fibrosis (CF).
KB407 is an engineered HSV-1-based, mutation-agnostic medicine that corrects the lung manifestations of CF by enabling the expression of full-length, healthy CFTR protein.
The multicentre dose-escalation trial intends to enrol nearly 20 patients with CF regardless of their underlying genotype. They will be divided into three cohorts.
Five participants in the first cohort will receive one dose of KB407 on day zero, while the same number of participants in the second cohort will receive KB407 on days zero and one.
The remaining ten participants in the third cohort will be randomised at a 4:1 ratio to receive either KB407 from day zero until day three or placebo.
Subjects who are not otherwise eligible for CFTR modulators are enrolled in the last cohort.
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By GlobalDataThe trial’s primary endpoint is to assess the safety and tolerability of nebulised KB407. It will also evaluate biodistribution and vector shedding in samples of sputum, buccal, blood and urine.
In addition, a bronchoscopy sub-study will be carried out to assess CFTR transgene expression in the airways at both the nucleic acid and protein levels.
The first patient was treated at the Cystic Fibrosis Institute of Chicago. Subjects can also undergo an optional bronchoscopy 24 to 96 hours after the last dose of the therapy at certain trial sites.
Krystal Biotech Clinical Development senior vice-president Hubert Chen said: “Not only is the Phase I study designed to provide key insights into the safety and efficacy of KB407, it is also a critical step in expanding our vector platform to tissues beyond the skin.
“We look forward to continued enrolment into the Phase I study with anticipated data in 2024.”
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