The U.S. Food and Drug Administration (FDA) granted Platform Technology Designation to the genetically modified, non-replicating herpes simplex virus type 1 (HSV-1) viral vector used in the pipeline of Krystal Biotech, Inc. This designation specifically highlighted the vector's inclusion in the company's redosable eye drop gene therapy, KB801, which is currently undergoing a randomized placebo-controlled trial for the treatment of neurotrophic keratitis (NK).

This designation was described by Suma Krishnan, President of Research and Development, as a "tremendous milestone" that recognizes the reproducibility and scalability of the HSV-1 gene delivery platform and offers significant potential for accelerating the development of Krystal's genetic medicines pipeline, starting with KB801.

The FDA’s platform technology designation program is intended to provide efficiencies in drug development, manufacturing, and the review process by allowing a company to leverage its prior experience, such as the manufacturing and nonclinical safety data from a previously approved product using the same platform—in this case, Krystal’s VYJUVEK® (beremagene geperpavec-svdt). For a technology to be eligible, it must be well-understood, reproducible, used in an FDA-approved drug, and have the potential to support the development of multiple drugs without compromising quality or safety while bringing significant efficiencies to both the development and FDA review processes.

KB801, the specific therapy mentioned, is designed to enable sustained, localized expression and secretion of nerve growth factor (NGF) by epithelial cells in the front of the eye for NK, a rare, degenerative corneal disease. By allowing cells to produce NGF locally, KB801 aims to maintain more consistent NGF levels and significantly reduce the burden for patients, who currently rely on intensive administration of existing recombinant NGF eye drops six times a day, with the safety and efficacy of the gene therapy currently under evaluation in the Phase 1/2 EMERALD-1 study.

Krystal Biotech is a commercial-stage biotechnology company focused on genetic medicines, with VYJUVEK being its first commercial gene therapy product approved across multiple global jurisdictions.