Krystal Biotech disclosed that its inhaled gene‑therapy program KB407 achieved a landmark interim milestone, with molecular confirmation of wild‑type CFTR protein expression in the lungs of patients carrying null mutations. The data, derived from the CORAL‑1 study, show transduction rates ranging from 29.4 % to 42.1 % across seven patients who received four daily doses of 10^9 PFU of KB407, followed by bronchoscopies that confirmed full‑length CFTR expression in ciliated and secretory cells.
The CORAL‑1 cohort‑3 results demonstrate that the HSV‑1‑based vector can deliver the full‑length CFTR payload safely and efficiently, with no serious adverse events reported. The study’s design—four daily inhalations, a 10^9 PFU dose, and post‑dose bronchoscopic sampling—provides robust evidence that the therapy can reach and transduce the target cells in the human lung, a critical step toward a mutation‑agnostic treatment for cystic fibrosis.
This clinical success validates Krystal’s proprietary platform and supports the company’s strategy to expand beyond dermatology into high‑value respiratory indications. The data have propelled the program into the next development phase, with a repeat‑dosing study (CORAL‑3) already submitted to the FDA and expected to begin enrollment in the first half of 2026. A mutation‑agnostic therapy could address more than 90 % of the cystic fibrosis population, representing a market opportunity estimated at over $2 billion.
Management emphasized the significance of the findings. CEO Krish Krishnan said, “We have now confirmed the successful delivery and expression of wild‑type CFTR protein in multiple patients with cystic fibrosis, a breakthrough that positions KB407 on an accelerated path toward a potential registrational data readout.” R&D President Suma Krishnan added, “The data give us the conviction to move rapidly into repeat dosing.” Professor Jorge Lascano noted, “High rates of KB407 transduction and broad distribution across patient airways underscore the transformative potential of KB407 as a mutation‑agnostic therapy.”
Analysts reacted positively; Jefferies raised its price target for Krystal to $310 from $252, citing the strong transduction rates and clean safety profile as key drivers. The company’s stock was trading near its 52‑week high at the time of the announcement, reflecting investor confidence in the platform’s potential to generate substantial future revenue.
Krystal’s financial foundation remains solid, with its commercial product VYJUVEK for epidermolysis bullosa generating strong revenue and margins. The positive KB407 data reinforce the company’s trajectory, suggesting that the HSV‑1 platform could become a versatile tool for treating a range of genetic diseases beyond dermatology.
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