KB407 CF gene therapy data: human CFTR expression 106–440% of endogenous in NHPs; IND accepted

Krystal Biotech, Inc.

KB407 transduced ciliated, club, and goblet cells in NHP airways; no transduction of basal cells.
Human CFTR transcript levels reached 106–440% of endogenous NHP CFTR in lung biopsies.
No adverse findings in GLP toxicology study; NOAEL at highest dose tested.
FDA accepted KB407 IND in July 2022.
No significant cytokine induction in human CF cells after KB407 transduction.

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