Krystal Biotech KB113 pilot: 83% of BCC lesions regressed in 3 Gorlin syndrome patients

Krystal Biotech, Inc.

Over 80% of assessed landmark lesions regressed: 69/83 (83.1%) any regression; 42/83 (50.6%) >50% reduction; 8/83 (9.6%) at least 90% reduction.
Substantial regression in non-injected lesions (40/47, 85.1%) suggests an abscopal effect; all three biopsied injected lesions showed histologically confirmed complete response.
Three adults received six intralesional doses of up to 2 mL of 10^8 PFU/mL KB113 over 14 weeks; all 22 adverse events were mild/moderate and resolved, with no serious AEs.
KB113 is the low dose of intratumoral KB707 from OPAL-1 (NCT05970497); Krystal has begun FDA discussions on advanced development for Gorlin syndrome.
Data presented by investigator Dr. Shireen Guide at the 14th PeDRA Annual Conference, Oct 15-17, 2026; US Gorlin syndrome prevalence may exceed 10,000 patients.

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