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Biotechnology: Biological Products (No Diagnostic Substances)
Clinical TrialOct 15, 2026, 07:06 AM

Krystal Biotech: KB113 Shows Over 80% Lesion Regression in Gorlin Syndrome Study

AI Summary

Krystal Biotech announced positive clinical data from its Gorlin Syndrome Pilot Study evaluating KB113 for basal cell carcinomas. The study showed over 80% of assessed lesions regressed, including non-injected lesions, indicating a potential abscopal effect. All injected lesions achieved complete clearance histologically. KB113 was well-tolerated with mild, transient adverse events. The company is discussing further clinical development with the FDA.

Key Highlights

  • Over 80% of assessed basal cell carcinoma (BCC) lesions regressed in three Gorlin syndrome patients treated with KB113.
  • Substantial regression observed in non-injected lesions, suggesting an abscopal effect.
  • Complete clearance of injected lesions confirmed histologically for all three patients.
  • KB113 injections were well tolerated, with all adverse events being mild to moderate and transient.
  • Data presented at the 14th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference.
  • Krystal Biotech is discussing advanced clinical development plans with the FDA for KB113.