KB113 Gene Therapy Shows Tumor Regression in Pilot Study
The treatment demonstrated lesion clearance in a study of three adults living with Gorlin syndrome.
Updated on Oct. 15, 2026 in Biotech
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Krystal Biotech has reported positive results from a three-patient pilot study for KB113, an experimental dermatology therapy for Gorlin syndrome. The study observed tumor regression in patients treated with the gene therapy over a 14-week period.
Why it matters
This pilot provides early data on a gene-based approach to tumor clearance in a rare condition, marking a step toward potential non-surgical treatments for basal cell carcinoma in Gorlin syndrome patients. The findings suggest the therapy may promote immune-mediated clearance beyond the immediate injection site.
In the pilot study, patients received six doses of 108 PFU/mL KB113 at a maximum volume of 2 mL per dose. More than 50% of landmark lesions showed clinical regression greater than 50% over the 14-week treatment course.
The players
Krystal Biotech
A Pittsburgh-based biotechnology company developing gene therapy platforms for dermatological and rare diseases.
Dr. Shireen Guide
A medical professional and researcher who presented the study findings at the 2026 PeDRA conference.
The details
KB113 is a therapeutic candidate designed to drive the localized expression of interleukin-2 and interleukin-12, which are proteins that stimulate immune system activity. By increasing these cytokines, the treatment aims to promote immune-mediated tumor clearance. Histological confirmation showed complete clearance of injected lesions, with substantial regression also observed in non-injected lesions during the study.
Timeline
The Gorlin Syndrome Pilot Study treatment lasted for a 14-week period.
Dr. Shireen Guide presented the results at the PeDRA conference between October 15-17, 2026.
The Tech Race
This pilot result serves as a foundational milestone for the KB113 program as it seeks to address a condition affecting approximately 10,000 people in the U.S. Krystal Biotech is now moving to align its future clinical development plans with the FDA.
The therapy is currently in the pilot study stage and is not available for clinical use. Patients and providers should monitor future company announcements regarding FDA-aligned clinical development plans.
The takeaway
The study suggests that localized gene therapy can induce regression of multiple tumors, even those not directly targeted. Readers should watch for upcoming guidance from the FDA regarding the next phase of the KB113 development program.
Further reading
For broader trends in gene therapy development, see our Biotech section.
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Should the government prioritize rapid development of genetic therapies for rare diseases despite health risks?
