Krystal Biotech’s Topical KB103 Gene Therapy: A Phase I/II Study Currently Enrolling RDEB Patients

About the Study:
Krystal Biotech is developing a topical gene therapy called KB103 which is currently being evaluated in an ongoing Phase I/II, randomized, intra-subject, placebo-controlled clinical trial. KB103 is a non-replicating virus modified to make the collagen VII (COL7) protein. It is formulated as a gel and applied topically to wounds. When KB103 is applied to wounds it is designed to introduce genetic material to skin cells which then enables cells to make functional COL7. The introduction of functional COL7 is anticipated to improve wound closure and the duration of wound healing.

Clinical and genetic diagnosis (confirmed COL7A1 mutation) for RDEB are among the requirements for entry. Upon entry, target wounds will be randomized to receive either KB103 or placebo and compared throughout the study. The time on study is approximately 4 months.  The study will evaluate if KB103 can safely and effectively restore functional COL7 expression in patient skin and promote healing of wounds.

Trial Entry Criteria:

  • Participants must be ages 5 years and older (Child, Adult, Older Adult)
  • Please visit https://clinicaltrials.gov/ct2/show/NCT03536143 for all inclusion and exclusion criteria.

Costs for travel and food are reimbursed. For more information about the study please click here or contact us by email at patientadvocacy [at] krystalbio [dot] com or by phone at 434-409-0176.


Listen to Suma M. Krishnan, Founder and COO, Krystal Biotech discusses their approach to treating rare genetic skin diseases that lack any truly effective treatments. Krystal is developing the first ever topically applied gene therapy for the rare disease epidermolysis, a genetic condition characterized by extremely fragile skin that leads to devastating physical developments. Listen here.