FDA Green-lights Krystal’s KB103 Gene Therapy Trial for DEB Patients

The U.S. Food and Drug Administration has cleared Krystal Biotech‘s investigational new drug application for KB103, a topical gene therapy for dystrophic epidermolysis bullosa (DEB).

The company can now start enrolling patients for a clinical trial to test the experimental therapy.

KB103 is based on the company’s STAR-D gene therapy platform, comprising a genetically engineered virus and a technology to efficiently transfer healthy genes to a variety of different types of skin cells. Krystal is using this approach to develop off-the-shelf treatments for skin diseases that currently have no effective therapies.

A virus is used to deliver a healthy COL7A1 gene to skin cells. This gene is defective in patients with DEB.

The herpes simplex virus type 1 (HSV-1) is used to transfer the healthy genes, although it has been modified so that it does not cause the virus. Because HSV-1 normally infects skin cells, it is the ideal delivery system for skin disease therapies, according to Krystal.

KB103’s formulation is designed to be applied directly onto the skin, without taking pills or receiving injections.

“We are excited about KB103 entering the clinic. Our goal is to demonstrate that KB103 could truly be a first-in-class potent treatment for DEB that has the potential to change the lives of people affected by this condition,” Suma Krishnan, founder and chief operating officer of Krystal, said in a press release.

“We plan on enrolling patients in this Phase 1/2 study as soon as we can and hope to have a meaningful data readout shortly,” Krishnan added.

The Phase 1/2 clinical trial is expected to start in May at Stanford University in California.

“Clearance of the IND by the FDA is the first of several important milestones for the KB103 program,” Krishnan said. “I am proud of the efforts of the Krystal team that has worked diligently to bring KB103 into the clinic in a timely manner.”

According to the company, KB103 is the first-ever topical HSV-1-based gene therapy created to deliver functional collagen protein to patients with DEB.