A South Side biotech company is working to treat rare skin diseases by delivering gene therapy with a topical gel embedded with a modified version of the herpes virus.
With the gel, Krystal Biotech has successfully treated two patients with dystrophic epidermolysis bullosa, sometimes called butterfly skin.
In healthy skin, the outer and inner layers are held together by a collagen structure. In patients with the disease, this structure is absent because of a missing gene. This results in skin that sheds off easily and looks raw and red, causing pain. Patients often die of skin cancer.
"We start with a Herpes simplex virus," said CEO Krish Krishnan said. "And we modify it so that all it does is safely deliver the missing gene to the skin cells."
The patient won't get herpes; the virus just acts as a vehicle so the treatment can penetrate the cell. When someone uses the gel, the gene that creates the collagen structure enters the skin cells. This can offer relief for patients who otherwise have few options, said Krishnan.
"Functionally speaking, it's no different than applying a cream, in this case by a caregiver, dermatologist or doctor," he said.
Based on initial findings, the treatment would only need to be applied once a skin cycle. That length of time depends on the person, but tends to be about every four to five months.
Krishnan said he hopes the gel will hit the market by 2021. Krystal Biotech is also pursuing other rare skin diseases that could be treated with gene therapy.
This company is opening an in-house manufacturing facility to produce their clinical materials in Pittsburgh.