The world's first genetic gel cured children of a disease they could not overcome for years

In a Phase XNUMX trial of the world's first topical gene therapy, a topical therapy gel was confirmed to completely heal wounds in children suffering from a rare blistering skin condition that has been open for years. New Atlas.

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Dystrophic epidermolysis bullosa (DEB) is a rare skin condition that results in permanent blistering of the skin and often leaves unhealed wounds. This condition is caused by a mutation in the COL7A1 gene that prevents a person from producing a key collagen protein.

The new therapy is a gel designed to deliver working copies of the COL7A1 gene directly to wound tissue. Theoretically, this should trigger the production of key collagen proteins needed for skin healing. Functional genes are delivered through a modified herpes virus that cannot replicate and spread to other parts of the body, but is able to evade the body's immune defenses.

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According to The New England Journal of Medicine, the results of a phase 3, double-blind, placebo-controlled trial are very impressive. The patients were treated with the gene therapy gel weekly, and after three months, 71% of patients treated with the active gel had made a full recovery.

One participant in the trial, 22-year-old Vincenzo Mascoli, suffered from open wounds all his life, but within a few months of using the gene gel, his wounds healed.

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“After four months, I saw improvement in a large wound on my back that I had had for 20 years,” Mascoli said. “After six months, the wound was completely healed and much less painful.”

Unfortunately, due to the fact that the effect of the gel is not permanent, many patients have seen some of their wounds reopen several months after the completion of the trial. The researchers suggest that this is to be expected. But given the absence of side effects with repeated administration of the gel, it is possible that continued treatment could be effective for extended periods of time.

Pharmaceutical company Krystal Biotech is already working closely with the US Food and Drug Administration (FDA) to obtain marketing approval. A decision is expected sometime in the first quarter of 2023.

Although DEB is a rare disease, this specific local gene therapy is likely to be the first of many applications of the new technology. There are a number of other skin conditions of epidermolysis bullosa caused by single gene mutations that can hypothetically be treated with this technology.

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In its current form, gene therapy cannot penetrate intact skin. Thus, the gel can only treat existing wounds, not prevent them from occurring. Future research on the technology will test different delivery methods that could target different skin conditions.

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