The Food and Drug Administration (FDA) has approved the first topical gene therapy, developed to treat a rare skin condition known as butterfly children.

A Novel Gene Replacement Treatment

Pittsburgh startup company Krystal Biotech developed the pioneer drug called Vyjuvek. It is applied topically as a skin ointment and as an eye drop which needs to be constantly reapplied every week to promote the turnover of skin cells.

As a gene-replacement treatment, it introduces a missing gene to skin cells to enable them to produce the collagen needed to hold together the layers of the skin. In a trial study conducted in 2022, the ointment was applied to the most severe wounds of participants composed of 31 volunteers. The wounds were cleared during the treatment, while others shrunk.

The novel delivery strategy showed promising results in the reduction of wound size and the improvement of vision in patients. The approval of Vyjuvek served as an important breakthrough in gene therapy because it is the first treatment method developed for repeated use on the same person.

The Food and Drug Administration has approved using this topical medication in patients older than six months. Since 2017, the Food and Drug Administration has approved five gene therapies for rare inherited diseases. Before Vyjuvek was developed, earlier treatment methods were delivered by injecting or altering the immune cells outside the patient's body. According to company CEO Krish Krishnan, formulating gene therapy in a topical medication provides a convenient, simple, and patient-friendly approach to supplying the missing genes.

In the future, gene replacement for the skin could have valuable applications in the cosmetic industry. Gene-based aesthetics company Jeune, a subsidiary built by Krishna,, started developing another version of the topical medication. It aims to reverse the formation of crow's feet and other facial wrinkles which appear when a person's body makes less collagen in their skin. If this project succeeds, it could pave the way to the future of gene therapy where the drug is recommended for non-medical enhancement.


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Causes and Symptoms of Butterfly Children

Butterfly children is an informal name given to the dystrophic epidermolysis bullosa skin condition. It is a rare inherited medical condition where the skin becomes so fragile that it tears or produces a blister at the slightest touch. People who acquire this disease suffer from wound blisters all over their bodies, including the throats and eyes. Children born with this disease are often called "butterfly children" because their skin resembles a fragile butterfly wing.

Mild forms of butterfly children can get better over time. However, some cases are too severe and can lead to pain or trigger other serious medical issues or even death.

Dystrophic epidermolysis bullosa is just one of the five types of epidermolysis bullosa,, categorized according to the area where the blisters form. The other four types include Junctional epidermolysis bullosa, Kindler syndrome, Epidermolysis Bullosa Simplex, and Epidermolysis bullosa acquisita.

These types of epidermolysis bullosa are inherited except for epidermolysis bullosa aquisita since it only happens as a result of immune systems malfunction.

 

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