Beremagene geperpavec for dystrophic epidermolysis bullosa

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Therapeutic Areas: Dermatology
Year: 2021

Dystrophic epidermolysis bullosa (DEB) is caused by a mutation in a collagen gene that controls skin strength and stability. This mutation can be inherited from one or both parents, which determines the severity of the condition (those who inherit the mutation from both parents have more severe symptoms). DEB symptoms develop from birth or early childhood and is characterised by painful skin blisters and scarring. Some people with DEB can have minor blisters mainly at areas that experience trauma such as hands, feet, arms and legs, whereas other people can have blistering externally and internally, which can cause many associated issues. Most people with DEB have blistering around and inside the mouth that affects eating and dental hygiene.
Beremagene geperpavec topical gel aims to treat DEB by delivering normal, functional collagen genes to the affected skin cells, by doing so this promotes wound-healing and reduces scarring. If licenced, beremagene geperpavec will offer the first DEB-specific treatment that addresses the underlying cause of the disease for paediatric and adult patients.