Bercolagene telserpavec

Unassigned

New Medicines

Dominant and recessive dystrophic epidermolysis bullosa (DEB)

Information

Advanced therapy medicinal product (ATMP) (gene therapy)
Krystal Biotech
Krystal Biotech

Development and Regulatory status

None
None
Phase II Clinical Trials
Yes

Category

Herpes simplex virus-1 vector (HSV-1) engineered to deliver functional human COL7A1 genes directly to the ps’ dividing and non-dividing skin cells via a topical gel formulation. Off-the shelf gene therpy product with low immunogenicity.[2]
Population studies are complicated by the fact that this is not a uniform disease but a wide number of diseases of varying severity. It is likely that many patients with the more mild forms of EB remain undiagnosed. It is thought to affect 1 in 17,000 births. Around 5,000 people are affected in the UK [1].
Dominant and recessive dystrophic epidermolysis bullosa (DEB)
Topical