- Orphan drug company Amryt revealed 'positive' results from two pre-clinical studies of a drug that could potentially treat patients with recessive dystrophic epidermolysis bullosa.

The pre-clinical studies sought to investigate the potential of the company's non-viral gene therapy AP103 as a topical gene therapy intervention to restore expression of the COL7A1 gene.

The COL7A1 gene codes for the production of collagen VII, a structural protein vital for the elastic and structural integrity of the skin, the company said.

In vitro tests on recessive dystrophic epidermolysis bullosa keratinocytes showed that a single delivery of the human collagen VII gene, by AP103, restored collagen VII production to levels exceeding those produced by healthy human keratinocytes, the company said.

AP103 exhibited no evidence of cellular toxicity after repeated administration, the company added.

'These results further support the potential of our novel gene therapy platform as a potentially transformative treatment for patients with RDEB, which is a particularly severe form of EB. If successful, this platform has potential in other genetic skin conditions and beyond, said Joe Wiley, CEO of Amryt Pharma.

'Unlike other gene therapies that rely on viral vectors, AP103 is a novel synthetic delivery technology, which provides a potential competitive advantage in the gene therapy field.'

At 8:15am: [LON:AMYT] Amryt Pharma share price was +0.05p at 17.15p

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