LONDON (Alliance News) - Amryt Pharma PLC on Monday reported positive results from two pre-clinical studies of its novel non-viral gene therapy for recessive dystrophic epidermolysis bullosa.
The company said that the studies of AP103 showed that the therapy restored production of collagen VII in pre-clinical models of the disease.
Dystrophic epidermolysis bullosa is caused by mutations in a single gene, COL7A1, which codes for the production of collagen VII, a structural protein vital for the elastic and structural integrity of the skin.
"Restoring production of collagen VII in skin cells could be transformative for these patients, potentially making their skin less fragile and more resistant to damage and blistering. As a result, the quality of life for patients with RDEB could be dramatically improved," the company explained.
Amryt shares were trading down 1.4% at 16.86 pence each.
