Amryt announces a new platform technology for gene therapy

Amryt announces the in-licencing of a new platform technology for gene therapy with potential applicability across a range of genetic disorders

The technology has been exclusively in-licenced from University College Dublin and involves the delivery of gene therapy using High Branched Poly (β-Amino Ester) (“HPAE”) polymer technology. The initial focus of development efforts to date has been in the area of EB and preliminary data suggests that the treatment could be potentially disease-modifying for patients with Recessive Dystrophic Epidermolysis Bullosa. Pre-clinical data in a xenograft model has shown significant levels of collagen VII in the skin post therapy.  Patients with RDEB have a defect in their gene coding for collagen VII, consequently the replacement of collagen VII could be transformative for these patients.

Potential competitors working in the area of gene therapy in EB are working with viral vectors to deliver collagen VII to the cell. The patented technology which Amryt has exclusively licenced from UCD involves the use of a novel gene delivery mechanism using HPAE polymer technology. 

Amryt intends to conduct various pre-clinical studies in the coming months and will report initial results in early Q4 2018.

Commenting on the new in-licence agreement, Joe Wiley, CEO of Amryt, said: “This is a great opportunity for Amryt to get involved in the area of gene therapy, which is one of the most exciting and potentially transformative areas of medicine today. Gene therapy has come of age in the last number of years and is being applied to multiple orphan therapeutic areas.”