Licensing deal signed with the aim of developing a treatment for the rare and debilitating skin disorder
With the aim of developing a treatment for the rare and debilitating skin disorder - Recessive Dystrophic Epidermolysis Bullosa (EB), Irish biopharma company Amryt and Professor Wenxin Wang have signed a licensing deal. The agreement, negotiated by NovaUCD’s technology transfer team, will see Prof Wang's team and Amryt working on a novel gene therapy platform for the treatment of EB.
Professor Wenxin Wang, Dr Lara Cutlar and Dr Dezhong Zhou developed and characterised this topical therapy for RDEB which could also be established as a gene therapy platform. One of the main bottlenecks for clinical gene therapy is the lack of safe and efficient gene delivery vectors. The polymer they developed carries the gene into the skin cells and then breaks down, releasing the gene so the patient’s own cells can use it to make collagen. The fully functional collagen can then travel out of the cell to where it’s needed, giving the skin back its flexible strength.
Amryt, as a biopharma company focuses on rare ‘orphan’ diseases and the signing of the in-licence agreement with University College Dublin (UCD) could prove immeasurably beneficial to the lives of those living with a rare and debilitating skin disorder. Preliminary data suggests that this gene therapy treatment could be potentially game-changing for patients living with RDEB.