March 2018

Prof Wang on the signing of the in-licence agreement with Amryt

Wed, 7 February 18 12:57 Dr Laura Cutlar

Polymers Deliver

Recessive Dystrophic Epidermolysis Bullosa (EB) is an extremely rare condition that causes enormous suffering for affected patients. Children born with RDEB are called ‘butterfly babies’ as their skin is seen to be as delicate and fragile as a butterfly. Their gene for making collagen has a fault in it resulting in severe wounds in their skin, mouth and throat leading to blistering or skin loss at the slightest friction or knock. It is progressive and very painful.

UCD researchers Professor Wenxin Wang, Dr Lara Cutlar and Dr Dezhong Zou developed and characterised a 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.

With the aim of developing a treatment for this rare and debilitating skin disorder, Irish biopharma company Amryt and UCD have signed a licensing deal.  The agreement, negotiated by NovaUCD’s technology transfer team will see UCD and Amryt working on a novel gene therapy platform for the treatment of epidermolysis bullosa (EB).

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.

“Gene therapy has come of age in the last number of years and is being applied to multiple orphan therapeutic areas.  The HPAE polymer technology gives us a potential platform technology, with an initial topical application in EB which does not rely on the use of viral vectors for the delivery of gene therapy. If successful, this platform has the potential to be broadly applicable in other dermatological conditions and possibly beyond,” said Amryt CEO Joe Wiley.

Despite there currently being no cure for EB, there have been major strides made in research to one day offer a solution. The expertise of Amryt Pharma in bringing topical therapies to patients means they are ideally placed to take this project on.