Irish researchers target gene editing therapy as potential EB treatment

UCD team uses novel polymer-based system to deliver corrective genes, an approach that appears to be better tolerated by patients

An award-winning Irish research team is leading the way with a novel approach to the future treatment of a rare genetic skin condition.

Endured by 300 people in Ireland, epidermolysis bullosa (EB) is caused by the absence of crucial proteins between the skin layers, meaning the skin becomes extremely fragile and blisters at the mildest touch.

Now, a UCD-based research team is turning its attention to on one such protein – known as type VII collagen – which helps keep skin layers bound together. A missing or faulty gene that codes for this protein is often a factor for people with EB.

“We’re focusing on restoring the missing type VII collagen protein,” said Dr Xianqing Wang, a postdoctoral researcher in the group which is led by Prof Wenxin Wang of the UCD Charles Institute of Dermatology.

“We are developing two types of gene therapy. In gene replacement therapy, we deliver a healthy copy of the gene into the skin cells so they can produce this collagen.

“In gene editing therapy, we correct the mutations in the faulty gene so it can work properly again.”

Dr Wang added: “Right now, gene replacement is safer and more established, but we believe gene editing is the future.

“Gene replacement therapy needs to be repeated periodically, but gene editing – which corrects the gene directly – might offer a permanent treatment.”

While current available treatments, such as Vyjuvek (approved in 2023), use viruses to deliver the corrective gene, the research team is using a novel polymer-based system – a synthetic and non-viral carrier.

“This method could be much safer than traditional viral approaches,” explained Dr Xianqing Wang.

“Our toxicity and degradation tests suggest our polymer is better tolerated by the body than even the gold-standard materials currently in clinical trials.”

This innovative work is partially funded by Debra, the national charity dedicated to EB research and patient support.

“Debra has supported Prof Wang’s research for over 15 years,” said Sinead Hickey, Head of Research at Debra.

“With limited funding, we focus on high-impact projects across different approaches. Our goal is to help deliver a real, lasting cure for EB.”

Prof Wang was awarded the 2025 NovaUCD Innovation Award in April. His intellectual property portfolio includes 12 invention disclosures to NovaUCD and he is a named inventor on over 30 patents.

He previously founded Branca Bunús in 2019, a UCD spin-out biotech company focused on developing and commercialising polymer-based gene therapies for patients suffering from genetic disorders, with a primary focus on EB.