UK biotech Amryt has secured itself access to a novel gene therapy platform that could give rise to a new treatment option for patients with Epidermolysis bullosa (EB), a group of rare inherited skin disorders that cause the skin to become very fragile.
The company has signed an agreement with University College Dublin for a non-viral gene therapy platform technology, on the back of early data showing that the treatment could be disease-modifying for patients with the Recessive Dystrophic forms of EB.
Financial details were not disclosed, but Amryt said it intends to conduct various preclinical studies in the coming months and will report initial results in early during the fourth quarter.
“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,” noted chief executive Joe Wiley.
The deal “gives us a potential platform technology, with an initial topical application in EB, that 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.”
In parallel, the firm said that Amicus Therapeutics is allowing it access to data from its landmark Phase III clinical study in EB, which will enable Amryt to refine its own ongoing global Phase study assessing AP101 for the condition.
The move has the potential to “increase the probability of success for the study,” Wiley noted, adding: “We are truly grateful to Amicus Therapeutics for the opportunity, which is a heartening example of collaboration in our industry in the best interests of patients.”