NOVEL GENE THERAPY SHOWS PROMISE Amryt Pharma and University College Dublin unite to bring hope to ‘butterfly children’, who suffer from an incurable skin condition.
mryt Pharma has signed an agreement with University College Dublin to in-license an exciting non-viral gene therapy platform technology. It offers a potential treatment for patients with a particularly severe form of an incurable and intensely painful skin condition known as epidermolysis bullosa (EB). Severe forms of EB are life-limiting and sufferers are known as ‘butterfly children’, due to their fragile skin. People with extreme sub-types of this disease often do not survive past childhood; in other cases, they may die in their teenage years or twenties due to a severe form of skin cancer, exacerbated by the inflammation and repeated cycles of wounding and healing of their skin. There are no approved treatments for EB but finding an effective treatment for their wounds, unbearable itch, pain and infection risk, is a patient’s most urgent need. In addition, gene therapy offers hope for a potential cure in the future. I N TH E D N A Gene therapy is a medical procedure designed to treat or prevent a disease by modifying the faulty DNA in a person’s cells. Many human diseases are caused by a defect in either a single gene or set of genes; gene therapy could allow doctors to switch off a faulty or missing gene, replacing it with a correct copy or introduce a new gene into the body altogether. Recessive dystrophic epidermolysis bullosa (RDEB) is a particularly severe form of EB caused by a fault in the gene responsible for instructing cells on how to produce the ‘velcro’ that keeps the first two layers of the skin together – the epidermis and the dermis – called collagen VII.
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TR A N S FO R M ATI V E TR E ATM E NT Unlike other therapies currently in development, the novel gene therapy platform in-licensed by Amryt Pharma, AP103, uses a special polymer which is designed to be applied topically and is intended to deliver a normally functioning collagen VII gene into skin cells, with the potential to restore the healthy production of collagen in people with RDEB. In turn, this may make the skin more robust and resistant to damage, thereby improving the patients’ quality of life. “While we continue investing in our global, EASE trial for AP101 with the potential to be the first approved topical wound care product to improve the speed of healing in EB, our gene therapy platform brings a great opportunity for Amryt to play a role in leading research in the area of gene therapy, one of the most exciting and potentially transformative areas of medicine today,” said Joe Wiley, CEO of Amryt Pharma. “If successful, it has the potential to be a breakthrough for patients with RDEB and other severe, life-limiting diseases for patients and their families.” amrytpharma.com
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