The impact on Advanced Therapy Medicinal Products (ATMPs) is one of the key emerging flashpoints of European Commission proposals to revise the EU’s pharmaceutical legislation which were tabled in April last year. ATMPs are medicines for human use based on genes, tissues or cells, and are mainly used to treat rare diseases, 80% of which are genetic and can be targeted by these innovative treatments. Although ATMPs have transformed treatment of rare diseases, high production and research costs make them inaccessible for many patients. Of 18 gene therapies authorised for EU use in recent years, …