Gene therapy is a transformative field of medicine that could halt diseases before patients become symptomatic, saving lives and improving quality of life for millions of people who suffer from conditions caused by genetic abnormalities1. But these breakthrough treatments require new approaches to pricing and reimbursement2.
While the science behind gene therapy has existed for decades, the first generation of treatments is now emerging for debilitating conditions including spinal muscular atrophy3 and inherited retinal diseases4 5. Although Europe was at the forefront6 of science a decade ago, other regions have invested heavily in research and Europe has work to do to re-gain7 its leadership role.
A cell is the fundamental biological building block of all living things8. Genes, found within cells, are small sections of DNA that carry the cell’s genetic information. Genes are the cell’s blueprint for making proteins that support functions such as muscle strength, and mutations in genes contribute to a range of diseases including those caused by recessive gene disorders, like hemophilia and sickle cell anemia, acquired genetic diseases such as certain cancers, and some viral infections like AIDS9.
Gene therapies improve and/or replace mutated genes, most often via the technique of recombinant DNA technology in which a molecular ‘carrier’ known as a vector is used to carry a healthy copy of the gene and introduce the new genetic material into the cell11. Most commonly, gene therapy allows cells to add an additional genetic unit, but new engineering techniques now exist to switch off, allow modification to or correct the problematic DNA sequence12.
Europe’s gene therapy leadership
Academic researchers, biotech and pharmaceutical companies in Europe have been instrumental in developing the field of gene therapy13. Between 2017 and 2019, the lead author of 120,000 papers in cell and gene therapy (CGT) publications was affiliated with a European institution, compared to 72,000 and 100,000 in the U.S. and China respectively14. Professor Emmanuelle Charpentier at the Max Planck Unit for the Science of Pathogens in Berlin was a joint winner of the Nobel Prize in Chemistry 2020, a recognition of her contribution to the development of tools for gene editing15.
The European Union’s medical regulatory institution, the European Medicines Agency (EMA), has been a world leader in assessing advanced therapy medicinal products (ATMPs), a class of innovative biological products of which gene therapy is part, approving a tissue-engineering product in 2009, a year before the U.S.’s first ATMP approval16. It also approved the world’s first gene therapy in 201216. The EMA has granted orphan drug status to the majority of CGT drugs currently in development and performed accelerated assessments for a number of cell and gene therapies14.
But the region needs to build on its momentum to take gene therapy from the lab to patients. The total number of gene-, cell- and tissue-based therapeutic developers worldwide lags behind the North American and Asia-Pacific regions17.