Dear Mr Johnson,

I read with great interest your letter of 15 September to The Telegraph entitled My vision for a bold, thriving Britain enabled by Brexit. I was thrilled to see that you chose to highlight the potential of my research field of gene therapy on this document. Quoting your words:

The NHS is a national asset whose data banks record the dizzying range of diseases that our flesh is heir to. Freed from EU regimes – often cumbersome and hard to change – we will be able to accelerate our work on gene therapy – an infant science, now taking its first faltering steps, whose potential is gigantic. Britain is already at the forefront of this, and we can lengthen our lead.

Same as you, I firmly believe that the potential of gene therapy is enormous. The use of genetic information and tools, together with stem cells, is driving a revolution in medicine. Genetic diagnostics are tremendously powerful and are helping patients, their families and doctors with unprecedented speed. The first gene and cell therapies have been marketed for rare diseases and some cancers, and many more will follow. I would agree with you that the UK is one of the countries leading the world in this area.

However, I was dismayed when I saw that, in your opinion, gene therapy work in this country would accelerate when "freed from EU regimes". My concern increased when I saw that gene therapy was the only biomedical field discussed in your letter. Being singled out, I would have expected it to have been a carefully selected example (by you and your advisers) to illustrate your position. I was so shocked to read that you thought gene therapy work would accelerate after Brexit that I informally canvassed colleagues and other gene therapy stakeholders and asked them whether they could see any merit in your choice of gene therapy as such an example. No one I have asked could.

Mr Johnson, gene therapy technology has largely been developed through testing on rare diseases and cancer. Rare diseases are those that individually affect fewer than 1 in 2,000 people; as there are more than 6,000 rare diseases, overall it is estimated that they will affect about 7% of people – that is 3,500,000 in the UK.

The first clear-cut successes of gene therapy -literally saving lives by curing children who would have otherwise died- have been in genetic immunodeficiencies, in which the immune system cannot respond adequately to infections. These diseases are ultra-rare. There are very few patients to study and help, very few research groups working on them, and precious little resources to support the research and therapies in clinical trials. Despite all the challenges, these diseases have driven the field of gene therapy, and spearheaded interest and support across the research area.

These successes and similar work on other diseases have been sustained by international academic collaboration, mostly involving leading European countries including the UK. This research has been strongly promoted and funded by the EU (for example here and here.) 

There is no concrete basis to your argument that Brexit would accelerate work in gene therapy.

There is very significant concern across the research community regarding the impact of Brexit on research and innovation funding, as the EU is the source of a sizeable proportion of total UK funding. Exiting the EU would make UK scientists ineligible for EU research funds, unless specific mechanisms are agreed. Recent history gives no reason for optimism: E-Rare, a highly successful EU funding scheme for collaborative research in the rare disease and gene therapy area, has not been available to us because the UK refused to sign up, despite significant pressure from our scientific community and the fact that the only contribution required was funding for the UK’s own teams.

In summary, gene therapy is a prominent example of the importance of international collaboration for the successful development of novel therapies. In the past the UK has not signed up to transnational EU programmes targeting rare disease and gene therapy. There is no basis to sustain the view that gene therapy research in the UK would improve outside of the EU.

In fact, Brexit is very likely to have a negative impact on gene therapy research and on patients in the UK.

Yours faithfully,

Prof. Rafael J. Yáñez-Muñoz

Supported by Scientists for EU

Rafael J. Yáñez-Muñoz is Professor of Advanced Therapy and Director of Planning and Resources at the School of Biological Sciences, Royal Holloway, University of London, UK. Rafael received his BSc and PhD in Biochemistry and Molecular Biology from the Autonomous University of Madrid, Spain. He leads the Advanced Gene and Cell Therapy lab (AGCTlab) at Royal Holloway, where they apply these technologies to rare and common diseases. Rafael has published more than 40 papers on these topics. Rafael is a member of the Board of the British Society for Gene and Cell Therapy, and currently its Treasurer. He is also Editor-in-Chief of Gene Therapy. Rafael believes it is very important to engage with the wider society, is a trustee of the Genetic Alliance UK and organises a yearly outreach event on Rare Disease Day.