The gene therapy market - France (2023)

1.1 Definition and scope of study

The therapy gene has a simple goal, to cure rare diseases by changing genes. The principle of gene therapy consists of inserting modified genes via a vector into a diseased cell in order to modify its functioning. The reality is more complex and the processes are multiple. Its aim is to deal mainly with genetic diseases, but also with new diseases, cancers and cardiovascular diseases.

Gene therapy drugs are part of the MTI (advanced therapy medicinal products) is a medicinal product manufactured from cells, or human genetic material, to treat cellular or genetic abnormalities.

The market demands investments colossal for results that are still very random. Although a first drug based on gene therapy was first developed in China in 2003, it was not until 2011 that a Dutch company became the first to offer such a drug in the West. On the other hand, the development possibilities are very promising because research is progressing. The United States is unquestionably the market leader, with some 15 companies, each listed at several billion dollars. Indeed, in a sector where investment is crucial, the major resources deployed by the United States give it a leading position

In the area of rare diseases gene therapy is relatively new. The first trials only date back to the 1990s. For a long time, these diseases did not arouse the interest of laboratories because they affect only 5 people out of 10,000 . Today, the stakes are high and many laboratories are working on these issues. Thus, competition is becoming more structured, but still remains weak because of the numerous fields of application and ethical controls.

A former pioneer with the pastoral institute, the France is a promising country of gene therapy in Europe. Various ITMs are currently being developed by companies such as Cellectis, Genethon, Gensight and Horama in private or public production sites (AP-HP, EFS). Today, France seems to be back in the race: the State has emphasized the importance of regaining a leading position in the sector, because the country that develops an ITM, has a good chance of producing it and therefore putting it on the market. The risk for France is that it will not be able to develop its ITNs quickly enough, and that in a few years' time it will be dependent on ITNs from the United States.

Numerous research labs are therefore publicly funded . In the absence of direct economic spin-offs, there are few companies and public-private synergies are frequent. The first treatment marketed in Europe by Uniqure has an estimated cost per patient of more than one million euros. Faced with such a sum, research and human tests can only be financed by governments, or by partnerships with private companies (often foreign)