The key facts about the bioproducion sector

👉 Crigler-Najjar: hope held back by regulation 

Despite promising clinical results, many gene therapies may never reach patients in France. In an investigation, Le Point cites the example of a treatment for Crigler-Najjar syndrome, an ultrarare disease that affects around one in a million births. This gene therapy, developed by Genethon researchers, uses a single injection to replace the defective gene responsible for an accumulation of toxic bilirubin. However, despite its clinical success, a regulatory and financial headache is casting doubt on its market launch. In particular, the HAS evaluation criteria impose a relatively large number of patients for studies. An objective that is often impossible to achieve for rare diseases. 

👉 Three new rare diseases included in newborn screening 

The French Ministry of Health announced on May 7 that newborn screening will be extended to include three new serious diseases from September onwards, following recommendations from the French National Authority for Health (HAS). This concerns infantile spinal muscular atrophy (SMA), characterized by neuromuscular degeneration, and severe combined immunodeficiency (SCID), which weakens babies' immune systems. These two diseases are now being treated with a range of therapies, including gene therapies that are effective when administered before symptoms appear. The third targeted disease, very long-chain fatty acid acyl-coenzyme A dehydrogenase (VLCAD) deficiency, is initially treated by dietary measures. This screening will be added to that recommended for 13 diseases at birth. 

👉 Montpellier prepares its future anti-cancer campus

On May 9, the Montpellier Cancer Institute (ICM) unveiled its plans for a Center for Transfer and Innovation in Oncology (CTIO), with the support of the French government and INSERM. Expected to open in the first half of 2028, it will integrate cutting-edge platforms such as molecular analysis of tumors, immuno-metabolic profiling and validation of circulating biomarkers, notes La Tribune. It will also house a computational biology cluster. On completion of the building, 60% of the space will be dedicated to research (some fifty scientists) and 40% to business. Extensions are also envisaged.