France has long been a European leader in rare diseases, from its scientific research and academic hospitals, through to patient advocacy and regulatory reforms that have encouraged R&D. Its biotech and pharmaceutical industries are now leading the way, discovering the next generation of therapies for patients that are often overlooked.

The French non-profit, whose IP is incorporated in Zolgensma, is taking its Crigler-Najjar gene therapy into a pivotal study on its own for now. It hopes to be ready for an EU filing in two years.

A total of four investigational products targeting unmet medical needs have made it onto the European Medicines Agency’s priority medicines scheme so far this year.

Immix collaborates with Israeli research institutions on BCMA-targeted CAR-T therapy. NeoImmuneTech/NIAID alliance could pave way for “animal rule” approval in acute radiation syndrome.