A success story from the Institut de neurobiologie de la méditerranée (Inmed – amU/Inserm) and the Institut Interdisciplinaire de Neurosciences (IINS – Bordeaux Neurocampus/CNRS).
A decisive step has just been taken in the treatment of drug-resistant medial temporal lobe epilepsy (MTLE), a severe and disabling form of the disease, thanks to the joint work of Valérie Crépel, Inserm research director at the Mediterranean Neurobiology Institute (Inmed – Inserm/Aix-Marseille University), and Christophe Mulle, CNRS research director at the Interdisciplinary Neuroscience Institute (IINS, Bordeaux Neurocampus). This breakthrough led to the creation of the start-up Corlieve Therapeutics, recently integrated into the uniQure group, a major player in gene therapy.
Launched in November 2024, the phase I/IIa clinical trial of the drug candidate AMT-260, developed on the basis of this research, is already delivering very encouraging results: in the first patient treated, a 92% reduction in epileptic seizures was observed over the first five months of follow-up, with no serious adverse effects. The patient, who had an average of seven seizures per month before treatment, reported no seizures during the last 60 days of follow-up (as of 17 April 2025).
This innovative therapy targets the neuronal circuits at the origin of seizures in the temporal lobe, offering considerable hope for the 30% of patients resistant to current treatments.
This project fully embodies Inserm’s drive to exploit public research. It had already been recognised in 2022 by the Inserm Innovation Prize, which rewarded the commitment of Valérie Crépel and her partners to developing therapeutic solutions with a high societal impact.
“It is immensely satisfying to see our laboratory work lead to a concrete treatment that could significantly improve the quality of life of patients who have had no solution until now.
– Valérie Crépel
This success also illustrates the importance of supporting innovation: Inserm Transfert has supported Corlieve Therapeutics from the outset, fostering the link between academic research and industrial development.
The clinical trial is continuing with the inclusion of new patients. The results expected in the coming months will confirm the efficacy and safety of this breakthrough therapy.
Contact :
Valérie Crépel, PhD, Research Director at Inserm
Institut de Neurobiologie de la Méditerranée – Inmed
Inserm & Aix-Marseille Université UMR1249 Parc scientifique de Luminy 163 avenue de Luminy BP13 – 13273 Marseille cedex 09 France
Tel.: +33 (0)4 91 82 81 15
Mail : valerie.crepel@inserm.fr