This WP involves Alexis Osseni (post-doctoral fellow) and Nicolas Hugo (research engineer) from Laurent Schaef­fer’s team, along with the services of EDELRIS, a company with expertise in medicinal che­mistry, through its experts in organic che­mistry and chemistry including Gérald Coste.

Alexis Osseni and colleagues have de­monstrated that HDAC6 inhibition is a promi­sing therapeutic strategy for several neuromuscular diseases such as Duchenne Muscular Dys­trophy and Spinal Muscular Atrophy (Osseni et al, nature comm et al, 2022 ; Osseni et al., BioXiV 2025)

The aim of this work package is to optimize HDAC6 inhibitors to generate adapted molecules for human therapy. Several candidate molecules have been identified and shown to specifically inhibit HDAC6 without inhibiting nuclear HDACs. Further characterization will be needed to validate them as suitable candidate drugs.