MAASiRNA

MAAsiRNA is a French biotechnology company founded in 2023, specializing in the development of targeted siRNA therapies for rare nervous system disorders. The company's lead program, SICMT, addresses Charcot-Marie-Tooth disease type 1A (CMT1A) by utilizing small interfering RNA (siRNA) to normalize the expression of the PMP22 protein, which is overexpressed in patients. This therapeutic approach employs a proprietary delivery platform featuring siRNA encapsulated in biocompatible squalene nanoparticles, designed to specifically target Schwann cells. Based on over a decade of interdisciplinary research from the Paris-Saclay cluster, MAAsiRNA has demonstrated efficacy in animal models and is currently advancing through regulatory preclinical studies with plans to enter clinical trials in 2026.