SAM1196

SAM1196 is a small molecule autophagy modulator being developed by Samsara Therapeutics in collaboration with the Charcot-Marie-Tooth Research Foundation (CMTRF). It is specifically designed for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A), a condition often characterized by the toxic accumulation of misfolded proteins due to PMP22 gene duplication. By targeting and modulating the autophagy pathway, SAM1196 aims to enhance the cell's natural ability to degrade and clear these protein aggregates, thereby preserving Schwann cell health and peripheral nerve function. The drug is currently in the discovery phase of development.