Targeting PIKFYVE and SYF2 for ALS; plus new protein degraders and more

A pair of studies led by AcuraStem Inc. and Modulo Bio Inc. co-founder Justin K. Ichida show that targeting the kinase PIKFYVE or the spliceosome-associated factor SYF2 ameliorated disease pathologies in models of different forms of ALS.

The first study reported in Cell that PIKFYVE inhibition increased exocytosis of aggregation-prone proteins and decreased intracellular pathology in ALS iPSC models. In mice, inhibition of the kinase increased motor function and survival. PIKFYVE is the target of Verge Genomics Inc.’s lead candidate, which is in development for ALS and started Phase I testing in October, 2022. ...