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Drug Names(s): IFB088, IFB 088, Sephin1
IFB-088 is an orally available small molecule drug candidate with a validated mechanism of action for targeting the central and peripheral nervous system. IFB-088 is a selective inhibitor of PPP1R15A (GADD34), a stress-induced PP1 phosphatase regulatory subunit involved in the unfolded protein response. PPP1R15A inhibition by IFB-088 inhibits the protein translation rate in stressed cells to a levelmanageable by the available cellular proteins that assist in protein folding (so-called “chaperones”), therebyrestoring proteostasis. IFB-088 is specific for stressed cells, avoiding persistent inhibition of proteinsynthesis in normal, non-stressed cells.
In some forms of Charcot-Marie-Tooth disease, the proteins involved in the structure and function of the myelin sheath around peripheral nerves do not fold properly and accumulate in cells. IFB-008 is expected to work by slowing down the production of these proteins, which is expected to allow them to fold properly. This would allow the correct formation of the myelin sheath, improving the transmission of nerve signals.
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