Preparation of Benzene-1,4-disulfonamide Derivatives Useful as Therapeutic TRPML1 Receptor Modulators for the Treatment of Lysosomal Dysfunction and Membrane Repair Disorders
This technology includes a series of novel benzene-1,4-disulfonamides that activate TRPML1 receptor. The TRPML1 receptor is a lysosomal Ca2+ channel that has been shown to be involved in controlling lysosome functions, among then the maintenance of the integrity of the plasma membrane and the modulation of autophagosome-lysosome fusion. The improved ability of the receptor to deliver Ca2+ ions to the cytosol had been correlated with its capacity to modulate autophagy and lysosome exocytosis.