Calporta is developing selective small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for the treatment of Niemann-Pick C Disease (NPC) and other lysosomal storage diseases. Mutations that cause NPC lead to impaired intracellular lipid trafficking and lead to lysosomal accumulation of cholesterol and biolipds. TRPML1 is an ion channel in the lysosome and a key regulator of lysosomal trafficking processes. Impaired TRPML1 function has been implicated in multiple pathological conditions, including NPC. Agonists of TRPML1 restore calcium efflux and normalize lysosomal trafficking and function. In addition to NPC and other lysosomal storage diseases, agonists of TRPML1 may have broad potential in other diseases such as muscular dystrophy and various taopathies such as Alzheimer's disease. Calporta's approach is based on research from the lab of Haoxing Xu, Ph.D., at the University of Michigan.

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