Progranulin is a natural protein that is expressed in several cell types in the central nervous system and in peripheral tissues and has a potent ability to protect neurons that are under stress. ACI is developing a specific form of progranulin, called Alpha-602, for the treatment of ALS patients. Alpha-602 regulates cell survival and certain inflammatory processes. The protein plays a major role in regulating lysosomal function and microglial responses to disease.
In pre-clinical studies, ACI has demonstrated that progranulin can correct the cellular defects caused by the altered patterns of TDP-43 and FUS proteins found in motor neurons of more than 90% of all ALS patients. ACI has been granted two EU
patents relating to the treatment of neurodegenerative diseases using progranulin. Additionally, the FDA, has granted ACI Orphan Drug Designation for its progranulin product platform for the treatment of ALS.