Progranulin is a secreted protein that maintains viability of neurons in the cerebral cortex in the brain. Low progranulin levels is a risk factor for Alzheimer’s Dementia, Parkinson’s Disease and ALS. Increasing expression of progranulin is a promising avenue for treatment.
ALPHA-0602 is a gene therapy program delivering progranulin, a neurotrophic protein. It is in preclinical development for the treatment of ALS. ALPHA-0602 is patented and has received Orphan Drug Designation from the FDA.