ALPHA-0602

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. 

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