ALPHA-0602

ALPHA-0602 is an investigational **AAV-mediated gene therapy** being developed by Alpha Cognition for **amyotrophic lateral sclerosis**. The therapy is designed to deliver the **progranulin gene** to increase progranulin expression in the central nervous system, with the goal of providing neuroprotection in ALS. Based on the provided information, increased progranulin is intended to promote neuronal survival, regulate inflammatory and lysosomal pathways, reduce toxic **TDP-43** and **FUS** protein aggregation, and mitigate oxidative stress associated with motor neuron degeneration. The program has reportedly received **FDA Orphan Drug Designation** for ALS and is described as being in **preclinical development**, with supportive activity observed in cell culture and mouse models.