AL001 – Frontotemporal dementia with a C9orf72 mutation – Phase 2

utation (FTD-C9orf).  FTD-C9orf72 is associated with abnormal accumulation of the protein TDP-43, which is also a hallmark in FTD. The Phase 2 study, which also includes a cohort of patients with progranulin mutations (FTD-GRN) was designed to assess the safety and tolerability of chronic dosing of AL001 and is also measuring biomarkers of disease and potential clinical activity.