Line Plot from Scientific Research

CSF samples were collected from study participants in the low-dose (2.1 x 10 13 vg, n = 5) and mid-dose (4.2 x 10 13 vg, n = 6) cohorts. Progranulin concentrations (ng ml -1 ) in CSF samples were measured by ELISA and plotted against timepoint of collection (baseline and months 2, 6 and 12; additional timepoints were collected at months 3 and 4 for two study participants). At baseline, progranulin concentrations were all below normal levels, as reported by Goossens et al. 35 (denoted in the figure by the red line). Upon dosing with PR006, at the month 2 timepoint, levels of progranulin concentration increased by 2.1–6.9x above the respective baseline levels. Among the nine patients with CSF progranulin data at month 6, eight (89%) had progranulin level within or above the normal range, whereas, among the four patients with 12-month data, three (75%) had levels within or above the normal range, as described by Goossens et al. 35 .

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