Figure 9. In vivo safety study in WT NHPs showed good viral biodistribution and hAP4B1 expression across the CNS, minimal-mild macroscopic changes in DRG, spinal cord and sciatic nerve 1 and 4 months following ICM treatment of AAV9/hAP4B1, with no adverse effect on nerve conductance.

V5-empty, low (3.2 × 10¹² vg/kg), and high (1.7 × 10¹³ vg/kg) doses of AAV9/hAP4B1 vector were administered intra-cisternal to aged matched NHPs (n = 2 NHPs for V5-empty, n = 3 NHPs per treatment group (low or high dose). At 29 days and 113 days after injection, NHP organs were harvested. (A) Viral vector distribution assessment by qPCR of total DNA extracted from tissues at 16 weeks post vector administration. (B) hAP4B1 transgene expression in various tissues by RT-qPCR of total RNA extracted from NHPs at 16 weeks post vector administration. (C) Nerve conduction velocity (NCV) tests were performed at baseline and demonstrated no difference between test article and control (see Appendix Table S2 for data set). All data are presented at mean ± SEM. Source data are available online for this figure.