Figure 2.

Stereopure oligonucleotide (MALAT1-200) is more efficacious and more potent than the stereorandom control in vivo in mouse eye. (A) Dosing regimen for 1-week mouse study, with mice receiving a single IVT injection on day 0 (d0, red arrow) and with sample collection 1 week later (d7, blue arrow). In vivo expression of Malat1 in anterior (top row) or posterior (bottom row) portions of mouse eyes 1 week after treatment with the indicated oligonucleotide (top of graph, dose indicated on x-axis) or PBS. n ≤ 7 depending on treatment; each point represents one treated eye. The red dotted line demarcates 50% Malat1 expression. (B) PK-PD relationships for stereorandom (50 µg) and stereopure (50 µg) oligonucleotides in the posterior portion of the eye at 1 week are shown. The percentage of Malat1 remaining is plotted with respect to the concentration of oligonucleotide detected in the tissue (n = 7); each point represents data from one treated eye.