Preclinical Efficacy

Dose-Response Data

YAC-GAA transgenic mouse model (n=15/group) — 12-week endpoint analysis

12x
Peak Frataxin
Restoration at high dose
Frataxin Fold-Change vs. Untreated Across Tissues
0 2x 4x 6x 8x 10x 12x 1x 1x 1x Untreated Baseline 6x 4.3x 5x Low 1e13 vg/kg 9x 6.7x 7.7x Mid 5e13 vg/kg 12x 9.3x 10.1x PEAK High 2e14 vg/kg *** p < 0.001
Heart
Brain
Liver
Cardiac Function
+35%
LVEF improvement at high dose vs. untreated controls at 12 weeks
Neurological Score
+4.2 pts
mFARS composite improvement — rotarod, grip, gait analysis at endpoint
Safety Profile
No dose-limiting toxicities
Normal liver enzymes (ALT/AST)
Minimal immune response (IgG)
No off-target biodistribution