ELN-128 (Post-MI HF): Single SC dose 2 hours post-reperfusion improved LV ejection fraction, reduced ventricular volumes, and decreased infarct size over 28 days in mice. RNA-seq confirmed restoration of oxidative phosphorylation and suppression of inflammatory pathways.
ELN-128 (DMD): 8-week treatment rescued chronic heart failure in a pig DMD model with improved cardiac output and reduced cardiac damage markers.
ELN-33: Systemic SC dosing reaches retina in NHP. Eliminates need for intravitreal injection.
Exon Skipping: Next-generation steric-block discovery capabilities with functional activity profiles not typically achievable through conventional splice-focused approaches.