
UB145
TargetsFactor XI (siRNA)
IndicationsAnticoagulant therapy; Stroke
ClinicalCN - I
TherapeuticsCardiovascular; Neurology
ModalitiesNucleic Acid Therapeutics
Exclusive Summary
- **Best-in-Class Potential**: UB145 acts on the 'Holy Grail' target Factor XI with preclinical NHP data showing a T50 >154 days, enabling ultra-infrequent subcutaneous dosing compared to twice-daily oral competitors like Milvexian.
- **Platform Innovation**: Built on the proprietary GalAhead (mxRNA) platform using single-stranded RNAi, offering a smaller molecule size, simplified CMC process, and lower manufacturing costs than traditional double-stranded siRNAs.
- **Clinical De-risking**: Currently in Phase I SAD study with the first two cohorts dosed; demonstrated a clean safety profile with no SAEs and dose-dependent aPTT prolongation.
- **Strategic Development**: Proposed entry strategy via VTE prevention in Total Knee Replacement (TKR) allows for a cost-effective, rapid Proof-of-Concept before expanding to massive indications like Stroke prevention in AFib.
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