503O13 is a next-generation, biodegradable lipid nanoparticle (LNP) engineered for highly efficient and targeted siRNA delivery. Designed through rational structure-activity criteria—including optimal tail length (O13), tertiary amines, and a surface pKa ≥5.5—this single-component LNP achieves unparalleled gene silencing with an ultra-low EC50 of 0.01 mg/kg in preclinical models.503O13 outperforms non-degradable counterparts (e.g., C12-200) with improved toxicity profiles—no hepatic necrosis or pancreatic inflammation—while maintaining rapid blood clearance (t1/2: 6 min) and organ-specific accumulation (liver/spleen).
Cat.No
DC49889
Name
503O13
Chemical Properties
CAS
1566560-20-0
Formula
C73H144N4O8
MW
1205.95
Storage
2 years -20°C Powder, 2 weeks 4°C in DMSO, 6 months -80°C in DMSO
References
Whitehead, K., Dorkin, J., Vegas, A. et al. Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity. Nat Commun 5, 4277 (2014).
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