Биологическая активность
Based on the data from patent US 20250127728A1, CICL-238 emerges as a highly promising ионизируемый липид candidate, demonstrating notable advantages for targeted delivery applications. It achieves exceptional transfection efficiency—reaching approximately 90% of CICL-207's performance in splenic T-cells even at a reduced липид ratio of 50% in LNP formulations. Additionally, CICL-238 exhibits minimal off-target expression in hepatocytes (<8%, comparable to CICL-207), underscoring its enhanced specificity for immune cells over liver tissues. Its optimized structure likely contributes to efficient endosomal escape and reduced Kupffer клетка uptake, making it ideal for liver-related therapies (e.g., siRNA silencing for metabolic diseases) and potentially broadening applications to genetic medicine where precision and safety are paramount. Further validation in disease models could solidify its role as a versatile, low-toxicity alternative to benchmark lipids.
Оригинал на английском
Based on the data from patent US 20250127728A1, CICL-238 emerges as a highly promising ionizable lipid candidate, demonstrating notable advantages for targeted delivery applications. It achieves exceptional transfection efficiency—reaching approximately 90% of CICL-207's performance in splenic T-cells even at a reduced lipid ratio of 50% in LNP formulations. Additionally, CICL-238 exhibits minimal off-target expression in hepatocytes (<8%, comparable to CICL-207), underscoring its enhanced specificity for immune cells over liver tissues. Its optimized structure likely contributes to efficient endosomal escape and reduced Kupffer cell uptake, making it ideal for liver-related therapies (e.g., siRNA silencing for metabolic diseases) and potentially broadening applications to genetic medicine where precision and safety are paramount. Further validation in disease models could solidify its role as a versatile, low-toxicity alternative to benchmark lipids.
Химические свойства
- Брутто-формула
- C₅₆H₁₀₀O₁₄N₂
- Мол. масса
- 1025.42 г/моль
- SMILES
- CCCCCCCCC(=O)OCC(COC(=O)CCCCCCCC)CC(=O)O[C@H]1C[C@@H](OC(=O)CC(COC(=O)CCCCCCCC)COC(=O)CCCCCCCC)CN(C(=O)OCCN(C)C)C1
- Синонимы
- CICL238，CICL 238
3D-структура молекулы
Вращайте мышью · Колесо — масштаб
Хранение и физические свойства
- Условия хранения
- 2 года -20°C порошок, 2 недели 4°C в ДМСО, 6 мес. -80°C в ДМСО
Безопасность и документация
Только для научно-исследовательского применения. Не предназначено для использования у пациентов или в качестве лекарственного средства.