| Description | Pelacarsen (AKCEA-APO(a)-LRx) is a hepatotropic antisense oligonucleotide targeting apolipoprotein(a) that effectively attenuates lipoprotein(a) levels by up to 80% and exhibits favorable tolerability [1]. |
| In vivo | 在小鼠身上，每周一次连续六周给予不同剂量的Pelacarsen（具体剂量为0.3、1、3和10 mg/kg）后，观察到所针对的mRNA表达量呈剂量依赖性下降。模型估算出使Pelacarsen达到最大药物抑制效应50%（ED50）所需的剂量，对于肝脏apo(a) mRNA和血浆apo(a)蛋白水平分别为0.32 mg/kg/week和0.54 mg/kg/week。 |
| Synonyms | TQJ230, AKCEA-APO(a)-LRx, ISIS 681257 |
| molecular weight | N/A |
| CAS | 1637637-70-7 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 year |