PEG-[RAPA]4

PEG-[RAPA]4 is a macromolecular prodrug consisting of four rapamycin molecules reversibly conjugated to a 4-arm bioeliminable polyethylene glycol (PEG) scaffold with a molecular weight of approximately 40 kDa. Developed by researchers at The Children's Hospital of Philadelphia, this construct is designed to provide sustained release and enhanced delivery of rapamycin to suppress the proliferative phenotype of activated fibroblasts in pulmonary hypertension (PH). By inhibiting the mechanistic target of rapamycin (mTOR) pathway, the prodrug aims to mitigate the progressive loss of vascular cell homeostasis and fibroproliferative transformation characteristic of the disease. In preclinical studies, PEG-[RAPA]4 demonstrated potent growth inhibitory effects against fetal lung fibroblasts and transformed fibroblast-type cells in vitro and significantly delayed growth in SK-LMS1 xenograft models in vivo.

Other names

4-arm PEG-rapamycin prodrugPEG-rapamycin prodrugpolyethylene glycol-linked rapamycin prodrug