申请人:[en]UNIVERSITY OF MARYLAND, BALTIMORE
公开号:WO2024112943A1
公开(公告)日:2024-05-30
We disclose here the nano-enabled delivery of highly potent 5 -hydroxymethyl furfural (5-HMF) to sickled blood cells in patients. 5-HMF, a superior antisickling agent, has been formulated to address the limitations of existing treatment modalities arising from disfavorable pharmacokinetics of the drug compound. Specifically, two complementary 5-HMF prodrugs are integrated as a "protected" biocompatible composite nanoparticle designed to readily fuse with RBCs and be amenable to transdermal delivery (5-HMF-grafted-phospholipid layered over a sucrose-derived graphitic carbon dot core). These RBC-targeted, protected, biocompatible, self-assembled 5-HMF prodrug nanoparticles for sickle cell disease are the first in class technology for offering a treatment for sickle cell disease which has improved potency, targeted payload delivery, and extended release with the red blood cells with enhanced pharmacodynamic effect in a treated patient.