Biomedical Engineering and Imaging Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Department of Medical Biochemistry, Amsterdam University Medical Centers, Amsterdam, Netherlands.
Sci Adv. 2021 Mar 5;7(10). doi: 10.1126/sciadv.abe7853. Print 2021 Mar.
Immunotherapies controlling the adaptive immune system are firmly established, but regulating the innate immune system remains much less explored. The intrinsic interactions between nanoparticles and phagocytic myeloid cells make these materials especially suited for engaging the innate immune system. However, developing nanotherapeutics is an elaborate process. Here, we demonstrate a modular approach that facilitates efficiently incorporating a broad variety of drugs in a nanobiologic platform. Using a microfluidic formulation strategy, we produced apolipoprotein A1-based nanobiologics with favorable innate immune system-engaging properties as evaluated by in vivo screening. Subsequently, rapamycin and three small-molecule inhibitors were derivatized with lipophilic promoieties, ensuring their seamless incorporation and efficient retention in nanobiologics. A short regimen of intravenously administered rapamycin-loaded nanobiologics (mTORi-NBs) significantly prolonged allograft survival in a heart transplantation mouse model. Last, we studied mTORi-NB biodistribution in nonhuman primates by PET/MR imaging and evaluated its safety, paving the way for clinical translation.
免疫疗法控制适应性免疫系统已得到充分证实,但调节先天免疫系统的研究还远远不够。纳米颗粒与吞噬性髓样细胞之间的固有相互作用使这些材料特别适合与先天免疫系统相互作用。然而,开发纳米疗法是一个精细的过程。在这里,我们展示了一种模块化方法,可有效地将多种药物纳入纳米生物平台。我们使用微流体制备策略,生产出载脂蛋白 A1 的纳米生物药物,通过体内筛选评估其具有良好的先天免疫系统参与特性。随后,用亲脂性促进剂衍生出雷帕霉素和三种小分子抑制剂,确保它们能够无缝整合并有效地保留在纳米生物药物中。静脉给予载有雷帕霉素的纳米生物药物(mTORi-NBs)的短疗程方案显著延长了心脏移植小鼠模型中的同种异体移植物存活期。最后,我们通过 PET/MR 成像研究了 mTORi-NB 在非人类灵长类动物中的生物分布,并评估了其安全性,为临床转化铺平了道路。