LNP Therapeutic Targeting

This collaborative multidisciplinary research group unites clinicians and basic science researches with primary appointments across the campus including the Schools of Medicine, Engineering, Arts and Sciences, CHOP. These researchers have interests and expertise in translational, mechanistic and methodological aspects of targeted drug delivery systems (DDSs) in the body.

Our primary focus is vascular targeting, in particular to endothealial cells lining the lumen. The PI has pioneered this field three decades ago and the team efforts advanced this approach to a rich continuum of diversified carriers, ligands, cargoes and target epitopes specifically and distinctly enriched in endothelia: normal vs abnormal, pulmonary vs cerebral, internalizable via different endocytic pathways vs surface retained, etc.

We also pursue targeting drugs and carriers to Red Blood Cells (RBC), ideal for intravascular transport of drugs and their hitchhiking to the vascular areas of interest. Further, we load, deliver and transfer drug carriers to many migratory and resident cells in blood and tissues: leukocytes, lymphocyte, macrophages and other host defense and immune cells of different varieties.

We devised successful targeting to these and other elements of the body a wide variety of therapeutic cargoes including enzymes, small molecules, imaging probes and nucleic acids. For these cargoes we use appropriate carriers, protein conjugates and fusion, cells and LNPs.

These targeting strategies and agents showing superior results in preclinical animal studies provide an extremely rich and multifaceted arsenal of means which undoubtedly will advance delivery of RNA therapeutics to the desirable sites of action, providing optimal spatiotemporal control of the effect of this novel therapeutic marvel.

Lipid nanoparticles (LNP) and other delivery systems

Delivery technologies such as lipid nanoparticles (LNP) offer significant advantages over the delivery of free RNA for various RNA therapeutic, vaccine, and basic science applications. LNP technologies protect and prevent RNA degradation in the bloodstream, avoid renal clearance of RNA, enable cellular targeting through ligand functionalization and/or the tailoring of LNP physicochemical properties, and mediate cellular entry and endosomal escape to enable RNA release in the cytoplasm.

The mission of the Lipid Nanoparticle (LNP) Core of the Institute for RNA Innovation is to design and implement novel LNP technologies for a range of RNA therapeutic, vaccine, and basic science applications for research groups across the University of Pennsylvania and the Children’s Hospital of Pennsylvania. The LNP Core offers a wealth of expertise and resources including combinatorial chemistry of ionizable lipids and polymers, microfluidic technologies for LNP formulation, as well as LNP characterization and scaleup for RNA vaccine and therapeutic studies.


RNA plays a central role in biology and has recently emerged as a therapeutic modality as new technologies have made it possible to use RNA to modulate biological pathways to cure disease. One of many diverse areas of RNA therapies includes RNA vaccines, which produce a protein for the development of antibodies and T cells to fight disease. mRNA vaccines were first used to treat infections, and this has made a fundamental impact globally to protect us from SARS-CoV-2. This platform is under development for additional pathogens as well as other diseases. Another therapeutic use of RNA involves anti-sense RNA, including the small RNAs such as siRNAs and AMOs which bind to their cognate mRNA. These small RNA can direct targeted degradation or suppression of mRNA and can also alter RNA splicing to correct genetic disorders. Small-molecule RNA modulators and RNA inhibitors are also underdevelopment to alter RNAs and thus biology. Many RNA-based therapeutics are targeting diseases for which conventional drugs have not been successful. Continued pioneering discoveries in how broadly we can implement existing technology and how we can create new technologies for RNA therapeutic use, hold great promise for new cures for human disease.

The Therapeutics branch of the Institute for RNA Innovation will bring together research groups from across the University of Pennsylvania and the Children’s Hospital of Pennsylvania to promote the development and implementation of RNA therapeutics. We will foster collaborations and promote new initiatives to facilitate these activities.


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