Nanomedicine has spent much of the past twenty years as a secondary tool for improving difficult drug formulations rather than a central driver of drug discovery. According to Dr. Kannan Rangaramanujam, Co-Director of the Center for Nanomedicine, this must change. “Putting drug delivery early in the process can enable transformative advances and significantly increase trial success rates,” he says. As drug developers continue searching for ways to improve both safety and effectiveness, early investment in delivery technologies is emerging as a key strategy.
The pandemic highlighted what nanoparticles can accomplish, yet the field still awaits the decisive clinical moment that pushes a therapeutic modality into the mainstream. Other major advances in biotech, such as RNA interference or gene editing, accelerated only after early breakthroughs demonstrated clear and compelling value. Nanomedicine has not yet had its defining proof point, but the foundation is being laid.
One particularly promising area involves dendrimer-enabled nanomedicines. Dr. Kannan and his collaborators have developed this platform over more than two decades through work at Johns Hopkins and Wayne State. The approach uses tree-like polymer structures that carry drugs directly into the specific cells driving disease. Unlike more complex targeting systems, dendrimers rely on the biology of disease itself. Injured or dysfunctional cells take them up at far higher rates than healthy cells, allowing precise delivery of drug payloads to the sites that matter.
This simplicity is one of the platform’s greatest strengths. Many technologies require complicated targeting ligands or large, multifaceted constructs that introduce new safety and manufacturing challenges. By contrast, dendrimer drug conjugates achieve intracellular delivery with a straightforward architecture and predictable behavior. They avoid liver metabolism, which reduces toxicity and the risk of drug-drug interactions. They consistently concentrate inside diseased cells at levels far beyond what the free drug can reach on its own, often improving the therapeutic index by one hundredfold or more. In several models, uptake improvements have exceeded one thousandfold.
These advantages have translated into clinical performance. The platform has been evaluated in nine clinical trials across multiple programs, all showing strong safety and promising signs of efficacy. The first dendrimer-based drugs are now entering Phase IIb and Phase III testing through Ashvattha Therapeutics, the company that licensed foundational intellectual property from Johns Hopkins and Wayne State. Early studies indicate that dendrimer conjugation allows dramatic dose reductions while maintaining or enhancing drug activity, a combination that offers meaningful advantages for both patients and providers.
The applications extend across a wide range of therapeutic areas. Programs are in development for neuroinflammation, retinal disorders, epilepsy, cancer, obesity, pain, and mental health. This breadth reflects the platform’s ability to solve a fundamental biological challenge rather than a niche engineering problem. Many diseases remain difficult to treat because traditional drugs simply cannot reach or accumulate within the necessary cell populations. By addressing this core limitation, dendrimer nanomedicines offer a path toward therapies that traditional approaches have repeatedly failed to deliver.
Dr. Kannan emphasizes that the field must embrace bold thinking if it hopes to solve the most difficult medical problems. “We cannot solve our tough healthcare problems without outrageous innovations as we have seen in the fields of AI, social media, and computers,” he says. Incremental improvements will not overcome barriers such as the blood-brain barrier, intracellular delivery, or systemic toxicity. These challenges require approaches that are both elegant and practical, which he believes dendrimer technologies uniquely provide.
Innovation in healthcare remains constrained by complex regulatory systems, risk-averse investment climates, and a preference for familiar strategies. Yet Dr. Kannan and his colleagues remain optimistic. With more than 50 preclinical models, 7 species, including humans, over 150 peer-reviewed publications, and nearly 200 patents, the scientific foundation is strong. Multiple affiliated start-ups are expanding the pipeline, and the first late-stage trials are underway.
Many in the field believe nanomedicine is nearing its breakthrough moment. If dendrimer-based therapies succeed in late-stage studies, they could become the catalyst the field has been waiting for and usher in a new era of precision therapeutics. Dr. Kannan and his team are preparing for exactly that future, one where delivery is not an afterthought but a central pillar of how we design the next generation of transformative medicines.
Spencer Hulse
Spencer Hulse is the Editorial Director for Grit Daily Group. He works alongside members of the platform’s Leadership Network and covers numerous segments of the news.
