Advancing Nanotechnology Research to Improve Diagnosis and Treatment Options

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Topic Description

Post Date: March 10, 2026

Expiration Date: March 10, 2027

Purpose 

This topic encourages the use of nanotechnology approaches, with a particular focus on extracellular vesicles (EVs) that are associated with homeostasis, development, regeneration, and pathogenesis in all areas of disease. This topic is aimed at developing improved nanotools for early disease detection as well as therapeutics and drug delivery.

Background

EVs are nano-sized vesicles secreted by cells that contain a variety of biological molecules, such as lipids, proteins, and nucleic acids. They have been recognized as important mediators for cell-to-cell communication and are involved in a variety of biological processes including cellular homeostasis and disease pathophysiology. These vesicles have been isolated from almost all biological fluids, can possess anti-inflammatory, regenerative, neuroprotective, and pro-survival effects, and have the potential to identify and monitor different stages of disorders and treatments. However, the link between EV biology/mechanisms and disease or injury is not well understood, with a limited understanding of EV biogenesis, cargo composition, and regulatory mechanisms associated with physiological and pathological conditions. This knowledge is critical for developing EV-based biomarkers and therapeutics. 

EVs have unique advantages, such as high bioavailability, biocompatibility, and low immunogenicity, positioning them as highly effective drug delivery tools and providing a distinct means of delivering various therapeutic agents to target cells. Recent studies show that EVs can be engineered to effectively target specific cell types or to allow for sustained drug delivery and more efficient controlled release in animal models. Utilization of EVs as delivery vectors to enable active targeting and multi-drug delivery could yield higher and sustained efficacy and avoid the unnecessary immune responses and offer opportunities in treating various diseases.

This highlighted topic encourages studies that incorporate new approach methodologies (NAMs) and novel technologies such as system-omics methods, high-throughput assays, digital Flow Cytometry, artificial intelligence (AI), and other computational approaches to elucidate the composition and mechanisms of EVs in diseases and to explore the utility of EVs in diagnosing and treating disorders. 

Participating ICOs

National Eye Institute (NEI)

NEI seeks applications to develop nanoparticles, including EVs, for the diagnoses and treatments of eye diseases such as autoimmune uveitis, Sjogren’s Syndrome, diabetic retinopathy, maculopathies, retinitis pigmentosa, retinopathy of prematurity, and glaucoma. The use of organoids, computational modeling, microphysiological systems, and other NAMs are encouraged to characterize the composition and pathways of the EVs associated with human eye health and disease.

Areas of interest include, but are not limited, to:

  • Link(s) between EV mechanisms and eye disease/injury, e.g., what shifts from normal homeostasis to the pathological state.
  • Engineered EV-based therapeutics or drug delivery tools for the prevention and treatment of ophthalmic disorders, including stem cell-derived EVs containing anti-inflammatory, neuroprotective, regenerative, or pro-survival targeting molecules.
  • EV-based biomarker discovery for early diagnosis of eye diseases and monitoring treatment response.
ICO Scientific Contact:
Hongman Song, Ph.D.
[email protected]

Lisa Neuhold, Ph.D.
[email protected]

National Cancer Institute (NCI)

NCI seeks applications to develop nanoparticle-based technologies, including EVs, for early cancer detection, diagnosis, prognosis, disease monitoring, and prediction of treatment response or resistance, as well as biomarker development for cancer prevention and control. Areas of interest include, but are not limited to:

  • Nanoparticle/EV-based biomarker discovery for early detection, diagnosis, prognosis, and monitoring of aggressive cancers.
  • Prospective, multi-site validation of EV-derived biomarkers, including head-to-head comparisons with existing liquid biopsy assays for early cancer detection.
  • Standardized and scalable methodologies for tumor-derived EV enrichment and characterization, and multimodal cargo analysis.
  • Understanding of functional roles of EVs in cancer initiation, progression, immune modulation, and therapeutic resistance.
  • Engineered EVs and other nanoparticle platforms in cancer prevention/interception, targeted drug delivery, and therapy.
IC may dedicate funds available to support applications in this Topic area depending upon the availability of funds, the number of meritorious applications, and competing ICO priorities.
ICO Scientific Contact:
Indu Kohaar, Ph.D.
[email protected]

Sidney Fu, M.D.
[email protected]

Piotr Grodzinski, Ph.D.
[email protected]

Yali Fu, Ph.D.
[email protected]


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