# Targeted therapeutic delivery to diseased lungs using enucleated mesenchymal stem cells

> **NIH NIH R43** · CYTONUS THERAPEUTICS, INC. · 2021 · $297,553

## Abstract

There is critical need for cell-based therapeutics that can be administered intravenously (IV), and effectively home to and
deliver therapeutics to the respiratory system, while maintaining patient safety. Cytonus Therapeutics and UC San Diego's
Medical Center are co-developing enucleated mesenchymal stem cells with potential to deliver a wide range of biologics to
treat respiratory diseases including acute respiratory distress syndrome (ARDS). Our novel platform for therapeutic delivery
is to genetically engineer mesenchymal stem cells (MSCs) with inflammation homing proteins and then gently remove the
nucleus, thereby providing a highly unique, viable, and safe cell therapeutic (CargocytesTM) with substantial lung homing
potential. Enucleation grants the ability to genetically engineer Cargocytes with multiple lung targeting moieties and a wide
range of biological payloads, while maintaining a clinically relevant safety profile. Our lung targeting strategy is built on
the key potential of Cargocyte therapeutics to perform active-targeted delivery to the lungs via an intravenous route (i.v.).
Nucleated MSCs will first be extensively engineered with established chemoattractant receptors CXCR4/CCR2 and
inflamed endothelial adhesion molecule PSGL-1 and then enucleated prior to i.v. administration. Proof-of-concept
preclinical studies will then be performed to determine whether Cargocytes engineered with lung trophic molecules home
to inflamed lungs in a clinically relevant murine model of ARDS. Therefore, Aim 1 studies will determine if Cargocytes
engineered with CCXCR4/CCR2 and PSGL-1 home to inflamed lung tissues and Aim 2 studies will determine if Cargocytes
exit the vasculature and move into the inflamed/damaged lung parenchyma. If Cargocytes home to inflamed lung tissues
and exit the vasculature, it could provide an effective means to treat a wide range of respiratory diseases.

## Key facts

- **NIH application ID:** 10156129
- **Project number:** 1R43HL156665-01
- **Recipient organization:** CYTONUS THERAPEUTICS, INC.
- **Principal Investigator:** HUAWEI WANG
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $297,553
- **Award type:** 1
- **Project period:** 2021-09-20 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10156129

## Citation

> US National Institutes of Health, RePORTER application 10156129, Targeted therapeutic delivery to diseased lungs using enucleated mesenchymal stem cells (1R43HL156665-01). Retrieved via AI Analytics 2026-06-11 from https://api.ai-analytics.org/grant/nih/10156129. Licensed CC0.

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