# Therapeutic platform to treat disease lung using enucleated mesenchymal stem cells0/01/2021

> **NIH NIH R43** · CYTONUS THERAPEUTICS, INC. · 2021 · $454,975

## 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:** 10257613
- **Project number:** 1R43HL158351-01
- **Recipient organization:** CYTONUS THERAPEUTICS, INC.
- **Principal Investigator:** Remo Moomiaie
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $454,975
- **Award type:** 1
- **Project period:** 2021-07-16 → 2023-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10257613

## Citation

> US National Institutes of Health, RePORTER application 10257613, Therapeutic platform to treat disease lung using enucleated mesenchymal stem cells0/01/2021 (1R43HL158351-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10257613. Licensed CC0.

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