# Bioengineering a Novel Therapeutic Transporter that Crosses the Blood Brain Barrier to Treat Brain Disorders

> **NIH NIH R41** · CYTONUS THERAPEUTICS, INC. · 2021 · $324,986

## Abstract

While the brain blood barrier (BBB) is an important physiological barrier that protects the brain, it is also represents a
formable barrier to therapeutic delivery. Therefore, there is a critical need for therapeutic transporters that can be injected
intravenously (i.v.), and effectively traverse the BBB and deliver therapeutic agents to the brain [1-8]. Cytonus
Therapeutics has pioneered the development of a new bioinspired delivery agent (CargocytesTM) with potential to meet
this critical need. Cargocytes are bioengineered enucleated mesenchymal stromal cells (MSCs) that specifically home to
diseased tissues such as the brain and deliver therapeutic payloads following i.v. administration. Substantial work in our
laboratory indicates that Cargocytes have potential to treat brain disorders as they readily extravasate through vascular
barriers, invade endothelial basement membranes, and chemotax though complex extracellular matrices to target tissues
deep within disease foci. Cargocytes can also be engineered to produce, secrete, and/or deliver a range of powerful
therapeutics within the brain milieu including cytokines, neurotrophic factors, antibodies, nanobodies, RNAs, and even
small molecule drugs. Overall, our findings demonstrate that Cargocytes are a new breakthrough technology platform for
maximum local bioprotein delivery and production while minimizing systemic distribution. Cargocyte technology
improves efficacy and reduces off-target toxicity, the “holy grail” of therapeutic delivery. The primary objective of this
phase I application is to develop brain homing Cargocytes that cross the BBB using an established model of ischemic
brain injury.

## Key facts

- **NIH application ID:** 10324736
- **Project number:** 1R41TR003960-01A1
- **Recipient organization:** CYTONUS THERAPEUTICS, INC.
- **Principal Investigator:** Richard L. Klemke
- **Activity code:** R41 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $324,986
- **Award type:** 1
- **Project period:** 2021-08-20 → 2023-08-19

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10324736

## Citation

> US National Institutes of Health, RePORTER application 10324736, Bioengineering a Novel Therapeutic Transporter that Crosses the Blood Brain Barrier to Treat Brain Disorders (1R41TR003960-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10324736. Licensed CC0.

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