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Development of a Novel Therapeutic Transporter to Treat Metastatic Cancers

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41CA257020-01
Agency Tracking Number: R41CA257020
Amount: $399,346.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: NCI
Solicitation Number: PA19-270
Solicitation Year: 2019
Award Year: 2020
Award Start Date (Proposal Award Date): 2020-09-01
Award End Date (Contract End Date): 2021-08-31
Small Business Information
Columbus, OH 43206-2627
United States
DUNS: 117145043
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: Yes
Principal Investigator
 (858) 822-5610
Business Contact
Phone: (631) 258-4070
Research Institution
9500 Gilman Drive, Mail Code 0934
LA JOLLA, CA 92093-0934
United States

 Nonprofit College or University

There is critical need for therapeutic delivery devices that can be administered intravenously (IV), and effectively home to
and deliver therapeutic agents to diseased tissues, while maintaining patient safety. Cytonus Therapeutics, in collaboration
with UC San Diegoandapos;s Moores Cancer Center, has pioneered a unique, drug transporter system with potential to treat to a
wide range of cancers including advanced-stage metastatic tumors. Our novel platform for therapeutic delivery is to
genetically engineer mesenchymal stem cells (MSCs) with specific tumor trophic proteins and then gently remove the
nucleus, thereby providing a highly unique, viable, and safe drug delivery vehicle (CargocytesTM). Cargocytes are viable
for at least 3-5 days and show dramatically reduced lung trapping after IV administration resulting in substantially improved
homing to inflamed tissues in vivo. Moreover, Cargocytes can be engineered to express chemoattractant receptors CXCR4,
CCR2, and the endothelial adhesion molecule PSGL-1, to enhance therapeutic delivery to tumors in response to their
cognate ligands SDF-1, CCL2, and P-selectin, commonly upregulate in tumors and metastatic tissues, respectively.
Therefore, feasibility studies will be performed to determine if cargocytes genetically engineered to simultaneously express
chemoattractant receptors (CXCR4, CCR2) and PSGL-1 (endothelial adhesion molecule) facilitate improved homing to
tumors (Aim 1) and lung metastases (Aim 2) in the MMTV-PyMT preclinical animal model of metastatic breast cancer.
This work is important because bioengineered cargocytes could serve as a new biotransporter device to deliver therapeutics
to malignant tumors and systemic metastases and maintain a clinically-relevant safety profile. Such innovative drug delivery
platforms like Cargocytes are sorely needed to advance treatments for cancer patients with metastatic disease.There is critical need for therapeutic transporters that can be administered intravenously 
(IV), and effectively home to and deliver therapeutic agents to tumors and/or metastatic 
sites, while maintaining patient safety. The objective of this Phase I application is to first 
develop a clinical prototype of an enucleated cargocyte transporter and demonstrate its 
ability to home to primary tumors and metastatic sites using an established, clinically 
relevant mouse model of breast cancer.

* Information listed above is at the time of submission. *

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