# Harnessing the power of exosomes for non-coding RNA delivery

> **NIH NIH R35** · UNIVERSITY OF TX MD ANDERSON CAN CTR · 2022 · $895,013

## Abstract

Project Abstract:
Despite the promise of RNA interference (RNAi) approach for targeting undruggable targets, major challenges
remain including specific delivery of siRNA into cell types of interest in vivo, poor stability and off-target effects.
We have been at the forefront of addressing these issues and my laboratory has pioneered many studies using
RNAi approaches for cancer treatment and has made key discoveries related to RNAi biology. We were
among the first to demonstrate that RNAi processing machinery is deregulated in a high proportion of ovarian
and other cancers (Merritt et al., New Engl J Med 2008). To achieve systemic delivery of RNAi therapeutics,
we systematically identified safe and effective methods for siRNA delivery. After extensive testing, our first
successful platform utilized the neutral DOPC nanoliposomal delivery system (Landen et al., Cancer Res 2005;
Ahmed et al., Cancer Cell 2010), which has subsequently been tested in multiple tumor model systems (Liu et
al., Nature 2015; Kim et al., Cell 2013). With a robust portfolio of preclinical studies and all of the requisite
safety studies based on FDA guidance, a first-in-human phase I clinical trial with EPHARNA (EphA2 targeted
siRNA in DOPC) is nearing completion for patients with solid tumors. We have made great strides in applying
this technology for cancer therapy. However, despite the promise of synthetic delivery systems that we and
others have developed, novel and biocompatible delivery strategies that are independent of reliance on
vascular leakiness are highly desirable. In this project, we propose to develop a biomimetic exosomal system
that will enable active delivery of cancer therapeutics to the tumor microenvironment. These naturally occurring
particles represent a promising, and safe alternative approach for delivering RNAi therapeutics. We will
package RNAi cargos into these particles and engineer their surface membrane to actively target distinct cell
types. Finally, we will develop this approach for enhancing anti-tumor immune response in ovarian and other
cancers. If successful, this biomimetic exosomal system can be rapidly applied more broadly to other
“undruggable” targets. Support through the R35 mechanism will greatly facilitate this undertaking, which would
not otherwise be possible.

## Key facts

- **NIH application ID:** 10461095
- **Project number:** 5R35CA209904-06
- **Recipient organization:** UNIVERSITY OF TX MD ANDERSON CAN CTR
- **Principal Investigator:** ANIL K SOOD
- **Activity code:** R35 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $895,013
- **Award type:** 5
- **Project period:** 2017-08-02 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10461095

## Citation

> US National Institutes of Health, RePORTER application 10461095, Harnessing the power of exosomes for non-coding RNA delivery (5R35CA209904-06). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10461095. Licensed CC0.

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