# Bone marrow-targeted extracellular vesicles as a novel non-viral gene editor delivery platform

> **NIH NIH R44** · STRM.BIO, INC. · 2022 · $1,004,289

## Abstract

Despite advances in gene therapy techniques, novel methods that facilitate tight targeting of gene
therapy products to specific tissues and cell types are necessary to achieve widespread clinical
applicability. Inherited hematologic diseases represent an ideal target for novel genetic therapies,
as gene therapy approaches can bypass the significant limitations associated with bone marrow
transplantation and symptomatic treatment. Extracellular vesicles (EVs) capable of targeting
hematopoietic stem cells (HSCs) are a promising option for the targeted delivery of gene editor
machinery to treat genetic hematologic diseases. Successful system development represents a
billion-dollar market opportunity that will be defined by the ability to show: (1) targeting specificity,
(2) a favorable safety profile, and (3) efficient in vivo delivery of gene editor machinery. We have
developed a proprietary EV-based system for in vivo nucleic acid and protein delivery that
specifically targets HSCs in bone marrow, is amenable to large scale/commercial manufacture,
and presents with low immunogenicity which confers unique potential for repeat dosing. Our
hypothesis is that the STRM.BIO EV-based system can be efficiently loaded with gene editor
cargo, safely home to the bone marrow following intravenous delivery, and effectively edit
targeted genes in HSCs. With funds provided by this Phase II award, we will (1) establish SOPs
for loading STRM.BIO EVs with cargo, (2) characterize the biodistribution and delivery pattern of
cargo-loaded EVs, and (3) verify feasibility of STRM.BIO EVs for in vivo cargo delivery in a mouse
model of human genetic hematologic disease. The current proposal aims to establish pre-clinical
proof-of-concept support for the use of our proprietary system as a non-viral, in vivo genome
editor delivery system for the treatment of inherited hematologic diseases.

## Key facts

- **NIH application ID:** 10484260
- **Project number:** 1R44TR004238-01
- **Recipient organization:** STRM.BIO, INC.
- **Principal Investigator:** JONATHAN N THON
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,004,289
- **Award type:** 1
- **Project period:** 2022-07-01 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10484260

## Citation

> US National Institutes of Health, RePORTER application 10484260, Bone marrow-targeted extracellular vesicles as a novel non-viral gene editor delivery platform (1R44TR004238-01). Retrieved via AI Analytics 2026-06-01 from https://api.ai-analytics.org/grant/nih/10484260. Licensed CC0.

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