# Efficient and scalable microfluidic gene delivery to hematopoietic stem cells to treat blood diseases

> **NIH NIH R44** · CELLFE INC. · 2023 · $843,224

## Abstract

Abstract
The two dominant methods for delivering genome modifying molecules to cells for cell therapies, viral
transduction and electroporation transfection, have process and technical limitations that hamper the
development of cutting-edge cell therapies accessible to all. While viral vectors result in a high cost of goods
with significant regulatory and health risks associated with manufacturing, electroporation results in unhealthy
cells with low viability and off-target phenotype changes. CellFE’s microfluidic VECT technology enables cell
transfections with high transfection efficiency, low off-target changes to the cell, and with an expanded cell
processing range.
This project aims to demonstrate the first commercial cell transfection platform designed for scalable ex vivo
genetic modification of CD34+ hematopoietic stem/progenitor cells (HSPC) to develop treatments of genetic and
infectious diseases including beta thalassemia, sickle cell disease, hemophilia, and HIV. Scalability means that
process parameters used in the research and development phase of cell engineering will immediately apply to
clinical scale (300-fold increase in number of processed cells) by simply exchanging a chip. A simpler
manufacturing scale-up means a faster path to clinical testing and will be a significant value proposition for the
technology. To achieve these goals, CellFE will pursue the following aims: (1) determine the optimal design to
scaleup device channels for high-throughput, (2) transfer device fabrication from PDMS to injection molded
plastic, (3) demonstrate genetic modification of a therapeutic gene at bench-top at a clinical processing rate, and
(4) develop alpha prototype systems for testing in the labs of our pharmaceutical partners to generate paid pilot
studies. These are the key technical aims of the SBIR Phase II project and are also the main milestones CellFE
needs to accomplish in order to execute its business plan of developing, marketing, and selling cell transfection
platforms for cell therapy manufacturing with the prospect to de-risk manufacturing development and reduce cost
of goods versus current delivery techniques.

## Key facts

- **NIH application ID:** 10656158
- **Project number:** 5R44HL149598-03
- **Recipient organization:** CELLFE INC.
- **Principal Investigator:** Sewoon Han
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $843,224
- **Award type:** 5
- **Project period:** 2019-08-15 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10656158

## Citation

> US National Institutes of Health, RePORTER application 10656158, Efficient and scalable microfluidic gene delivery to hematopoietic stem cells to treat blood diseases (5R44HL149598-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10656158. Licensed CC0.

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