SBIR Phase II: Development of an Intracellular Delivery Platform for Accelerated Drug Discovery Using Genetically Engineered Human Immune Cells

Period of Performance: 04/15/2016 - 03/31/2018

$750K

Phase 2 SBIR

Recipient Firm

SQZ Biotechnologies Company, Inc.
WATERTOWN, MA 02472
Firm POC, Principal Investigator

Abstract

The broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase II project will be the development of technology for the intracellular delivery of biomolecules directly into cells. This microfluidics-based platform has the potential to become an enabling technology for intracellular delivery, which may be used to accelerate drug discovery R&D by allowing reliable, efficient delivery of diverse material classes without having to engineer the material or the cell to natively uptake these molecules. Such capabilities could allow pharmaceutical companies to assess the efficacy of drug candidates faster than ever before, especially with integration into high-throughput robotic workflows that are already well-established and efficacious. The technology could dramatically reduce the time to market for new drugs by decoupling determination of a candidate's activity from the cell's affinity for the molecule. It also could facilitate a deeper understanding of biological processes and pathways. Initial studies with leading drug developers and academic laboratories towards this goal have been very encouraging, and, in the future, the platform could potentially enable robust engineering of cell function for cell-based therapies targeting a diversity of diseases including influenza, cancer, and even autoimmune disorders. This SBIR Phase II project proposes the continued development of the intracellular delivery technology to address relevant applications in drug discovery R&D. New drug discovery is often hampered by the inability of membrane-impermeable drug candidates to enter the cell cytosol, necessitating exogenous materials for delivery such as strong electric fields or viral vectors. However, these materials tend to cause off-target effects or toxicity, presenting a need for a technology that can facilitate delivery without altering post-treatment cellular function. The goal of this project is to demonstrate a platform geared towards market adoption of microfluidic hardware as the standard method for transfection and intracellular delivery. During Phase II, the platform will be fully-characterized, validated, and verified in order to produce the consistent, repeatable results necessary to achieve market entry. In addition, research is planned to demonstrate the ability of the platform to support drug discovery R&D by developing the use of the CRISPR/Cas9 gene editing system for use with this intracellular delivery technology.