SQZ Biotechnologies Presents Regenerative Medicine Data Demonstrating Neuron Generation from Human Pluripotent Stem Cells with Single Delivery of mRNA Transcription Factor

Results Further Establish Company’s Broad Cell Engineering Capabilities and Potential to Direct Cell Fate for Multiple Therapeutic Applications

Findings Presented at 2021 International Society for Stem Cell Research Annual Meeting

WATERTOWN, MA, USA I June 23, 2021 I SQZ Biotechnologies Company (NYSE: SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, today presented preclinical regenerative medicine data showing that the company’s Cell Squeeze® technology can be used to generate neurons from induced human pluripotent stem cells (iPSCs) through the single delivery of an mRNA encoding fate-specifying transcription factor. The new findings build upon recent clinical and pre-clinical research presentations from the company’s Oncology and Autoimmune Disease programs and further demonstrates SQZ’s broad cell engineering capabilities. The regenerative medicine research was presented at the 2021 International Society for Stem Cell Research virtual annual meeting.

“The ability to precisely stimulate key natural signaling pathways through the delivery of one or multiple transcription factors provides broad cell reprogramming potential that could lead to many new cell therapies and offers a foundation for future regenerative medicines,” said Jonathan Gilbert, Ph.D., Vice President and Head of Exploratory Research, SQZ Biotechnologies. “We are excited by these preclinical proof-of-concept data demonstrating that we can rapidly generate neurons with mature markers using optimized, transient mRNA transcription factor expression. This work complements and expands on our earlier published research using SQZ technology to reprogram fibroblasts into iPSCs.”i

Common methods to generate specific cell types from iPSCs include sequential treatment of cells using multiple extracellular signaling molecules, a process that can be lengthy, inefficient, and result in variable purity, or using viral vectors, which have the risk of undesirable genomic integration.

Cell Squeeze® technology can generate reprogrammed cells by controlling the magnitude and timing of single or multiple cell programming factors. Specific cells generated with this process have the potential to translate therapeutically due to the rapid process, lack of viral or genetic manipulations, and use of our clinically validated manufacturing.

Major Findings from Cell Reprogramming Research:

  • Rapid Induction of Neuronal Differentiation:Within 24 hours after the single squeeze delivery of Neurog2 mRNA, a key neuronal transcription factor, expression of the early neuronal marker TUJ1 was observed. Additionally, two key mature neuronal makers, MAP2 and NeuN, were identified through continued longitudinal observation.
  • Potent Target Gene Expression:SQZ scientists developed an optimized mRNA sequence for Neurog2 that showed a 10,000-fold increase in expression of downstream target genes from a single squeeze delivery.
  • Multiplexed mRNA Capability: The Cell Squeeze® process also co-delivered two mRNA cargos simultaneously, specifically Neurog2 and puromycin N-acetyltransferase, a commonly used enzyme for cell purification. Mature neuronal marker expression and contrast images showed the selective survival of the induced cells, demonstrating that multiple functional mRNAs can be delivered into iPSCs.
  • Maintaining Cell Health: SQZ investigators demonstrated that the Cell Squeeze process alone does not disrupt normal iPSC gene and protein expression. Findings verified that 24 hours after processing the iPSCs through a SQZ™ chip, there was no significant difference in the expression of 32 common housekeeping genes as well as no disruption in pluripotent marker gene and protein expression.

Together these data show the potential of the Cell Squeeze® technology to rapidly reprogram cells for regenerative medicine applications.

Poster Presentation Details

Title: Microfluidic cell squeezing enables the in vitro generation of induced neurons from human pluripotent stem cells through non-viral transcription factor delivery
Abstract Number: 318
Poster Session: Poster Session 3, New Technologies; Wednesday, June 23, 2021, 12:00 PM - 1:00 PM EDT

About SQZ Biotechnologies

SQZ Biotechnologies is a clinical-stage biotechnology company focused on unlocking the full potential of cell therapies for patients around the world and has active programs in Oncology, Autoimmune and Infectious Diseases, and additional exploratory initiatives to support future pipeline growth. The company’s proprietary Cell Squeeze® technology offers the unique ability to deliver multiple biological materials into many patient cell types to engineer what we believe can be a broad range of potential therapeutics. With demonstrated production timelines under 24 hours and the opportunity to eliminate preconditioning and lengthy hospital stays, our approach could significantly broaden the therapeutic range and accessibility of cell therapies. The company’s first therapeutic applications seek to generate target-specific immune responses, both in activation for the treatment of solid tumors and infectious diseases, and in immune tolerance for the treatment of unwanted immune reactions and autoimmune diseases. For more information, please visit www.sqzbiotech.com.

SOURCE: SQZ Biotechnologies

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