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SQZ Biotechnologies Announces $2 Million Grant From the National Institutes of Health to Develop a Novel, Scalable Cell Replacement Therapy for Parkinson’s Disease

Project to Focus on Creating Dopamine-Producing Neurons Using Direct mRNA-based Reprogramming of Immune Cells

SQZ Biotechnologies, focused on unlocking the full potential of cell therapies for multiple therapeutic areas, announced that it has been awarded a $2 million SBIR Phase II grant from the National Institute of General Medical Sciences, a division of the National Institutes of Health. Awarded through a competitive process, the two-year grant will support the development of cell engineering methods that are designed to reprogram a patient’s own immune cells directly into dopamine-producing neurons, a potential novel therapeutic approach for the treatment of Parkinson’s disease.

“Directly creating dopamine-producing neurons by reprogramming a patient’s own immune cells would be a major breakthrough and could support a new Parkinson’s disease treatment paradigm,” said Jonathan Gilbert, Ph.D., Vice President and Head of Exploratory Research at SQZ Biotechnologies. “Unlike alternative allogeneic cell replacement approaches in development for Parkinson’s disease, by using a patient’s own cells, treatment might not require chronic immunosuppression. Moreover, in altering cell fate with RNA-based cell engineering methods, no changes to the genome are likely to occur that could carry long-term risks.”

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Reprogramming a patient’s cells to replace lost or diseased cells has significant therapeutic potential. Beyond Parkinson’s Disease, applications for cell replacement therapies include Multiple Sclerosis and Type 1 diabetes. However, traditional expensive, time-intensive, and inefficient cell reprogramming methods has hindered clinical progress and patient impact.

At the 2021 International Society for Stem Cell Research annual meeting, the company presented preclinical data showing that proprietary Cell Squeeze® technology can be used to generate neurons from induced human pluripotent stem cells through the delivery of an mRNA encoding for a fate-specifying transcription factor.

With the support of the NIH grant, and building upon our experience in multiplex engineering of immune cells, SQZ researchers will attempt to generate dopaminergic neurons directly from somatic cells. The Cell Squeeze® technology may allow for a unique complex combination of transcription factors, dosing, and timing.

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