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SQZ Biotech Awarded $2 Million to Develop Parkinson Disease Cell Therapy

The company recently received IND clearance for a solid tumor cell therapy.

The National Institute of General Medical Sciences at National Institutes of Health (NIH) has awarded a $2 million grant to SQZ Biotechnologies to support the development of a novel cell therapy for the potential treatment of Parkinson disease (PD).

“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,” Jonathan Gilbert, PhD, vice president and head, exploratory research, SQZ Biotechnologies said in a statement.

The 2-year grant will support SQZ’s efforts to develop cell engineering methods to reprogram patients’ immune cells into dopamine-producing neurons. SQZ will leverage its Cell Squeeze technology, which temporarily disrupts cell membranes and allows the entry of biologic cargo. The technology is designed to allow for a unique complex combination of transcription factors, dosing, and timing.

SQZ's approach differs from many emerging cell therapies that rely on viral vectors and multi-step manufacturing processes. The company hopes that this relatively simple approach may cause less dysfunction to the altered cells allow and for a wider application of cell therapies. Researchers will attempt to generate dopaminergic neurons directly from somatic cells.

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“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,” Gilbert added.

The FDA cleared SQZ’s investigational new drug application for SQZ-eAPC-HPV in January 2022, an mRNA-based cell therapy created with the Cell Squeeze technology. SQZ will soon initiate the COMMANDER-001 phase 1/2 clinical trial of the therapy in patients who have HPV16+ solid tumors, including head and neck, cervical, and anal cancers, and have progressed following standard therapies.

SQZ presented positive data on their Cell Squeeze technology at the 2021 International Society for Stem Cell Research annual meeting, June 21, 2021, that showed that the 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.

CGTLive previously spoke with Armon Sharei, PhD, the founder and chief executive officer of SQZ, to learn more about its Cell Squeeze technology and specifically its Antigen Presenting Cell Platform created via Cell Squeeze that can be used to activate killer T-cell responses. He discussed how the ease of manufacturing offered by the Cell Squeeze technology allows for easy switches between antigens in creating APCs that can work against different targets. Watch the video below.

REFERENCES
1. SQZ Biotechnologies announces $2 million grant from the National Institutes of Health to develop a novel, scalable cell replacement therapy for Parkinson’s disease. News release. SQZ Biotechnologies. March 21, 2022. https://investors.sqzbiotech.com/news/news-details/2022/SQZ-Biotechnologies-Announces-2-Million-Grant-From-the-National-Institutes-of-Health-to-Develop-a-Novel-Scalable-Cell-Replacement-Therapy-for-Parkinsons-Disease/default.aspx
2. SQZ Biotechnologies announces FDA clearance of investigational new drug (IND) application for SQZ-eAPC-HPV, a novel mRNA-based cell therapy for the treatment of HPV16 positive solid tumors. News release. SQZ Biotechnologies. January 24, 2022. https://investors.sqzbiotech.com/news/news-details/2022/SQZ-Biotechnologies-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-SQZ-eAPC-HPV-a-Novel-mRNA-based-Cell-Therapy-for-the-Treatment-of-HPV16-Positive-Solid-Tumors/default.aspx