【Portfolio】SQZ Biotechnologies Announces Integrated Point-of-Care Cell Therapy Manufacturing System Demonstrated Comparable or Better Performance Than a More Conventional Clean Room-Based Process

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  • Average Processing Time Per Batch Less Than 6 Hours – a 55% Reduction with Comparable or Improved Product Metrics

  • First Clinical Use Planned for SQZ® TACs in Celiac Disease with IND Submission Anticipated in 1H 2023

  • Preclinical Data on Hematopoietic Stem Cell Engineering and Planned Commercial Research-Use-Only System Presented at ASGCT 2022 Support Potential for Broad Future Point-of-Care Manufacturing Implementation


May 18, 2022 06:45 AM Eastern Daylight Time


WATERTOWN, Mass.--(BUSINESS WIRE)--SQZ Biotechnologies (NYSE: SQZ) announced today that the company’s first generation, integrated point-of-care (POC) cell therapy manufacturing system demonstrated superior process performance and comparable or improved product specifications relative to current clean room-based processes used in clinical development.

 

The non-clinical studies showed that the SQZ® POC system could produce the company’s red blood cell derived SQZ® Activating Antigen Carrier (AAC) therapeutic candidate for the treatment of HPV16 positive solid tumors in half the time (under 6 hours), with 90 percent fewer operator hours, while producing comparable drug product. The system is also compatible with hematopoietic stem cells and peripheral immune cells, including B cells, T cells, NK cells, and Monocytes. Data was presented today at the 2022 American Society for Gene and Cell Therapy (ASGCT) annual meeting.

“We are tremendously excited with the cell therapy production data from our prototype point-of-care manufacturing system, which has the potential to produce cell therapies using a range of cell types,” said Howard Bernstein, M.D., Ph.D., Chief Scientific Officer at SQZ Biotechnologies. “Our current clinical manufacturing process is under 24 hours - comparatively faster than other cell therapy manufacturing approaches. Our integrated point-of-care platform represents a further leap with faster production in a closed system that could eliminate the need for an ISO 7 clean room. We look forward to its planned first clinical use in our red blood cell derived SQZ TAC program for celiac disease.”

The company anticipates a SQZ® TAC IND submission for celiac disease in the first half of 2023. Celiac disease impacts a large patient population globally and it, like many other diseases, could benefit from a scalable, decentralized cell therapy manufacturing model. Manufacturing the first SQZ® TAC clinical candidate on the SQZ® POC system will further the development of this decentralized model and support potential future use of the POC system across disease areas.

The SQZ® POC manufacturing system integrates and automates, among other things, cell isolation, cell washing, Cell Squeeze® intracellular delivery technology, and product filling. The system is designed to process patient material within a closed, single-use sterile disposable kit. The system architecture offers the potential to be operated outside of an ISO 7 clean room typically required of cell therapies—which could ultimately allow cell therapy manufacturing by SQZ® POC systems in a variety of care settings.

In addition to the SQZ® POC presentation at ASGCT, preclinical data were presented on the first study findings of the planned commercial research-use-only (RUO) microfluidic delivery system. The RUO presentation highlighted the gentle, yet efficient, nature of the intracellular delivery system. Data demonstrated the ability to effectively deliver and express mRNA in unactivated T and NK cells as well as to gene edit unactivated T cells with limited impact to viability and downstream function.

Cell Squeeze® gene editing of autologous hematopoietic stem cells (HSCs) was also presented at the conference. The HSC editing preclinical data demonstrated that the Cell Squeeze® platform could efficiently deliver mRNA and RNPs into HSCs, a sensitive cell type that has been limited previously by toxicity, cargo restrictions, and scalability using other cell engineering approaches. Analysis of the Cell Squeeze® processed HSCs determined that their phenotype was maintained in vitro and in vivo illustrating its broad potential for use in certain rare and genetic disease indications.

ASGCT POINT-OF-CARE MANUFACTURING DATA HIGHLIGHTS

  • Average processing time per batch run reduced by 55 percent, from 12 hours 45 minutes to 5 hours 45 minutes

  • Required person-hours reduced by 90 percent, from 150 hours to 15 hours

  • Batches showed comparable attributes, including drug and adjuvant content, cell purity, and interferon gamma secretion (a measure of T cell activation)

 

ASGCT RESEARCH-USE-ONLY (RUO) SYSTEM DATA HIGHLIGHTS

  • RUO system demonstrated robust CRISPR-Cas9 editing in unactivated human T cells, and efficient delivery and expression of mRNA to unactivated T and NK cells

  • The gentle nature of the RUO system’s intracellular delivery mechanism resulted in limited impact to T cells. Unactivated T cells retained their unactivated phenotype and demonstrated similar activation and expansion compared to untreated samples

  • In contrast, electroporation of unactivated T cells resulted in spontaneous increases in pro-inflammatory cytokine transcripts and secretion, independent of TCR engagement

 

ASGCT HEMATOPOIETIC STEM CELL (HSC) DATA HIGHLIGHTS

  • Cell Squeeze® technology efficiently delivered mRNA and RNPs into HSCs; 90 percent of HSCs expressed transferred mRNA, and high editing rates were achieved using B2M and CCR5 targeting RNPs

  • HSC phenotype was maintained following Cell Squeeze® treatment compared to controls as determined by microarray analysis, CFC assay, and NBSGW mouse engraftment study

  • Cell Squeeze® treated HSCs could be transferred immediately following treatment, and demonstrated superior engraftment versus electroporation

About SQZ Biotechnologies

 

SQZ Biotechnologies Company 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, as well as 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 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 autoimmune diseases. For more information, please visit www.sqzbiotech.com.