Factor Bioscience Announces Publication of Preclinical Results of Aerosolized mRNA For the Treatment of Pneumonia

February 28, 2023

-mRNA encoding immunomodulatory proteins IκBα-SR and SOD3 ameliorated symptoms in bacterial pneumonia model

-Factor is investigating nebulized mRNA formulations for other therapeutic indications and next-generation vaccine applications

CAMBRIDGE, Mass., February 28, 2023 /PRNewswire/ — Factor Bioscience Inc. (“Factor”), a Cambridge-based biotechnology company focused on developing mRNA and cell-engineering technologies, announced the results of preclinical testing of aerosolized mRNA formulations currently under development for the treatment of pneumonia-associated lung inflammation. The study was led by Factor’s collaborator, Dr. Daniel O’Toole, Senior Research Fellow, Senior Lecturer in Lung Regenerative Medicine and Principal Investigator in the CÚRAM and REMEDI institutes at the University of Galway.

“Pneumonia is a debilitating disease in which infection causes inflammation of lung tissue, often leading to pulmonary edema, and in severe cases, acute respiratory distress syndrome (ARDS) and death,” said Dr. O’Toole. “There are currently no effective specific therapies for ARDS, and the mortality rate remains high. The ability to directly express anti-inflammatory proteins in lung tissue makes aerosolized mRNA an attractive candidate therapy for pneumonia. In particular, the speed with which cells express proteins when treated with mRNA is particularly important in the context of a rapid-onset disease such as ARDS.”

These results are the latest in the long-standing collaboration between Factor and CÚRAM, the Science Foundation Ireland Centre for Research in Medical Devices, which is now in its sixth year. Factor and CÚRAM have multiple active collaborative projects, including projects focused on the development of mRNA-reprogrammed induced pluripotent stem (iPS) cell-derived therapies for the treatment of pulmonary diseases as well as next-generation aerosolized mRNA vaccines.

“We are incredibly proud of our collaboration with CÚRAM, and are excited about the results of this study,” said Dr. Christopher Rohde, Chief Technology Officer of Factor. “mRNA offers unmatched flexibility as a vector for expressing therapeutic proteins safely and reliably, and the results of this study support the use of aerosolized mRNA formulations for expression of therapeutic proteins in the lung. We are excited to continue this work with the team at CÚRAM and advance product candidates based on these results towards clinical development.”

The results of this study were published last week in Nucleic Acid Therapeutics: https://doi.org/10.1089/nat.2022.0049

About Factor Bioscience

Founded in 2011, Factor Bioscience develops technologies for engineering cells to advance the study and treatment of disease. Factor’s gene-editing technologies enable the precise deletion, insertion, and repair of DNA sequences in living cells to correct disease-causing mutations, make cells resistant to infection and degenerative disease, modulate the expression of immunoregulatory proteins to enable the generation of durable allogeneic cell therapies, and engineer immune cells to more effectively fight cancer. Factor’s cell-reprogramming technologies enable the generation of clonal lines of pluripotent stem cells that can be expanded and differentiated into any desired cell type for the development of regenerative cell therapies. For more information, visit www.factorbio.com.

About CÚRAM

CÚRAM is the Science Foundation Ireland Centre for Research in Medical Devices, based at the University of Galway. CÚRAM’s innovative approach incorporates biomaterials, drug delivery, cell-based technologies, glycosciences and device design to enhance, develop and validate both traditional and new combinational medical devices from molecular design stage to implant manufacturing. For more information, visit www.universityofgalway.ie/curam/