Factor Bioscience develops technologies to advance the study and treatment of disease.
Our gene-editing technologies enable the precise deletion, insertion, and repair of DNA sequences in living cells, while our 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.
Our disease-focused technologies can be used to create treatments for cancer, genetic, infectious, and degenerative diseases.
Our nucleic-acid delivery technologies enable efficient ex vivo delivery of mRNA encoding gene-editing proteins and reprogramming factors, including to primary cells, insertion of exogenous sequences into genomic safe-harbor loci, and efficient in vivo delivery of mRNA to the brain, eye, skin, and lung.
Gene Editing & Cell Reprogramming
Our gene-editing technologies enable the precise deletion, insertion, and repair of DNA sequences in living cells, while our 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.
Our disease-focused technologies can be used to create treatments for cancer, genetic, infectious, and degenerative diseases.
Our nucleic-acid delivery technologies enable efficient ex vivo delivery of mRNA encoding gene-editing proteins and reprogramming factors, including to primary cells, insertion of exogenous sequences into genomic safe-harbor loci, and efficient in vivo delivery of mRNA to the brain, eye, skin, and lung.
Macrophages’ ability to infiltrate solid tumors and engage in both direct killing of cancer cells and recruitment of other immune cells has made them a promising target for development of next-generation cancer immunotherapies. The innate ability of macrophages to ingest foreign genetic material also facilitates their engineering with formulated nucleic acids, including mRNA. The oncoantigen […]
Lipid nanoparticles (LNPs) containing cationic or ionizable lipids offer several advantages compared to other vehicles for nucleic acid delivery and have seen expanded clinical use with the introduction the COVID-19 mRNA vaccines and in treatments for genetic diseases. However, poor targeting, insufficient cellular uptake, and low endosomal release currently limit the use of lipid nanoparticles […]
Indoleamine 2,3-dioxygenase 1 (IDO1) is an inducible, heme-containing enzyme that is critically involved in tryptophan catabolism and known to be a prominent immune regulator. Cell therapies with increased IDO1 expression are of high interest for a variety of indications, including autoimmune disorders, inflammatory diseases, transplant recovery, and wound healing. In particular, iPSC-derived mesenchymal stem cells […]
Macrophages’ ability to infiltrate solid tumors and engage in both direct killing of cancer cells and recruitment of other immune cells has made them a promising target for development of next-generation cancer immunotherapies. The innate ability of macrophages to ingest foreign genetic material also facilitates their engineering with formulated nucleic acids, including mRNA. The oncoantigen […]
Novel Ionizable Lipids Derived from 2-Hydroxypropylamine and Spermine for mRNA-LNP Delivery
Lipid nanoparticles (LNPs) containing cationic or ionizable lipids offer several advantages compared to other vehicles for nucleic acid delivery and have seen expanded clinical use with the introduction the COVID-19 mRNA vaccines and in treatments for genetic diseases. However, poor targeting, insufficient cellular uptake, and low endosomal release currently limit the use of lipid nanoparticles […]
Indoleamine 2,3-dioxygenase 1 (IDO1) is an inducible, heme-containing enzyme that is critically involved in tryptophan catabolism and known to be a prominent immune regulator. Cell therapies with increased IDO1 expression are of high interest for a variety of indications, including autoimmune disorders, inflammatory diseases, transplant recovery, and wound healing. In particular, iPSC-derived mesenchymal stem cells […]