Ian Hay1, Abigail Blatchford1, Christopher B. Rohde1, Matthew Angel1
1Factor Bioscience Inc., Cambridge, MAMol Ther, Vol 31, No 4S1, 2023
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 […]
Ariadna Lubinus1, Joseph Pisano1, Elizabeth Belcher2, Christopher B. Rohde1, Matthew Angel1,2
1Factor Bioscience Inc., Cambridge, MA, 2Eterna Therapeutics Inc., Cambridge, MA
Mol Ther, Vol 31, No 4S1, 2023
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 […]
Raven Dance Klee1, Elizabeth Belcher1, Taeyun Kim2, Kyle Garland1, Christopher B. Rohde2, Matthew Angel1,2
1Eterna Therapeutics Inc., Cambridge, MA, 2Factor Bioscience Inc., Cambridge, MAMol Ther, Vol 31, No 4S1, 2023
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 […]
Abigail J. Blatchford1, Mackenzie Parmenter1, Ian Hay1, Christopher B. Rohde1, Matthew Angel1
1Factor Bioscience Inc., Cambridge, MA
Induced pluripotent stem (iPS) cell therapies have the potential to treat a wide variety of devastating diseases. iPS$ cell-derived lymphocytes (e.g., T cells and NK cells) engineered to express targeting molecules such as chimeric antigen receptors (CARs) have shown clinical promise to treat hematological malignancies. More recently, iPS cell-derived myeloid cells are being developed to […]
Claire Aibel1, I. Caglar Tanrikulu1, Christopher B. Rohde1, Matthew Angel1
Genome-editing technology provides a means of modifying genes in living cells, and is being explored for the development of therapies to treat cancer and a variety of genetic disorders. Gene-editing proteins can be used to create single- and double-strand breaks at specific genomic sites for knocking out a gene or, when combined with an exogenous […]
Taeyun Kim1, I. Caglar Tanrikulu1, Christopher B. Rohde1, Matthew Angel1
Mesenchymal stem cells (MSCs) are a promising cell-therapy platform with the potential to treat a diverse array of diseases due to their immunomodulator properties – properties which can be enhanced through gene editing. Gene editing autologous or donor- derived MSCs is challenging due to the non-clonal nature of these cell sources, and associated risks of […]
Elizabeth Belcher1, Joseph Pisano2, Ariadna Lubinus2, Kyle Garland1, Christopher B. Rohde2, Matthew Angel1,2
1Eterna Therapeutics Inc., Cambridge, MA, 2Factor Bioscience Inc., Cambridge, MA
In recent years, lipid-based mRNA delivery has become the gold standard method for inducing exogenous protein production in vivo as evidenced by the success of the COVID-19 mRNA vaccines. While intramuscular injections are ideal for vaccine applications, intravenous injections are generally more suitable for achieving broad internal distribution of therapeutic payloads. Following systemic administration, blood […]
Elizabeth Belcher1, Taeyun Kim1, Kyle Garland1, Christopher B. Rohde2, Matthew Angel1,2
Many gene editing strategies involve the use of nucleases to generate targeted double-strand breaks (DSBs) in genomic DNA, which is often associated with cytotoxicity and off-target effects that can prevent clinical translation. Such undesirable outcomes have led to the development of gene-editing nickases, which instead create targeted single-strand breaks (SSBs) that favor high- fidelity repair […]
Mol Ther, Vol 30, No 4S1, 2022
Gene editing technology, which enables the precision modification of DNA in living cells, is being developed for the treatment of various diseases, including genetic diseases and cancer. Gene editing commonly employs sequence-specific endonucleases to create double strand breaks in genomic DNA, and relies on the cell’s DNA repair mechanisms to apply the desired changes. Precise […]
Ian Hay1, Christopher B. Rohde1, Matthew Angel1
Cancer immunotherapy has advanced rapidly over the past two decades, with several autologous chimeric antigen receptor (CAR)-T cell therapies approved for the treatment of hematologic cancers. However, CAR-T cells have shown limited activity against solid tumors, in part due to the immunosuppressive nature of the tumor microenvironment preventing CAR-T cell infiltration. This has led to […]
