Cell Reprogramming & Gene Editing

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Cells contain two programs that work together to determine their behavior. A genetic program, written in the sequence of a cell’s DNA, encodes information about each protein that the cell produces, while an epigenetic program not encoded in the sequence of a cell’s DNA determines other heritable characteristics, including the amount of each protein produced. These two programs ensure that the trillions of cells that make up a complex multicellular organism act in concert to perform the many specialized functions needed for the organism to survive and grow.

We develop technologies to re-write these two cellular programs to treat disease and improve the way cells function.

Our 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.

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.

mRNA Cell Reprogramming

Conventional reprogramming methods (e.g., using Sendai virus or episomal vectors) can result in very low-efficiency reprogramming, can select for cells with abnormal growth characteristics, and can leave traces of the vector in reprogrammed cells. Our scientists developed a technology for reprogramming cells that uses mRNA to express reprogramming factors¹. mRNA Cell Reprogramming is protected […]

Cell Reprogramming Medium

Conventional cell-culture media, including serum-free and animal component-free media, can result in very low-efficiency cell reprogramming. Our scientists developed a novel cell-culture medium that can enable dramatically higher efficiency cell reprogramming than conventional media, including when mRNA is used to express reprogramming factors².

mRNA Vectorization of Gene-Editing Proteins

Gene-editing proteins can be used to inactivate, repair, or insert sequences in living cells. Conventional approaches using plasmids or viruses to express gene-editing proteins can result in low-efficiency editing and unwanted mutagenesis when an exogenous nucleic acid fragment is inserted at random locations in the genome. Our scientists developed a technology […]

Combined mRNA Cell Reprogramming & Gene Editing

Combining gene editing with cell reprogramming enables the generation of gene-corrected personalized cell therapies, models of genetic disease, engineered cell therapies, including allogeneic (i.e., immuno-nonreactive or “stealth”) cell therapies, including CAR-T, CAR-NK, and engineered mesenchymal stem cell (MSC) therapies for regenerative medicine, wound-healing, inflammatory and auto-immune diseases, and tumor-targeting applications. Our scientists developed a […]

Chromatin Context-Sensitive Gene-Editing Endonuclease

Many uses of gene-editing proteins are limited by their specificity (“off-target” effects), which can be of particular concern when these proteins are delivered or expressed in vivo. Our scientists developed a novel high-specificity gene-editing endonuclease that exhibits high efficiency on target cutting and enhanced sensitivity to the chromatin context of the target site³. The protein comprises […]

Temperature-Tunable Gene-Editing Endonuclease

Conventional approaches to in vivo gene editing using viral vectors or lipid nanoparticles can result in limited tissue-targeting. Our scientists developed a novel high-specificity gene-editing endonuclease that exhibits high-efficiency ontarget cutting at physiological and sub-physiological temperatures⁴. This technology can be used to target cutting activity to specific […]

Gene-Editing Endonuclease with Nickase Functionality

Many gene-editing strategies involve the use of nucleases to generate targeted double-strand breaks in genomic DNA, which can cause cytotoxicity and off-target effects, limiting clinical translation. Our scientists developed a novel high-specificity gene-editing endonuclease with nickase functionality that enables high-efficiency on-target single-strand breaks in genomic DNA and scarless targeted gene insertion in cells⁵. This technology […]

Gene-Edited Allogeneic Cell Therapies

Allogeneic cell therapies can enable “off-the-shelf” treatment options. Conventional allogeneic cell therapy approaches can result in rejection, low potency, and in the case of allogeneic immune cell therapy, graft-versushost disease. Our scientists developed a technology that uses gene editing to address limitations of conventional approaches to allogeneic cell therapy⁶. This technology can be used […]

Directed Differentiation of Gene-Edited Pluripotent Stem Cells

Pluripotent stem cell-derived therapies can involve directed differentiation of pluripotent stem cells to specific cell lineages. In gene-edited cells, directed differentiation can be hindered by transgene-encoded proteins. Our scientists developed a technology for differentiating gene-edited pluripotent stem cells comprising a transgene that uses small-molecule inhibitors of the transgene-encoded protein⁷.