Gene Editing & Cell Reprogramming
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.
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 that uses mRNA to […]
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 site1. The protein […]
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 factors2. mRNA Cell Reprogramming is protected by […]
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) cell therapies for regenerative medicine, wound-healing, inflammatory and auto-immune diseases, and tumor-targeting applications. Our scientists developed a technology […]
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 factors3. The Cell Reprogramming Medium is protected by U.S. Patent Number 9,127,248, as […]
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 sub-physiological temperatures4. This technology can be used to target cutting activity to specific organs and tissues, allowing higher doses, minimizing systemic effects, and […]
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-versus-host disease. Our scientists developed a technology that uses gene editing to address limitations of conventional approaches to allogeneic cell therapy5. This technology can be used to […]