Disease-Focused Technologies

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Human disease is caused by cellular dysfunction. Genetic diseases and cancer result from errors in a cell’s genetic program (either in the organism itself or its ancestors), while infectious, autoimmune, and degenerative diseases result from cells’ susceptibility to pathogenic, environmental, and metabolic factors. While every disease is unique, the link to cellular function provides opportunities to intervene in the disease state using cell engineering technologies.

We develop technologies that can be used to create treatments for cancer, genetic, infectious, and degenerative diseases.

Our cell engineering technologies can enable the unmasking of immunosuppressive (i.e., “cold”) tumors, correction of disease-causing mutations, inactivation of cell surface receptors to generate infectionresistant cells, inactivation of genes responsible for degenerative proteopathies, and inactivation of ion channels responsible for aberrant neuronal activity associated with chronic pain.

mRNA Therapies for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

Dystrophic Epidermolysis Bullosa (DEB) is a disease caused by mutations in the COL7A1 gene, which encodes collagen 7. Many DEB patients have extremely delicate skin, and in the most severe cases, live with painful blisters and open wounds over large areas of their bodies. Our scientists developed a method for treating dystrophic epidermolysis bullosa […]

Gene-Editing Therapies for the Treatment of Epidermolysis Bullosa (EB)

Epidermolysis Bullosa (EB) is a collection of genetic diseases caused by mutations in genes that are important for normal skin function. Many EB patients have extremely delicate skin, and in the most severe cases, live with painful blisters and open wounds over large areas of their bodies. Our scientists developed a method for treating […]

Gene-Editing Therapies for the Treatment of Parkinson’s Disease

Parkinson’s disease is associated with the accumulation of alpha-synuclein aggregates in dopaminergic neurons of the substantia nigra pars compacta, leading to cell death, and resulting loss of motor control. Our scientists developed a method for treating Parkinson’s disease by editing the gene that encodes alphasynuclein to prevent aggregation of the encoded protein in affected […]

Gene-Editing Therapies for the Treatment of Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, which encodes dystrophin, a protein normally expressed in skeletal muscle. Our scientists developed a method for treating DMD by using gene-editing proteins to edit the DMD gene to result in the production of a functional form of dystrophin protein. Gene-Editing Therapies for […]

Gene-Editing Therapies for the Treatment of Alpha-1-Antitrypsin Deficiency

Alpha-1-antitrypsin deficiency is a disease caused by mutations in the A1AT gene. Patients with this disease experience progressive liver failure, as well as lung irritation and damage. Most patients with AAT deficiency are homozygous for the Glu342Lys mutation, which causes cells to produce a form of AAT that aggregates into toxic intracellular polymers. Our […]

Gene-Editing Therapies for the Treatment of HIV/AIDS

Human Immunodeficiency Virus uses surface proteins such as CCR5 to infect immune cells. People with a rare natural variant of the CCR5 gene exhibit resistance to HIV infection. Our scientists developed a method for treating HIV using mRNA encoding gene-editing proteins to inactivate CCR5 and/or CXCR4 in hematopoietic cells. Gene-Editing Therapies for the Treatment […]

Gene-Editing Checkpoint Molecule Genes for the Treatment of Cancer

Checkpoint molecule genes act to prevent the immune system from attacking normal cells. In many cancers, the expression of checkpoint molecule genes by cancer cells prevents their destruction by the immune system (a “cold” tumor). Our scientists developed a method for treating cancer by inactivating checkpoint molecule genes in cancer cells using […]

Gene-Editing Therapies for the Treatment of Sickle Cell Disease

Sickle cell disease is caused by a mutation in the HBB gene that results in a lack of functional hemoglobin and sickle-shaped red blood cells that can accumulate in blood vessels and lead to vaso-occlusive crises. Our scientists developed a method for treating sickle cell disease by using mRNA encoding gene-editing proteins in donor […]

Gene-Editing Therapies for the Treatment of Chronic Pain

Chronic pain is a debilitating disorder associated with aberrant neuronal activity. Our scientists developed a method for treating chronic pain by editing genes encoding voltage-gated sodium channels in cells of the central nervous system or peripheral nervous system. Gene-Editing Therapies for the Treatment of Chronic Pain is protected by a pending U.S. patent (with […]

Rapid Prototyping of Gene-Editing Strategies for the Treatment of Cancer

Cancer is a heterogeneous set of diseases that are characterized by diverse cellular phenotypes. This heterogeneity can lead to patient populations responding differently to different therapies. Our scientists developed a method for screening cancer therapies by rapidly prototyping gene-editing strategies using engineered protein-encoding RNA13 Rapid Prototyping of Gene-Editing Strategies for the Treatment of Cancer […]

RNA Vaccines for the Prevention & Treatment of Infectious Diseases & Cancer

mRNA vaccines can be useful for the prevention of certain diseases. However, the design and formulation of the RNA in these vaccines can limit the diseases that they can be used to prevent or treat. Our scientists developed a method for preventing and treating infectious diseases and cancer using RNA vaccines¹⁴. RNA Vaccines […]

Pluripotent Stem Cell-Derived Therapies for the Treatment of Hematological Cancers

Engineered cell therapies can effectively treat certain hematological cancers. However, conventional approaches using viruses to express targeting molecules on autologously derived cells can result in high manufacturing complexity and limited cancer targeting. Our scientists developed a method for treating hematological cancers using engineered pluripotent stem cells¹⁵. Pluripotent Stem Cell-Derived Therapies […]

Pluripotent Stem Cell-Derived Therapies for the Treatment of Solid Tumors

Solid tumors can be refractory to many cytotoxic T cell therapies, such as CAR-T therapy, which can have difficulty infiltrating the tumor microenvironment to exert their cancer-killing function. Our scientists developed a method for treating solid tumors by engineering pluripotent stem cells to create cell therapies that can promote tumor infiltration of cancer-killing immune […]

Pluripotent Stem Cell-Derived Therapies for the Treatment of Genetic Blood Disorders

Genetic blood disorders can be caused by mutations in genes that are important for normal blood cell function. Our scientists developed a method for treating genetic blood disorders by engineering pluripotent stem cells to create cell therapies that can restore the function of the hematopoietic system. Pluripotent Stem Cell-Derived Therapies for the Treatment of […]

Pluripotent Stem Cell-Derived Therapies for the Treatment of Autoimmune Diseases

Autoimmune diseases involve dysregulation of one or more aspects of a patient’s immune system, often leading to inflammation that can ultimately result in irreversible tissue and organ damage. Our scientists developed a method for treating autoimmune and inflammatory diseases by engineering pluripotent stem cells to create cell therapies that can restore the function of […]