Disease-Focused Technologies

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 mRNA encoding […]

Gene-Editing Therapies for 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 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 […]

mRNA Therapies for 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 HIV

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 HIV is […]

Gene-Editing Therapies for 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 alpha-synuclein to prevent aggregation of the encoded protein in affected […]

Gene-Editing Therapies for 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 Chronic Pain is protected by a pending international patent application (with plans to […]

Gene-Editing Therapies for 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 […]