OUR APPROACH | Gene Therapy

A genetic disease is type of disorder caused by a deviation or “misspelling” in the genome of an individual that results in a physiological impairment.

Genetic diseases are often rare and life threatening with few – if any – adequate treatments available to the patient and their families. While there exist a vast number of genetic diseases, a few common examples include cystic fibrosis, hemophilia, muscular dystrophy, and sickle-cell disease.

“Traditional” gene therapy has the potential to overcome certain genetic diseases by adding a functional copy of a gene that is missing or defective in the patient. Unfortunately, this approach can only be applied to a subset of genetic diseases and is rarely a permanent cure.

Gene therapy via genome editing takes this approach a significant step further by correcting the genetic problem or “misspelling” at the source – the genome – to permanently cure the disease. This may be accomplished by inserting a missing gene into the genome, removing a disease-causing gene, excising a repeated, diseased segment of DNA, or by fixing the “spelling” of an incorrectly coded gene.

Precision BioSciences is focused on applying its ARCUS genome editing technology to overcome genetic diseases that have a clearly established genetic cause and where there exist no adequate treatments for the disorder. Our hope is that we can utilize this approach to address the unmet needs of patients and families dealing with devastating and, as yet, untreatable diseases.