In Vivo Gene Editing

Several companies are experimenting on human subjects to develop gene editing that is performed inside the body. Such therapies differ from a recently approved treatment for sickle-cell anemia that removes bone marrow from the body before editing the genetic material and reintroducing it into the patient.

Intellia Therapeutics and Regeneron Pharmaceuticals are in clinical trials to use gene editing to knock out a gene that causes fibrous protein clumps, which can accumulate in organs and nerves, increasing the risk of heart failure and other serious complications. Verve Therapeutics is enrolling patients to test a gene-editing protocol designed to reverse the effects of a genetic disease that leads to severely elevated cholesterol and increased risk of early-onset cardiovascular disease.

One caveat: These therapies carry the risk of editing unrelated genes.