Gene therapy is the insertion of genes into an individual’s cells and tissues to treat a disease. Although the technology is still in its infancy, the successes to date represent a groundbreaking opportunity to improve human health.

Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Researchers are still studying how and when to use gene therapy, but the results to date have been very encouraging.

Our Current Gene Therapy Focus

X-linked severe combined immunodeficiency (XSCID) is an inherited disorder of the immune system that occurs almost exclusively in males that diminishes and shortens the lifespan of males. Without treatment, males with X-linked SCID usually do not live beyond infancy.

Our XSCID gene therapy, being developed via a collaboration with St. Jude involves ex vivo lentiviral transduction of patients’ own hematopoietic stem cells with a normal copy of the mutated gene. The process used is analogous to the manufacturing process for our CAR Ts.

To view more details and the current status of our XSCID program, visit the Pipeline page.